January 29, 2010 Senator Charles E. Grassley Ranking Member Committee on Finance U.S. Senate Washington, D.C. C/o Brian Downey Brian_Downey@finance-rep.senate.gov Dear Senator Grassley: This is in response to your letter dated December 7, 2009, with regard to funding that pharmaceutical, medical device companies, foundations established by these companies or the insurance industry have provided to the National Organization for Rare Disorders (NORD) since January 2006. As the national public voice and advocate for the rare disease community, NORD fully supports the concept of transparency in disclosing the sources of its funding and, more generally, in public policies that favor disclosure. In fact, in the July 2008 edition of its NORD On-Line newsletter, NORD published an article entitled "Chuck Grassley: A Senate Health Crusader" strongly supporting your efforts on behalf of transparency and noting that you were "demanding reforms that should make the healthcare system more equitable to consumers." (See attachment.) NORD has been recognized for its appropriate utilization of donated funds. For the past six consecutive years, NORD has received the highest (4-star) rating for sound fiscal management from Charity Navigator, a leading independent evaluator of charities. We regularly re-evaluate our policies to assure that the various sources of our funding are well understood by our patient and public constituencies. In fact, we are in the process of developing more transparent disclosure policies with regard to all charitable and philanthropic contributions we receive. We have processes in place to assure that the philanthropy we receive from any source does not influence the information we provide or the positions we take on public policy or legislative issues, which are focused on the patients we serve. NORD's commitment to transparency and disclosure stems from our mission and our heritage. As the primary advocate of the rare disease patient community, NORD strives to focus public attention on the need for more funding for research, for access to treatments, and for fair and reasonable insurance and reimbursement options. We also provide services to patients and their families, ranging from helping them find resources for daily life issues to running patient assistance programs that enable patients in need to access treatments they cannot afford. NORD is a unique federation of voluntary health organizations and individuals who collectively represent the 30 million American patients with rare diseases, defined by federal law as diseases affecting 200,000 or fewer patients. NIH estimates there are nearly 7,000 rare diseases. 1 NORD was founded in 1983 from a coalition of patient organizations that advocated for the enactment of the Orphan Drug Act - a law that has served our country very well. This law aimed to address the challenge posed by the lack of development of medical products for rare diseases. One of the goals of the law was to establish a partnership among the patient community, the pharmaceutical industry, academic scientists and the federal government to advance the development of innovative medical products. The law provided for financial incentives to entice drug companies into developing treatments for small populations of patients. Fortunately, the Congress was able to reach a consensus on how to provide the economic incentives needed to encourage orphan drug development. Since the enactment of the law, more than 340 drugs and biological products for rare diseases have been brought to market. In contrast, in the decade before 1983 fewer than ten products developed by the pharmaceutical industry came to market. For details on incentives please call FDA's Office of Orphan Product Development at (301) 8273666 or visit the website www.fda.gov. With this brief background on NORD, in reference to your letter please find enclosed answers: Question #1: Please describe the policies for accepting industry funding and whether or not NORD allows companies to place restrictions or provide guidance on how funding will be spent. Answer #1: NORD does have policies in place in regards to accepting industry funding for all of its programs and services. NORD does not allow industry to place restrictions or provide guidance on the utilization of funds. Any funds raised from industry sources as unrestricted grants are utilized at NORD's discretion, typically to support general operating expenses for NORD, and the list of supporters is posted on NORD's website. For programs that do utilize restricted funds, the funds are directed strictly for administering the specific programs without any influence or guidance from the industry sponsor and in accordance with OIG guidelines and prudent policies and procedures for each program. (Details included as part of Answer #2) Question #2: If NORD allows companies to place restrictions on industry funding, then please explain all restrictions and/or guidance for each transfer of value from industry. For every transfer of value what restriction, please provide the following information: year of transfer, name of company, and restriction placed on funding. Answer #2: Although NORD has received funding from industry restricted for some of its programs i.e. Patient Assistance Programs (PAP), physician booklets and PNH meetings, the nature of restrictions are devoid of guidance from the companies and all attempts are made to maintain a safe and arms-length relationship for each of the programs with appropriate policy framework serving as a guide. NORD has been managing PAP programs since 1988 to assist uninsured or underinsured individuals in securing life-saving or life-sustaining medications. NORD got the impetus to administer PAP programs on an ongoing basis from its mention by name in the Settlement Agreement dated September 3, 1992, in the Clozapine Antitrust Litigation in which 23 states and the District of Columbia settled with Sandoz Pharmaceutical Corporation and Caremark Inc. The settlement provided that Sandoz, "through NORD," shall provide a rebate for patients taking Clozaril who are on Social Security Disability Income. This court-ordered program was one of the points of entry for NORD into providing patient assistance programs for uninsured (or underinsured) patients who are financially eligible. NORD administers two types of PAPs: (1) Medication Assistance Programs (MAP) that provide free drug product donated by drug and biotechnology manufacturers and (2) Premium Copayment Assistance Programs (Co-Pay Programs) designed to assist patients who have prescription drug insurance (e.g. Medicare Part B and D as well as commercially insured), but 2 cannot afford associated Medicare premiums and co-payments. NORD administers MAPs and disease specific Co-Pay Programs to cover qualifying Medicare beneficiaries who meet financial eligibility and disease specific criteria for various rare disorders without any influence or guidance from the sponsor. NORD's PAPs have set the standards for fairness, equity, confidentiality and unbiased eligibility, and have gained respect from the patient communities, pharmaceutical companies, healthcare professionals, government officials, and the public. There is no conflict of interest with respect to NORD's eligibility decisions. NORD's financial arrangement with the pharmaceutical industry with regard to PAPs follows the guidance provided by the Department of Health and Human Services' Office of Inspector General (OIG), which published a notice in the November 22, 2005 Federal Register (page 70623) providing a Special Advisory Bulletin on patient assistance programs under Medicare Part D. The principles set forth in the Bulletin reflect the policies and processes that NORD follows in implementing all of its PAPs. The Bulletin explains the safeguards needed for pharmaceutical companies to subsidize PAPs run by bona fide, independent charities (which NORD is) without running afoul of the fraud and abuse and anti-kickback statutes. "Simply