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. wDa :/ 60/6 /2 D00 00 urAngSCy :/ 60/6 /2AwDaMC MNnwdlmCesyb CTtaph Mh: /001 EoC hUocREMNC00 u rG UEOChUM Tis book was made possible through an unrestricted grant from Medunik Canada whose mission is to improve the health and quality of life of Canadians living with rare diseases. ISBN 978-0-692-572177 Printed and bound in Canada. None of the contents may be reproduced or transmitted in any way or form or by any means, without prior written consent of the publisher, Abbey S. Meyers. © 2016 Abbey S. Meyers. All rights reserved. www.abbeysmeyers.com I have written this book based on my memories and I have made every efort to ensure that the information in this book is accurate. - Abbey S. Meyers Orphan Drugs: A Global Crusade | 5 TABLE OF CONTENTS Forward 9 Chapter 1 Many Questions, Few Answers 15 Chapter 2 Quest for Treatment 33 Chapter 3 The Road to Passage 57 Chapter 4 NORD 95 Chapter 5 Baby Steps 117 Chapter 6 Amendments & Consequences 137 Chapter 7 Stemming the Tide 157 Chapter 8 Questionable Intentions 181 Chapter 9 A Constant Struggle 203 Chapter 10 International Impact 217 Chapter 11 Entrepreneurs 233 Chapter 12 Working with the Government 247 Chapter 13 The Rise, Fall and Rebirth of Gene Therapy 269 Chapter 14 Refections 291 Orphan Drugs: A Global Crusade | 7 Forward I am no historian or politician, nevertheless, the story of the Orphan Drug Act begs to be told. I am simply an observer of extraordinary events that unfurled before my eyes, like a fower in bloom. The story involves people with serious health problems who were treated as if their lives were disposable, like a paper plate that is relegated to a garbage bag after the picnic is completed. My memory of the events in this book is largely accurate, but the sequence or timing of the events may be faulty. This Memoir is simply my efort to document what happened, when and why. The most important factor is nothing ever occurred because of one person’s eforts. All advancements occurred because many people put their personal interests aside and worked together with others to reach common goals. That coalition, which became the National Organization for Rare Disorders (NORD), represented unprecedented political strength that could not be ignored. In 1983, starting with myself, other volunteers and a part time secretary, we built NORD as a national refuge for people with rare disorders and their families. NORD’s programs were built Orphan Drugs: A Global Crusade | 9 not upon things that we thought might help them, but on the services and programs that patients, families and health care workers told us they needed. Since my retirement, I have experienced health problems that make traveling very difcult so I decided it was time to write this book. I no longer have input into NORD’s programs and activities. I do not know if the programs we put in place still exist today. I can only say that I am proud of the non-proft corporation that I left to my successor, and I am astounded at the growth and efectiveness of the orphan disease movement around the world today. This is a story about the needs of millions of people with rare diseases who were consistently ignored simply because there were not enough of them with each diagnosis. In other words they would not be proftable enough in comparison