put, the independent charity PAP must not function as a conduit for payments by the pharmaceutical manufacturer to patients and must not impermissibly influence beneficiaries' drug choices," the OIG document says. NORD assiduously follows the guidance provided in the Special Advisory Bulletin with regard to all Patient Assistance Programs that it runs. NORD receives funding from one of its Corporate Council members to conduct a series of educational meetings around the U.S. for patients with a rare disease, paroxysmal nocturnal hemoglobinuria (PNH), and their families. PNH patients have not had the benefit of a strong national patient advocacy group dedicated to serving their specific needs. The meetings provide these patients and their families with an opportunity to come together in small groups, and gain the encouragement, reinforcement and hope that comes from meeting face-to-face with other PNH patients. While this Corporate Council member makes the only drug to treat this disease, these meetings are non-promotional and strictly educational in nature and consistent with NORD's mission in providing a service to patients. Although there is a huge unmet need in the medical community for education on rare diseases, NORD is not a CME provider and only publishes a series of "Physician's Guide" booklets on 10 topics that are primarily funded by patient organizations. Only two booklets have been published with money from educational grants provided by companies, but in those cases, the companies have not been permitted to influence the content nor the distribution of the booklets. NORD is extremely careful to have the content reviewed by physician experts not affiliated with those companies and to clearly identify in the booklet the source of funding that made the publication possible. Question #3 Please explain what policies, if any that NORD plans to adopt to ensure transparency of funding in order to provide a greater public trust in the independence of your organization. Answer #3: While NORD has always embraced a culture of transparency and has worked hard to maintain the public's trust in its independence from industry, we are striving to make our policies in this area even stronger. NORD's financial report is posted on our website along with the list of industry donors. To further ensure transparency related to funding, we are incorporating additional new policies as follows: o o o Posting of funding details on NORD's website for corporate and foundation support that exceeds $5,000. annually Financial statement to have dedicated space to illustrate funding support from industry. Incorporating a guidance document for the role of Corporate Council members to further foster an "arms-length" policy between NORD and its Corporate Council members 3 o Reinforce our efforts to ensure that all NORD materials receiving any industry support identify the sources of support Question #4: Please explain your policies on disclosure of outside income by your top executives and board members. AND Question #5: Please provide the disclosures of outside income filed with your organizations by your top executives and board members. Answer #4 & 5: NORD does have policies in place in the by-laws for its board members and senior leaders to file conflict of interest disclosures to assure that any potential conflicts are disclosed and addressed if necessary. These by-laws undergo frequent revisions and updates in an on-going effort to maintain transparency and disclosure. All individuals being considered for appointment to the NORD board of directors must meet very stringent qualification criteria. There is a nominating committee within NORD's board of directors that is entrusted with the responsibility of ensuring that the nominated individuals fulfill criteria prior to being appointed to the board. No member of the board of directors receives any financial remuneration for participation on NORD's board. Since the board members are not dependent on NORD for any financial support, NORD does not require board members to provide specific details of outside income except to be in compliance with requirements that are set in the conflict of interest disclosures. NORD's efforts at maintaining a culture of transparency and avoiding conflict of interest are not limited only to the senior management and board members but also apply to all NORD staff members. In closing, for 2009, NORD's total revenues are expected to be approximately $14 million. Of this amount, $3.8 million represents unrestricted monies to fund NORD's operations (i.e., the operating budget). The remaining revenues ($10.2 million) represent funds restricted for NORD's PAPs and research funds. Although NORD accepts unrestricted donations from pharmaceutical and medical device companies, the vast majority of donations are modest amounts. NORD also receives donations from individuals, many in memory of lost loved ones. No donation to NORD influences the policy positions we take or the information we provide, all of which are based exclusively on the needs of the patients that we represent. As an example of the care we take in our fiscal policies, we have for the past several years charged $7.50 for each disease report purchased from our Rare Disease Database. While we regret having to charge patients for disease information, we decided we did not want to ask companies to assist us with the cost of maintaining the Database. We wanted to ensure that the medical information we provide is not only free of any industry influence, but free from any perception that a company was subsidizing the information. Names of patients or others who request disease information from NORD are never released, shared or sold to companies. NORD has always been scrupulous about keeping the medical information we provide completely independent of industry input. We never comment on, or recommend, specific products in our written materials, conversations or communications with patients. Further, in our Rare Disease Database, we are extremely careful to publish information only from neutral sources, such as the National Institutes of Health, the FDA, and academic papers in journals. We are also very careful to have our information reviewed by medical experts without industry ties for the topic they are reviewing. The physicians who write and/or review NORD reports are not paid. They donate their time without recompense because they understand that patients will benefit from their efforts 4 to expand awareness rare diseases. NORD does not ask researchers directly involved in studies of particular products to provide or review information for the standard therapies or investigational therapies sections of the reports. NORD holds the copyright for all reports in its Rare Disease Database. Many companies participate in NORD's Corporate Council, created more than a decade ago to provide opportunities for companies to work with NORD to advance medical research and product development. The membership dues go into our general fund and are used for general support for our programs and to support the Corporate Council meetings; the money is not earmarked for any specific program or usage. NORD views the Corporate Council members as an important constituency because, as discussed above, they are the companies that develop and provide the medical products that patients need. At the same time, they do not influence our policies. Corporate Council members are invited to attend two or three meetings a year sponsored by NORD and also receive email updates on our activities. A list of Corporate Council members and the dues they paid is in the attached Excel chart. The same Excel chart sets forth the annual amounts of industry funding of NORD. The chart includes the donations received from companies from 2006 through November 2009 for Patient Assistance Programs; a listing of the administrative fees to service the PAPs; a list of the Corporate Council members and the dues they paid; a listing of money received for the annual gala from companies that bought ads in the tribute journal or that purchased tables; and a listing of unrestricted donations from companies. Senator Grassley, I would be pleased to meet with you at your convenience to address any additional questions you have about NORD or the patient and research services we provide. To reiterate, NORD supports transparency and disclosure and we always seek to conduct our organization's activities with that principle in mind. Sincerely, PETER SALTONSTALL Peter L. Saltonstall President & CEO 5 Dale: July 2008 NORD. NA TlONAL ORGANIZA TION FOR RARE DISORDERS, INC" Page 8 taxpayers have paid fines for not signing up for health insurance. During the first year, some newly insured peop~ had trouble finding medical providers, and there were problems getting appointments because so many people came into the healthcare system at one time . Also, experts say there were not enough primary care doctors in the state. Under the new system. the very poor get free insurance, and the nearty poor get subsidized coverage. Employers get incentives to partiCipate, and there are penalties for those who don 't. No employer stopped providing health insurance to employees, which critics predicted would happen. The state budgeted $469 million for the first year but they had to add $153 million more. Those who chose not to partiCipate paid a total of $9.7 million in fines on their income tax. The size of the fines will grow in future years which may convince people it is cheaper to get the insurance than pay penalties. The state says there are two groups that tend to remain uninsured: Those who are not poor enough to qualify for a subsidy, but still feel they cannot afford to pay for it; and ~the younginvincibles? who think they are young and healthy enough not to need insurance. Nevertheless, when an uninsured person gets seriously hurt or becomes ill, all taxpayers often end up covering the cost. New York also sent a similar letter to Internet-based genetiC testing companies after receiving consumer complaints that the tests are not accurate and cost too much. A few weeks ago, 60 Minutes aired a segment on genetic tests marketed to African Americans showing that three companies came up with different answers on the genetics of a woman trying to investigate her heritage. CHUCK GRASSLEY: A SENATE HEALTH CRUSADER When Republicans held the majority of the Senate, Senator Cha~es Grassley (R-IA) was a committee chairman . This gave him sufficient staff and a budget to launch investigations that could lead to genuine reform . When the Democrats won the majority, he lost the committee chairmanship, but that has not stopped Senator Grassley from investigating health-related injustices and demanding reforms that should make the healthcare system more equitable to consumers : Transplant Investigation Senator Grassley is trying to find out why UCLA Medical Center did liver transplants on four Japanese gangsters. He says, at the very least, he expects that criminal figures should wait in line with everyone else who needs a transplant. Grassley also sent a letter to the FBI asking why, in 2001 , the agency helped a Japanese gangster get a visa to the USA for a liver transplant in exchange for information about Japanese gangsters In the USA He later donated $100,000 to the hospital. Senator Grassley says the transplants were performed between 2000 and 2004, during which time more than 100 patients died waiting for a liver transplant in the Los Angeles area. The hospital and the surgeon who did the transplants said they do not make moral judgments about patients, and all transplants are based on medical need. ACTION ON GENETIC TESTS In the absence of federal action, the state of California is cracking down on direct-to-consumer genetic tests. The state sent cease-and-desist letters to 13 Internet-based genetiC testing companies telling them they can no longer sell genetiC tests to California residents unless they can prove they are not breaking state laws. California law requires that a doctor must order the test , the lab that processes the test must be licensed by the state, and the test must be validated for clinical utility and accuracy. Most of the Intemetbased testing companies have a disclaimer that their tests are not medically useful, they represent preliminary findings , and they are merely for educational purposes. To KfHtP you up?to.dBttJ HaRD ON?LINE ~ 2008 Reprint permission II granted to NORD member agencies with appropriate credits given to NORD , Date: July 2008 NORD. NATIONAL ORGANIZA TION FOR RARE DISORDERS. IHCtI PageS o Conflicts-of-lnterest In Research Senator Grassley is investigating why world-famous Harvard psycl1iatrists Joseph Biederman and Timothy Wilens may have Violated federal and university conflict-of-interest rules by not reporting income from drug companies wh ile they also worked on federal research grants. According to the New York Times , Dr. Biederman is a world-renowned child psychiatrist whose research has -fueled an explosion in the use of antipsychotic medicines in children- (Researchers Fail to Reveal Full Drug Pay; New York Times, June 8, 2008: G . Harris and B. Carey) . He is cred~ed ~h a 40-fold increase in the number of children diagnosed with bipolar disorder in recent years. However, Dr. Biederman did not report $1 .6 million in consulting fees from drug companies between 2000 and 2007. Similarly, Dr. Wilens also earned $1 .6 million during that time period , and another Harvard colleague (Or. Thomas Spenser) earned $1 million in fees but also did not report it. The physicians must fill out a financial disclosure report for the university each year. If federal grantees do not provide honest answers, the NIH can place restrictions on grants to a hospital where the research is done. The grants to the Harvard researchers were paid to Massachusetts General Hospital. NIH requires grantees to report to universities the source of any income of $10,000 or more per year. Universities usually require that any possible conflict-of-interest money also be reported to research subjects. The NIH relies on universities to monitor these issues, but universities rarely do anything to verify the accuracy of financial disclosures. Senator Grassley found out about the drug company payments because he asked drug companies to give him a list of any consutting fees they have paid to doctors in recent years. At the time, he was investigating how much influence drug companies have over physicians. When he noticed the money given to the three researchers, he asked Harvard for their financial disclosure forms . It was obvious that the amounts did not match the figures submitted to him by drug manufacturers. Harvard's rules require that a researcher is not supposed to do research on a drug if he has been given more than $10,000 in consuHlng fees by the drug company that makes the drug. (See: Senate Inquiry on Research Conflicts Shins to Grantees; J. Kaiser, Science. Vol.320: June 27, 2008, pg 1708). Senator Grassley then sent a letter to NIH Director Elias Zerhouni asking why NIH had not taken oversight responsibility for extramural grantees, ensuring that universities are monitoring conflict-ofinterest by researchers. He also sent a letter to the American Psychiatric Association asking how much money the APA has received from drug companies. He questions whether the APA is truly independent in its viewpoints and actions when it relies on the drug industry for much of its operating costs. ELECTRONIC HEALTH RECORDS There continues to be a major push for electronic heaHh records (EHR) in Washinglon DC, despite a report from the Govemment Accountability Office (GAO) indicating that even ~ all health records were required to be electronic, it would not save any money. A study published in the June 2008 New England Journal o( Medicine (NEJM) indicates that even though doctors admit EHR would improve quality and timeliness of patient care, fewer than one in five of American doctors have computerized the medical records in their practice. The main issue is financial . Half of all medical care in the USA is provided in small medical offices run by one to three physicians. Only nine percent of these small practices have computerized their medical records because it would cost too much. Medicare is sponsoring a small project next year to test whether financial incentives would get doctors to move their patient records from paper to computer. CMS will spend $150 million on up to 1,200 small medical practiceS in 12 cities if they make the conversion to electronic records . Doctors will be offered up to $58,000 each. To keep you up-to-date NORD ON-LINE " 2008 Reprint pennlsslon Is granted to NORD member agencies with .pproprtale ctedits given to NORD 2006 CORPORATE COUNCIL Company Allergan Astra Zeneca Shire Alexion Medtronic Sanofi Aventis Actelion United Therapeutics Jazz Pharmaceuticals Celgene Ikaria Genzyme Johnson & Johnson Medicis BioMarin Purdue Pharma Rare Disease Therapeutics Vifor Pharma-Aspreva Duchesnay FFF Enterprises PTC Therapeutics Impax Labs Prestwick Neurochem Amicus Therapeutics NexGenix Pharmaceuticals Acorda Therapeutics Insmed Inc. Questcor NPS Pharmaceuticals Accredo Health Avigen Inc. Endo Pharmaceuticals ZioPharm Oncology Sub-total Pharmaceutical Cos. Mark Krueger & Associates Sub-total Other Entities Total Corporate Council Dues Amount $25,000 $25,000 $25,000 $10,000 $10,000 $10,000 $10,000 $10,000 $10,000 $10,000 $10,000 $10,000 $10,000 $10,000 $10,000 $5,000 $5,000 $5,000 $5,000 $5,000 $5,000 $5,000 $5,000 $5,000 $5,000 $5,000 $5,000 $5,000 $5,000 $5,000 $5,000 $5,000 $5,000 $1,000 $286,000 $2,500 $288,500 Reason 2006 Membership Dues 2006 Membership Dues 2006 Membership Dues 2006 Membership Dues 2006 Membership Dues 2006 Membership Dues 2006 Membership Dues 2006 Membership Dues 2006 Membership Dues 2006 Membership Dues 2006 Membership Dues 2006 Membership Dues 2006 Membership Dues 2006 Membership Dues 2006 Membership Dues 2006 Membership Dues 2006 Membership Dues 2006 Membership Dues 2006 Membership Dues 2006 Membership Dues 2006 Membership Dues 2006 Membership Dues 2006 Membership Dues 2006 Membership Dues 2006 Membership Dues 2006 Membership Dues 2006 Membership Dues 2006 Membership Dues 2006 Membership Dues 2006 Membership Dues 2006 Membership Dues 2006 Membership Dues 2006 Membership Dues 2006 Membership Dues 2006 Membership Dues 2006 ANNUAL TRIBUTE BANQUET Company Journal Ads Allergan Genzyme Astra Zeneca Sigma Tau Novartis BioMarin EMD Serono Lab Celgene Alexion Medtronic Barr Labs Ligand Ikaria Cephalon Questcor OrphanEurope/Recordet Actelion Swedish Orphan Schering-Plough Bausch & Lomb CSL Behring Nova Factor Teva Neuroscience Teva North America Amicus therapeutics Acorda therapeutics Ucycld Pharma GlaxoSmithKline Centocor Insmed Rare Disease Therapeutics Johnson & Johnson Tercica Mission Pharmacal PTC Therapeutics Pfizer Duchesnay Prestwick Talecris Connetics Genta Lundbeck ZioPharma Sub-total Pharmaceutical Cos. Engelberg Foundation Mark Krueger & Associates GPHA Ropes & Gray NACDS The Redstone Group Hyman, Phelps, McNamara Canreg Inc. HPS Medco Health Jennings Policy Engage Health SturgeWeber Sub-total Other Entities Total Journal Ads $25,000 $15,000 $15,000 $15,000 $15,000 $15,000 $15,000 $10,000 $10,000 $10,000 $10,000 $10,000 $10,000 $10,000 $7,500 $7,500 $7,500 $7,500 $7,500 $7,500 $7,500 $7,500 $7,500 $7,500 $7,500 $7,500 $5,500 $5,000 $5,000 $5,000 $5,000 $5,000 $5,000 $5,000 $3,000 $2,500 $2,500 $2,500 $2,500 $2,500 $2,500 $2,000 $1,000 $336,500 $7,500 $7,500 $7,500 $5,000 $2,500 $2,500 $2,500 $1,000 $1,000 $750 $750 $500 $250 $39,250 $375,750 Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Amount Reason Banquet Tables/Seats Genzyme Shire Human Genetics Alexion Bristol Myers Squibb Knopp Neuro Sub-total Pharmaceutical Cos. Waggener, Edstrom Worldwide Individuals/Member Organizations Sub-total Other Entities Total Banquet Tables/Seats TOTAL TRIBUTE BANQUET REVENUES $5,000 $5,000 $1,000 $1,000 $500 $12,500 $1,000 $4,975 $5,975 $18,475 $394,225 Tables/Seats Tables/Seats Tables/Seats Tables/Seats Tables/Seats Tables/Seats Tables/Seats 2006 Patient Services Programs (Free Drug & Co-pay) Company (Co-pay Fund Grants) BiogenIdec Bayer Genzyme Astellas Teva Neuroscience Elan EMD Serono Berlex Bausch & Lomb Shire BioMarin Administrative Fees Deducted from Grant Proceeds Net Co-pay Grants Administrative Fees for Co-pay Programs Invoiced Separately Free Drug Administrative Fees Acorda Therapeutics Actelion Allergan Sanofi Aventis Sanofi Pasteur Bayer Cephalon GlaxoSmithKline Medicis Medtronic Jazz Pharmaceuticals QOL Medical Questcor Rare Disease Therapeutics Sigma-Tau Teva Neuroscience Administrative Fees Re-cap: Co-pay Programs Free Drug Programs Fees Invoiced Separately $73,176 $28,760 $48,900 $98,536 $9,528 $88,395 $261,400 $74,692 $5,000 $34,652 $60,240 $2,500 $21,552 $7,652 $40,095 $201,500 $1,056,578 $1,237,232 $1,056,578 $154,993 $2,448,803 Fees to NORD for Servicing Programs Fees to NORD for Servicing Programs Fees to NORD for Servicing Programs Fees to NORD for Servicing Programs Fees to NORD for Servicing Programs Fees to NORD for Servicing Programs Fees to NORD for Servicing Programs Fees to NORD for Servicing Programs Fees to NORD for Servicing Programs Fees to NORD for Servicing Programs Fees to NORD for Servicing Programs Fees to NORD for Servicing Programs Fees to NORD for Servicing Programs Fees to NORD for Servicing Programs Fees to NORD for Servicing Programs Fees to NORD for Servicing Programs Amount $2,500,105 $1,500,000 $352,648 $200,000 $5,478,300 $100,000 $650,000 $250,000 $141,300 $50,000 $50,000 $11,272,353 $1,237,232 $10,035,121 $154,993 Fees to NORD for Servicing Programs Reason Grant Donation to Support Co-pay Fund Grant Donation to Support Co-pay Fund Grant Donation to Support Co-pay Fund Grant Donation to Support Co-pay Fund Grant Donation to Support Co-pay Fund Grant Donation to Support Co-pay Fund Grant Donation to Support Co-pay Fund Grant Donation to Support Co-pay Fund Grant Donation to Support Co-pay Fund Grant Donation to Support Co-pay Fund Grant Donation to Support Co-pay Fund Fees to NORD for Servicing Programs 2006 UNRESTRICTED GENERAL CONTRIBUTIONS Company Allergan Actelion Allergan GTC Biotherapeutics Amgen Political Action Committee Johnson & johnson Sub-total Pharmaceutical Cos. Individual - Stock Donation Beatrice Snyder Foundation Nvidia Corporation Schwab Charitable Fund Vanguard Charitable Endowment New South Investment Group Individuals As A Group (183) Sub-total Individuals/Other Entities TOTAL GENERAL CONTRIBUTIONS Amount $10,268 $10,000 $5,000 $2,500 $459 $200 $28,427 $20,655 $5,000 $2,041 $1,000 $1,000 $900 $31,602 $62,198 $90,625 RESTRICTED FELLOWSHIP & OTHER GRANTS FELLOWSHIP Genzyme OTHER GRANTS Medtronic Foundation Medtronic Foundation Medtronic Foundation Medtronic Foundation Catt Family Foundation Ligand Allergan Baxter AstraZeneca AstraZeneca Actelion Amyloidosis Support Group Amicus Therapeutics TOTAL OTHER GRANTS $75,000 Research Grant for Lysosomal Storage Diseases Grant for Awareness, Advocacy Urban Scholarship Grant Patient Support Group Mentoring "Patient Link" Advocacy Grant Grant for General Operating Exp Medicare Task Force Medicare Task Force Medicare Task Force Educational Grant to update 10 Rare Disease Reports Grant to produce/print booklet on Medullary Thyroid Cancer Educational Grant to update 12 Rare Disease Reports Educational Grant to publish Amyloidosis Booklet Clinical Broadcast Reason Contribution Contribution Contribution Contribution Contribution Matched Employee Gift Contribution Contribution Contribution Contribution Contribution Contribution Contributions $100,000 $50,000 $45,000 $25,000 $30,000 $15,000 $10,000 $10,000 $10,000 $39,100 $20,000 $20,000 $3,000 $377,100 2006 RECONCILIATION TO AUDITED FINANCIAL STATEMENTS Contributions & Bequests: Unrestricted General Contributions Bequests Corporate Council Dues Direct Mail Federated Workplace Campaigns Patient Family Conference IMO/IHO Unrestricted Miscellaneous Income Amount Per Audited Financial Statements Special Events Revenue: Annual Tribute Banquet Amount Per Audited Financial Statements Restricted Contributions & Grants: Travel & Co-pay Grants Fellowship Grant Research Grants IMO/IHO Other Grants Amount Per Audited Financial Statements $90,625 See worksheet for details $3,914 $288,500 See worksheet for details $36,410 $40,897 $24,920 $39,730 $944 $525,940 $525,940 $394,225 See worksheet for details $394,225 $10,035,122 See worksheet for details $75,000 See worksheet for details $110,491 $58,997 $377,100 See worksheet for details $10,656,710 $10,656,610 2007 CORPORATE COUNCIL Company Allergan Shire Bausch & Lomb Sigma Tau Astra Zeneca Jazz Pharmaceuticals Medtronic United Therapeutics Altus Pharmaceuticals Celgene Ikaria Genzyme Johnson & Johnson Aton Pharma Medicis Alexion BioMarin Purdue Pharma Questcor Avigen IDIS Pharmaceuticals Fold Rx Pharmaceuticals Impax Labs Rare Disease Therapeutics Vifor Pharma - Aspreva Duchesnay FFF Enterprises PTC Therapeutics Millenium Neurochem Amicus Therapeutics NexGenix Pharmaceuticals Acorda Therapeutics Endo Pharmaceuticals Insmed Inc. Accredo Health ExSAR Corporation ZioPharm Oncology Sub-total Pharmaceutical Cos. Mark Krueger & Associates Sub-total Other Entities Total Corporate Council Dues Amount $25,000 $25,000 $25,000 $25,000 $25,000 $10,000 $10,000 $10,000 $10,000 $10,000 $10,000 $10,000 $10,000 $10,000 $10,000 $10,000 $10,000 $5,000 $5,000 $5,000 $5,000 $5,000 $5,000 $5,000 $5,000 $5,000 $5,000 $5,000 $5,000 $5,000 $5,000 $5,000 $5,000 $5,000 $5,000 $5,000 $2,500 $1,000 $343,500 $2,500 $2,500 $346,000 Reason 2007 Membership Dues 2007 Membership Dues 2007 Membership Dues 2007 Membership Dues 2007 Membership Dues 2007 Membership Dues 2007 Membership Dues 2007 Membership Dues 2007 Membership Dues 2007 Membership Dues 2007 Membership Dues 2007 Membership Dues 2007 Membership Dues 2007 Membership Dues 2007 Membership Dues 2007 Membership Dues 2007 Membership Dues 2007 Membership Dues 2007 Membership Dues 2007 Membership Dues 2007 Membership Dues 2007 Membership Dues 2007 Membership Dues 2007 Membership Dues 2007 Membership Dues 2007 Membership Dues 2007 Membership Dues 2007 Membership Dues 2007 Membership Dues 2007 Membership Dues 2007 Membership Dues 2007 Membership Dues 2007 Membership Dues 2007 Membership Dues 2007 Membership Dues 2007 Membership Dues 2007 Membership Dues 2007 Membership Dues 2007 Membership Dues 2007 ANNUAL TRIBUTE BANQUET Company Journal Ads Accredo Health Inc. Acorda Therapeutics Actelion Pharmaceuticals U.S. Alexion Pharmaceuticals Allergan Inc. Amicus Therapeutics AstraZeneca Barr Pharmaceuticals Inc. Bausch & Lomb Bayer Corporation BioMarin Pharmaceutical Inc. Celgene Corporation Cephalon Inc. CSL Behring Duchesnay, Inc. Genzyme Corp. INO Therapeutics Insmed Inc. Inspire Pharmaceuticals Introgen Pharmaceuticals Inc. Johnson & Johnson Karl Storz Endoscopy-America LEV Pharmaceuticals Medtronic, Inc. Millenium Pharmaceutical Mylan Laboratories Neurochem Novartis Pharmaceuticals Orphan Europe Ovation Pharmaceuticals PTC Therapeutics Questcor Pharmaceuticals, Inc. Rare Disease Therapeutics Inc. Serono Inc. Shire Human Genetics Therapies Sigma-Tau Pharmaceuticals Inc. Swedish Orphan International Tercica Inc. Teva N.A./Teva USA Ucyclyd Pharma, Inc. ZioPharm Sub-total Pharmaceutical Cos. Agvar Chemicals Bell Pottinger, USA Engage Health, Inc. GPhA Hyman, Phelps & McNamara Jennings Policy Strategies Mark Krueger & Associates NACDS Sturge Weber Foundation The Engelberg Foundation The Redstone Group Sub-total Other Entities Total Journal Ads $7,500 $7,500 $10,000 $10,000 $25,000 $7,500 $25,000 $10,000 $5,000 $3,000 $15,000 $10,000 $10,000 $7,500 $3,000 $20,000 $15,000 $5,000 $1,500 $1,500 $5,000 $1,000 $2,500 $10,000 $3,000 $5,000 $5,000 $15,000 $7,500 $3,000 $4,000 $7,500 $3,000 $5,000 $15,000 $15,000 $14,973 $7,500 $15,000 $5,000 $1,000 $348,973 $25,000 $1,500 $1,000 $7,500 $3,000 $500 $7,500 $3,000 $450 $7,500 $3,000 $59,950 $408,923 Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Amount Reason Banquet Tables/Seats Shire FoldRx IDIS LEV Pharmaceutical Knopp Neurosci Asklepion Pharma PHARMA Purdue Pharma Vifor Pharma Argent Development Group Altus Sub-total Pharmaceutical Cos. Makovsky & Co. Medtronic Foundation Campaign for Tobacco Free Kids Jennings Policy Strategies Fleishman & Hilliard Zuckerman Spaeder Seed One Ventures Research America HPS Group Individuals Sub-total Other Entiites TOTAL TRIBUTE BANQUET REVENUES $5,000 $1,500 $1,000 $1,000 $1,000 $500 $5,000 $500 $500 $500 $500 $17,000 $2,000 $1,250 $500 $500 $500 $500 $500 $500 $250 $8,300 $14,800 $440,723 Tables/Seats Tables/Seats Tables/Seats Tables/Seats Tables/Seats Tables/Seats Tables/Seats Tables/Seats Tables/Seats Tables/Seats Tables/Seats Tables/Seats Tables/Seats Tables/Seats Tables/Seats Tables/Seats Tables/Seats Tables/Seats Tables/Seats Tables/Seats Tables/Seats 2007 Patient Services Programs (Free Drug & Co-pay) Company (Co-pay Fund Grants) BiogenIdec Bayer Genzyme Astellas Teva Neuroscience Sigma Tau Shire BioMarin Allergan Alexion Teva USA Questcor Administrative Fees Deducted from Grant Proceeds Net Co-pay Grants Administrative Fees for Co-pay Programs Invoiced Separately Free Drug Administrative Fees Acorda Therapeutics Alexion AstraZeneca Aton Pharma Cephalon GlaxoSmithKline Jazz Pharmaceuticals Medicis Medtronic Questcor Rare Disease Therapeutics Sanofi Aventis Sanofi Pasteur Sigma Tau Teva Neuroscience Teva USA Administrative Fees Recap: Co-pay Programs Free Drug Programs Fees Invoiced Separately $41,660 $28,925 $21,250 $28,400 $276,900 $59,607 $56,000 $5,000 $38,116 $24,052 $10,652 $87,700 $17,220 $44,084 $202,752 $3,106 $945,424 $724,810 $945,424 $121,905 $1,792,139 Fees to NORD for Servicing Programs Fees to NORD for Servicing Programs Fees to NORD for Servicing Programs Fees to NORD for Servicing Programs Fees to NORD for Servicing Programs Fees to NORD for Servicing Programs Fees to NORD for Servicing Programs Fees to NORD for Servicing Programs Fees to NORD for Servicing Programs Fees to NORD for Servicing Programs Fees to NORD for Servicing Programs Fees to NORD for Servicing Programs Fees to NORD for Servicing Programs Fees to NORD for Servicing Programs Fees to NORD for Servicing Programs Fees to NORD for Servicing Programs Amount $2,500,000 $4,391,000 $208,088 $350,000 $632,850 $25,000 $300,000 $450,000 $100,000 $1,550,000 $26,020 $85,000 $10,617,958 $724,810 $9,893,148 $121,905 Fees to NORD for Servicing Programs Reason Grant donation to Support Co-pay Fund Grant donation to Support Co-pay Fund Grant donation to Support Co-pay Fund Grant donation to Support Co-pay Fund Grant donation to Support Co-pay Fund Grant donation to Support Co-pay Fund Grant donation to Support Co-pay Fund Grant donation to Support Co-pay Fund Grant donation to Support Co-pay Fund Grant donation to Support Co-pay Fund Grant donation to Support Co-pay Fund Grant donation to Support Co-pay Fund Fees to NORD for Servicing Programs 2007 UNRESTRICTED GENERAL CONTRIBUTIONS Company Celgene Genzyme Barr Pharmaceuticals Sanofi Aventis Allergan BioMarin Shire Enobia GTC Biotherapeutics Sanofi Pasteur Johnson & Johnson Merck Sub-total Pharmaceutical Cos. Skowronski Family Foundation Charles P. Ferro foundation Edoward Foundation Beatrice Snyder Foundation Adolph & Ruth Schumacher Fdtn Schecter Foundation Fidelity Charitable Foundation Littell Consulting Group ACS Commercial Solutions Cargill Inc. Jerome & Grace Murray Fdtn Agatha Costa Charitable Fdtn Director Foundation Schwab Charitable Foundation Enterprise Rent A Car Vanguard Pajwell Foundation Tech Law Group JP Morgan Chase Foundation Strategy X Trustmark Foundation Lincoln Financial Group Schlesinger Medical Quad Graphics Avon Products Aetna Giving Campaign Ameriprise Financial Employee Fund of Boeing Individuals as a Group (247) Sub-total Other Entities/Individuals TOTAL GENERAL CONTRIBUTIONS Amount $25,000 $25,000 $25,000 $20,000 $14,000 $5,000 $2,500 $1,000 $300 $250 $200 $120 $118,370 $10,000 $7,500 $5,000 $5,000 $2,500 $2,000 $2,650 $1,500 $1,460 $1,350 $1,200 $1,000 $1,000 $1,000 $1,000 $2,000 $1,000 $1,000 $300 $425 $216 $200 $200 $200 $380 $125 $109 $102 $24,156 $74,573 $192,943 ABBEY MEYERS RETIREMENT GALA Amount Reason $5,000 Payment for Table/Seats $5,000 Payment for Table/Seats $5,000 Payment for Table/Seats $5,000 Payment for Table/Seats Reason Contribution Contribution Contribution Contribution Contribution Contribution Contribution Contribution Contribution Contribution Matched Employee Gift Matched Employee Gift Contribution Contribution Contribution Contribution Contribution Contribution Contribution Contribution Contribution Contribution Contribution Contribution Contribution Contribution Contribution Contribution Contribution Contribution Contribution Contribution Contribution Matched Employee Gift Matched Employee Gift Matched Employee Gift Matched Employee Gift Matched Employee Gift Matched Employee Gift Matched Employee Gift Contributions Company Allergan Shire Sanofi Aventis Alexion BioMarin Sigma Tau Talecris Biotherapeutics Baxter CSL Behring Amicus Therapeutics Genzyme PTC Therapeutics Hyperion Therapeutics Questcor Duchesnay Inc. EMD Serono Inc. Acorda therapeutics AstraZeneca Ikaria Medicis/Ucycld Pharmaceutical Aton Pharma ZioPharm Oncology Inc. Asklepion Pharma Lev Pharma Argent Development Group Accredo/Medco Swedish Biomimetrics AB Biomedical Frontiers Inc Sub-total Pharmaceutical Cos. Leonard McDowell, LLC Technology Law Group HPS Group LLC Weinberg Group Wilmeer, Cutler, Pickering, Hale & Dorr, LLP Fleisham-Hillard, Inc. The Redstone Group Mark Krueger & Associates Ropes & Gray Jennings Policy Strategies Critical Path Institute Children's Hospital of Philadelphia U.S. Dept of HHS Americans for Medical Progress Zuckerman Spaeder LLP Individuals/Member Organizations Sub-total Other Entities/Individuals TOTAL ASM RETIREMENT GALA Company Genzyme Medtronic Foundation PTC Therapeutics Barr Pharmaceuticals Amicus Therapeutics Rare Disease Therapeutics Sub-total Pharmaceutical Cos. Mark Krueger & Associates Engelberg Foundation HPS Group $5,000 $5,000 $5,000 $3,000 $3,000 $3,000 $2,000 $2,000 $2,000 $1,000 $1,000 $1,000 $1,000 $1,000 $500 $500 $500 $400 $400 $350 $400 $200 $200 $200 $58,650 $1,200 $1,000 $500 $500 $1,200 $300 $300 $400 $200 $200 $200 $200 $400 $200 $200 $19,500 $26,500 $85,150 Payment for Table/Seats Payment for Table/Seats Payment for Table/Seats Payment for Table/Seats Payment for Table/Seats Payment for Table/Seats Payment for Table/Seats Payment for Table/Seats Payment for Table/Seats Payment for Table/Seats Payment for Table/Seats Payment for Table/Seats Payment for Table/Seats Payment for Table/Seats Payment for Table/Seats Payment for Table/Seats Payment for Table/Seats Payment for Table/Seats Payment for Table/Seats Payment for Table/Seats Payment for Table/Seats Payment for Table/Seats Payment for Table/Seats Payment for Table/Seats Payment for Table/Seats Payment for Table/Seats Payment for Table/Seats Payment for Table/Seats Payment for Table/Seats Payment for Table/Seats Payment for Table/Seats Payment for Table/Seats Payment for Table/Seats Payment for Table/Seats Payment for Table/Seats Payment for Table/Seats Payment for Table/Seats Payment for Table/Seats Payment for Table/Seats Payment for Table/Seats ABBEY MEYERS EDUCATION FUND Amount Reason $25,000 Donation to Fund $25,000 Donation to Fund $2,000 Donation to Fund $2,000 Donation to Fund $2,000 Donation to Fund $1,000 Donation to Fund $57,000 $2,500 $2,000 $500 Donation to Fund Donation to Fund Donation to Fund Individuals/Member Organizations Sub-total Other Entities/Individuals TOTAL ASM EDUCATION FUND $12,200 $17,200 $74,200 Donation to Fund 2007 RESTRICTED FELLOWSHIP & OTHER GRANTS Company Genzyme Total Fellowship Medtronic Foundation Medtronic Foundation Catt Family foundation Baxter Lev Pharmaceuticals Total Other Grants Amount $100,000 $100,000 $100,000 $63,000 $15,000 $10,000 $55,712 $243,712 Awareness & Advocacy Grant Grant for National Urban Fellow Scholarship Grant for Operating Expenses Medicare Task Force Educational Grant to Produce Booklet on Angioedema Reason Fellowship Research Grant for Lysosomal Storage Diseases 2007 RECONCILIATION TO AUDITED FINANCIAL STATEMENTS Contributions & Bequests: Unrestricted General Contributions Bequests Corporate Council Dues Direct Mail Federated Workplace Campaigns Patient Family Conference IMO/IHO Unrestricted Abbey Meyers Retirement Event Abbey Meyers Education Fund Miscellaneous Income Amount Per Audited Financial Statements Special Events Revenue: Annual Tribute Banquet Amount Per Audited Financial Statements Restricted Contributions & Grants: Travel & Co-pay Grants Fellowship Grant Research Grants IMO/IHO Amount Per Audited Financial Statements Other Grants: (grouped with "Indigent Care Drug and Co-pay Assistance Programs on 2007 Audited Financial Statements) $192,943 $0 $346,000 $42,390 $52,583 $25,215 $25,693 $85,150 $74,200 $225 $844,399 $844,399 $440,723 See Worksheet for Details $440,723 $9,893,148 See Worksheet for Details $100,000 See Worksheet for Details $44,749 $115,605 $10,153,502 $10,153,502 $243,712 See Worksheet for Details See Worksheet for Details See Worksheet for Details See Worksheet for Details See Worksheet for Details 2008 CORPORATE COUNCIL Company Shire Sigma Tau Questcor Jazz Pharmaceuticals Genzyme Medtronic United Therapeutic Celgene Ikaria Johnson & Johnson Medicis Alexion BioMarin Baxter Hyperion Aton Pharmaceutical Purdue Pharma Rare Disease Therapeutics Duchesnay FFF Enterprises PTC Therapeutics FoldRx IDIS Prestwick Vifor Pharma Aspreva Neurochem Amicus Therapeutics Acorda therapeutics Insmed Inc. Accredo Health Zio Pharma Sub-total Pharmaceutical Cos. Mark Krueger Associates Sub-total Other Entities Total Corporate Council Dues Amount $25,000 $25,000 $25,000 $10,000 $10,000 $10,000 $10,000 $10,000 $10,000 $10,000 $10,000 $10,000 $10,000 $10,000 $10,000 $10,000 $5,000 $5,000 $5,000 $5,000 $5,000 $5,000 $5,000 $5,000 $5,000 $5,000 $5,000 $5,000 $5,000 $5,000 $2,000 $277,000 $2,500 $2,500 $279,500 Reason 2008 Membership Dues 2008 Membership Dues 2008 Membership Dues 2008 Membership Dues 2008 Membership Dues 2008 Membership Dues 2008 Membership Dues 2008 Membership Dues 2008 Membership Dues 2008 Membership Dues 2008 Membership Dues 2008 Membership Dues 2008 Membership Dues 2008 Membership Dues 2008 Membership Dues 2008 Membership Dues 2008 Membership Dues 2008 Membership Dues 2008 Membership Dues 2008 Membership Dues 2008 Membership Dues 2008 Membership Dues 2008 Membership Dues 2008 Membership Dues 2008 Membership Dues 2008 Membership Dues 2008 Membership Dues 2008 Membership Dues 2008 Membership Dues 2008 Membership Dues 2008 Membership Dues 2008 Membership Dues 2008 ANNUAL TRIBUTE BANQUET Company Journal Ads Accredo Health Acordia Thwerapeutics Actelion Pharmaceuticals Alexion Pharmaceutical Allergan Inc. Amicus Therapeutics Argent Development Group Aton Pharma Barr Laboratories Inc. Baxter Healthcare BioMarin Pharmaceuticals Biotechnology Industry Organization Celgene Corporation Cephalon Inc. CSL Behring DOR Biopharma EMD Serono Genzyme Corporation IDM Pharma Ikaria Insmed Inc. Lev Pharmaceuticals Medtronic Millenium Pharmaceuticals Mission Pharmacal Novartis Oncology Ovation Pharmaceuticals Pfizer PHARMA Prestwick Pharmaceuticals Questcor Pharmaceuticals PTC Therapeutics Rare Disease Therapeutics Recordati (Orphan Europe) Sanofi Aventis US Sanofi Pasteur Shire Human Genetics Sigma Tau Pharmaceuticals Swedish Orphan International Teva Neuroscience Teva USA Sub-total Pharmaceutical Cos. Association of American Medical Colleges Agvar Chemicals Inc. Generic Pharmaceutical Association Hyman Phelps & McNamara Mark Krueger & Associates NACDS Foundation Sub-total Other Entities Total Journal Ads Amount $15,000 $30,000 $15,000 $15,000 $50,000 $7,500 $1,500 $2,500 $10,000 $5,000 $30,000 $20,000 $50,000 $10,000 $15,000 $1,500 $2,500 $20,000 $1,500 $10,000 $2,500 $15,000 $10,000 $1,500 $2,500 $15,000 $2,500 $5,000 $50,000 $2,500 $10,000 $6,500 $25,000 $25,000 $25,000 $7,000 $20,000 $50,000 $9,973 $7,500 $7,500 $611,973 $1,500 $30,000 $7,500 $2,500 $15,000 $2,500 $59,000 $670,973 Reason Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Journal Ad - Amount Based on Ad Size Banquet Table/Seats Mondo Biotech Zio Pharm Jerini Hyperion Therapeutics K M Pharma FoldRx Pharma Chelsea Therapeutics Purdue Pharma Momenta Pharma Motif Biosci Sub-total Pharmaceutical Cos. Individuals GBS/CIDP Foundation ICORD HPS Group Sub-total Individuals/Other Entities Total Banquet Seats Program Line Listing Solakian, Caiafa LLC (Accounting Firm) TOTAL TRIBUTE BANQUET REVENUES $150 $705,223 Program Listing $1,000 $1,000 $500 $1,500 $1,000 $1,000 $500 $500 $500 $75 $7,575 $17,175 $5,000 $2,850 $1,500 $26,525 $34,100 Tables/Seats Tables/Seats Tables/Seats Tables/Seats Tables/Seats Tables/Seats Tables/Seats Tables/Seats Tables/Seats Tables/Seats Tables/Seats Tables/Seats Tables/Seats Tables/Seats 2008 Patient Services Programs ( Free Drug & Co-pay) Company (Co-pay Fund Grants) Alexion Bayer BiogenIdec BioMarin Genzyme PTC Therapeutics Questcor Regeneron Shire Sigma Tau Teva USA Administrative Fees Deducted from Grant Proceeds Deferred Grant Revenue to 2009 Net Co-pay Grants Administrative Fees Invoiced Separately Free Drug Administrative Fees Acorda Therapeutics Aton Pharma Cephalon Jazz Pharmaceutical Medicis Medtronic Questcor Rare Disease Therapeutics Sanofi Aventis Sanofi Pasteur Sigma Tau Teva Neurosciences Administrative Fees Re-cap: Co-pay Programs Free Drug Programs Fees Invoiced Separately Amount $200,000 $6,000,000 $3,900,000 $500,000 $89,592 $130,000 $760,000 $500,000 $100,000 $25,000 $20,500 $12,225,092 $934,645 $2,900,000 $8,390,447 $56,525 $27,100 $26,900 $213,700 $55,000 $5,000 $32,000 $30,000 $11,652 $97,700 $7,140 $44,311 $202,750 $753,253 $934,645 $753,253 $56,525 $1,744,423 Fees to NORD for Servicing Programs Fees to NORD for Servicing Programs Fees to NORD for Servicing Programs Fees to NORD for Servicing Programs Fees to NORD for Servicing Programs Fees to NORD for Servicing Programs Fees to NORD for Servicing Programs Fees to NORD for Servicing Programs Fees to NORD for Servicing Programs Fees to NORD for Servicing Programs Fees to NORD for Servicing Programs Fees to NORD for Servicing Programs Fees to NORD for Servicing Programs Reason Grant Donation to support Co-pay Fund Grant Donation to support Co-pay Fund Grant Donation to support Co-pay Fund Grant Donation to support Co-pay Fund Grant Donation to support Co-pay Fund Grant Donation to support Co-pay Fund Grant Donation to support Co-pay Fund Grant Donation to support Co-pay Fund Grant Donation to support Co-pay Fund Grant Donation to support Co-pay Fund Grant Donation to support Co-pay Fund Fees to NORD for Servicing Programs 2008 UNRESTRICTED GENERAL CONTRIBUTIONS Company Allergan Genzyme Dyax Corporation BioMarin GTC Biotherapeutics Merck Partnership for Giving Sub-total Pharmaceutical Cos. Individual Stock Donations (2) Skowronski Family Foundation Edoward Foundation Schecter Foundation Adolph & Ruth Schnumacher Foundation U.S. Pharmacopia New South Investment Group Vanguard Charitable Trust Fidelity Charitable Trust Maxell Corp. Hitachi Foundation Agatha M. Costa Charitable Foundation California Comm. Foundation General Reinsurance Grace Rapinchek Charitable Foundation Master Tax LLC Amgen Altred & Harriet Feinman Foundation Pajwell Foundation Aetna Giving Campaign Goldstein Foundation Defined Health JP Morgan Chase Quad Graphics Giving Express R & E Demple Family Foundation Barroway, Topaz, Kessler LLP Johnson & Johnson Aetna Giving Campaign Cargill AVON Products Merck Partnership for Giving Individuals As A Group (383) Sub-total Other Entities/Individuals TOTAL UNRESTRICTED CONTRIBUTIONS Amount $25,000 $17,793 $2,500 $2,236 $1,500 $180 $49,209 $12,797 $10,000 $10,000 $6,000 $5,500 $5,000 $2,750 $2,000 $2,500 $2,355 $2,355 $1,000 $1,000 $1,000 $1,000 $1,000 $930 $500 $500 $500 $500 $500 $500 $300 $293 $250 $200 $200 $200 $195 $140 $120 $45,543 $117,628 $166,837 Reason Contribution NORD named as recipient of Boston Marathon proceeds Contribution Contribution Clinical Broadcast Matched Employee Gift Contribution Contribution Contribution Contribution Contribution Contribution Contribution Contribution Contribution Contribution Contribution Contribution Contribution Contribution Contribution Contribution Matched Employee Gift Contribution Contribution Contribution Contribution Contribution Matched Employee Gift Matched Employee Gift Matched Employee Gift Contribution Contribution Matched Employee Gift Matched Employee Gift Matched Employee Gift Matched Employee Gift Matched Employee Gift 2008 OTHER GRANTS Company Alexion Pharmaceuticals Medtronic Foundation Medtronic Foundation Catt Family Foundation Medtronic Foundation TOTAL OTHER GRANTS Amount $150,000 $125,000 $60,000 $15,000 $125,000 $475,000 Reason 6 Patient Support Meetings for PNH Medical meetings, Infrastructure Upgrade and General Operating Expenses (2008) Patient Organization Mentoring Grant for Operating Expenses Medical meetings, Infrastructure Upgrade and General Operating Expenses (2009) Funds received in Dec'2008. 2008 RECONCILIATION TO AUDITED FINANCIAL STATEMENTS Contributions & Bequests: Unrestricted General Contributions Bequests Corporate Council Dues Abbey Meyers Education Fund Federated Workplace Campaigns IMO/IHO Unrestricted Amount Per Audited Financial Statements Special Events Revenue: Annual Tribute Banquet Amount Per Audited Financial Statements Restricted Contributions & Grants: Travel & Co-pay Grants Research Grants IMO/IHO Other Grants Amount Per Audited Financial Statements $166,837 See Worksheet for Details $27,387 $279,500 See Worksheet for Details $4,850 $66,769 $35,790 $581,133 $581,133 $705,223 See Worksheet for Details $705,223 $8,390,447 See Worksheet for Details $105,891 $138,899 $475,000 See Worksheet for Details $9,110,237 $9,110,237 2009 CORPORATE COUNCIL Company Accredo Health Actelion Pharmaceuticals, U.S. Alexion Pharmaceuticals Allergan Inc. Amicus Therapeutics Inc. Aspreva/Vifor AstraZeneca Aton Pharma Inc. Baxter BioScience BioMarin Pharmaceuticals Celgene Corporation CSL Behring Duchesnay Inc. Enobia Pharma FoldRx Pharmaceuticals Inc. Genzyme Corporation Hyperion Therapeutics Ikaria Lundbeck Inc. Medicis Global Services Corp. Medtronic Novartis Pharmaceuticals NPS Pharmaceuticals Pfizer Purdue Pharma Questcor Pharmaceuticals Raptor Pharmaceuticals Corp. Rare Disease Therapeutics Shire Human Genetics Soligenix, Inc. Synageva BioPharma United Therapeutics Corp. ZioPharm Inc. Sub-total Pharmaceutical Cos. Mark Kruger & Associates Total Corporate Council Dues Amount $5,000 $10,000 $10,000 $25,000 $5,000 $5,000 $5,000 $10,000 $10,000 $10,000 $10,000 $10,000 $5,000 $10,000 $5,000 $10,000 $5,000 $10,000 $25,000 $10,000 $10,000 $10,000 $10,000 $25,000 $5,000 $25,000 $5,000 $5,000 $25,000 $3,000 $5,000 $10,000 $1,000 $334,000 $2,500 $336,500 Reason 2009 Membership Dues 2009 Membership Dues 2009 Membership Dues 2009 Membership Dues 2009 Membership Dues 2009 Membership Dues 2009 Membership Dues 2009 Membership Dues 2009 Membership Dues 2009 Membership Dues 2009 Membership Dues 2009 Membership Dues 2009 Membership Dues 2009 Membership Dues 2009 Membership Dues 2009 Membership Dues 2009 Membership Dues 2009 Membership Dues 2009 Membership Dues 2009 Membership Dues 2009 Membership Dues 2009 Membership Dues 2009 Membership Dues 2009 Membership Dues 2009 Membership Dues 2009 Membership Dues 2009 Membership Dues 2009 Membership Dues 2009 Membership Dues 2009 Membership Dues 2009 Membership Dues 2009 Membership Dues 2009 Membership Dues 2009 ANNUAL TRIBUTE BANQUET Company Journal Ads Accredo Health Acorda Therapeutics Actelion Pharmaceuticals, U.S. Alexion Pharmaceuticals Allergan Inc. Amicus Therapeutics Inc. AstraZeneca Aton Pharma Inc. Baxter BioScience BioMarin Pharmaceuticals Biotechnology Industry Org (BIO) Biovail Celgene Corporation Centric Health Resources Cephalon CSL Behring Duchesnay Inc. Eisai, Inc. Endo Pharma Engage Health Enobia Pharma Genzyme Corporation GPnA GTC Biotherapeutics Hyman Phelps McNamara Kakkis EveryLife Foundation Keck Graduate Institiute Lundbeck Inc. Mark Kruger & Associates Medtronic Millennium mondoBIOTECH Novartis Oncology Optherion Orphan Europe Pfizer PhRMA PTC Therapeutics Questcor Pharmaceuticals Rare Disease Therapeutics Regeneron Sanofi Aventis Shire Human Genetics Sigma Tau Strategic Diagnostics Swedish Orphan International Talecris Teva Neuroscience ViroPharma Inc. ZioPharm Inc. Sub-total Journal Ads Program Listing Amount $15,000 $30,000 $10,000 $15,000 $30,000 $7,500 $10,000 $3,000 $15,000 $20,000 $5,000 $3,000 $50,000 $3,000 $15,000 $50,000 $3,000 $3,000 $10,000 $1,000 $3,000 $18,000 $7,500 $2,000 $3,000 $10,000 $1,150 $3,000 $15,000 $10,000 $10,000 $7,500 $15,000 $3,000 $15,000 $15,000 $10,000 $3,000 $7,500 $7,500 $3,000 $25,000 $20,000 $10,000 $3,000 $7,480 $10,000 $15,000 $5,000 $1,000 $564,130 Reason Journal Ad - Amount Based on Size of Ad Journal Ad - Amount Based on Size of Ad Journal Ad - Amount Based on Size of Ad Journal Ad - Amount Based on Size of Ad Journal Ad - Amount Based on Size of Ad Journal Ad - Amount Based on Size of Ad Journal Ad - Amount Based on Size of Ad Journal Ad - Amount Based on Size of Ad Journal Ad - Amount Based on Size of Ad Journal Ad - Amount Based on Size of Ad Journal Ad - Amount Based on Size of Ad Journal Ad - Amount Based on Size of Ad Journal Ad - Amount Based on Size of Ad Journal Ad - Amount Based on Size of Ad Journal Ad - Amount Based on Size of Ad Journal Ad - Amount Based on Size of Ad Journal Ad - Amount Based on Size of Ad Journal Ad - Amount Based on Size of Ad Journal Ad - Amount Based on Size of Ad Journal Ad - Amount Based on Size of Ad Journal Ad - Amount Based on Size of Ad Journal Ad - Amount Based on Size of Ad Journal Ad - Amount Based on Size of Ad Journal Ad - Amount Based on Size of Ad Journal Ad - Amount Based on Size of Ad Journal Ad - Amount Based on Size of Ad Journal Ad - Amount Based on Size of Ad Journal Ad - Amount Based on Size of Ad Journal Ad - Amount Based on Size of Ad Journal Ad - Amount Based on Size of Ad Journal Ad - Amount Based on Size of Ad Journal Ad - Amount Based on Size of Ad Journal Ad - Amount Based on Size of Ad Journal Ad - Amount Based on Size of Ad Journal Ad - Amount Based on Size of Ad Journal Ad - Amount Based on Size of Ad Journal Ad - Amount Based on Size of Ad Journal Ad - Amount Based on Size of Ad Journal Ad - Amount Based on Size of Ad Journal Ad - Amount Based on Size of Ad Journal Ad - Amount Based on Size of Ad Journal Ad - Amount Based on Size of Ad Journal Ad - Amount Based on Size of Ad Journal Ad - Amount Based on Size of Ad Journal Ad - Amount Based on Size of Ad Journal Ad - Amount Based on Size of Ad Journal Ad - Amount Based on Size of Ad Journal Ad - Amount Based on Size of Ad Journal Ad - Amount Based on Size of Ad Journal Ad - Amount Based on Size of Ad ApoPharma Inc. Argent Development Group Copilevitz & Canter Simon-Kucher Pharma Siren Interactive The IDEA League Sub-total Program Listing Banquet Seats America's Health Insurance Plans American Porphyria Foundation Amyloidosis Support Group ApoPharma Argent Development Group Benign Essential Blepharospasm Bennu Phar/Raptor Conquer Pediatric Chiari Cystinosis Research Network DesignWrite Diplomat Specialty Pharmacy DEBRA DystoniaMedResearch FoldRx Pharma HPS Group IDEA League IDIS Ltd Lapidus Data Mercy Medical Airlift MME, LLC Neurofibromatosis NORD Board Members Palo Alto Investors Parry Romberg Connection Purdue Pharma Russell-Silver Syndrome Santhera Pharm Simon-Kucher Partners Siren Interactive Ucyclyd/Medicis United Leukodystrophy Foundation Voisin Consulting Individuals Sub-total Banquet Seats TOTAL TRIBUTE REVENUES $150 $150 $150 $150 $150 $50 $800 Line Listing in Program Line Listing in Program Line Listing in Program Line Listing in Program Line Listing in Program Line Listing in Program $1,000 $200 $800 $500 $1,000 $100 $1,000 $1,000 $300 $500 $500 $100 $100 $500 $500 $200 $1,520 $500 $100 $500 $100 $1,250 $500 $100 $500 $200 $500 $1,000 $200 $500 $400 $500 $1,000 $17,670 $582,600 Tables/Seats Tables/Seats Tables/Seats Tables/Seats Tables/Seats Tables/Seats Tables/Seats Tables/Seats Tables/Seats Tables/Seats Tables/Seats Tables/Seats Tables/Seats Tables/Seats Tables/Seats Tables/Seats Tables/Seats Tables/Seats Tables/Seats Tables/Seats Tables/Seats Tables/Seats Tables/Seats Tables/Seats Tables/Seats Tables/Seats Tables/Seats Tables/Seats Tables/Seats Tables/Seats Tables/Seats Tables/Seats NOTE: For the 11 months ended November 30, 2009 Company (Co-pay Fund Grants) Alexion Allergan Allos Therapeutics Bayer BiogenIdec BioMarin Elan Enobia Pharma Marathon Medtronic Novartis PTC Therapeutics Questcor Shire Sigma Tau Teva USA BiogenIdec 2008 Grant Deferred Administrative Fees Deducted from Grant Proceeds Net Co-pay Grants Administrative Fees Invoiced Separately Free Drug Administrative Fees Acorda Pharmaceuticals Aton Pharmaceuticals Cephalon Jazz Pharmaceuticals Medicis/Ucyclyd Medtronic Questcor Rare Disease Therapeutics Sanofi-Aventis Sigma Tau Pharmaceuticals Teva Neoroscience Administrative Fees Re-cap: Co-pay Programs Free Drug Programs Fees Invoiced Separately 2009 Patient Services Programs (Free Drug & Co-pay) Amount $500,000 $393,500 $400,000 $1,300,000 $1,000,000 $1,242,000 $100,000 $81,467 $59,526 $50,000 $500,000 $665,000 $950,000 $135,000 $75,000 $75,000 $7,526,493 $2,900,000 $923,863 $9,502,630 $65,710 Fees to NORD for Servicing Programs Reason Grant donation to Support Co-pay Fund Grant donation to Support Co-pay Fund Grant donation to Support Co-pay Fund Grant donation to Support Co-pay Fund Grant donation to Support Co-pay Fund Grant donation to Support Co-pay Fund Grant donation to Support Co-pay Fund Grant donation to Support Co-pay Fund Grant donation to Support Co-pay Fund Grant donation to Support Co-pay Fund Grant donation to Support Co-pay Fund Grant donation to Support Co-pay Fund Grant donation to Support Co-pay Fund Grant donation to Support Co-pay Fund Grant donation to Support Co-pay Fund Grant donation to Support Co-pay Fund Grant donation to Support Co-pay Fund Fees to NORD for Servicing Programs $27,100 $32,900 $123,940 $13,750 $5,000 $32,000 $30,000 $11,652 $67,500 $43,909 $217,900 $605,651 $923,863 $605,651 $65,710 $1,595,224 Fees to NORD for Servicing Programs Fees to NORD for Servicing Programs Fees to NORD for Servicing Programs Fees to NORD for Servicing Programs Fees to NORD for Servicing Programs Fees to NORD for Servicing Programs Fees to NORD for Servicing Programs Fees to NORD for Servicing Programs Fees to NORD for Servicing Programs Fees to NORD for Servicing Programs Fees to NORD for Servicing Programs 2009 UNRESTRICTED GENERAL CONTRIBUTIONS Company Sigma Tau Genzyme Shire Amgen Johnson & Johnson Sub-total Pharmaceutical Cos. Catt Family Foundation Fidelity Charitable Trust Adolph & Ruth Schnumacher Fdtn Pioneer Credit Recovery Gilbane Building Co. Vanguard Charitable Trust Horace Greeley High School Nokia Aetna Giving Campaign Simpson JP Morgan APCO Worldwide Trustmark Foundation IBM Individuals As A Group (286) Sub-total Other Entities/Individuals Total General Contributions Amount $50,000 $27,942 $5,000 $1,560 $200 $84,702 $10,000 $6,000 $2,500 $2,474 $2,000 $2,000 $1,790 $500 $500 $500 $300 $250 $232 $120 $26,611 $55,777 $140,479 OTHER GRANTS Company Medtronic Foundation Alexion Pharmaceuticals Amount $63,000 $270,000 Reason Urban Fellow Scholarship 11 Patient Support Meetings for PNH Reason Discretionary Gift NORD named as recipient of Boston Marathon donations. NORD named as recipient of Shire hosted event Matched Employee Gift Matched Employee Gift Contribution Contribution Contribution Contribution Contribution Contribution Contribution Contribution Contribution Contribution Matched Employee Gift Contribution Contribution Anonymous Employee Donation Contributions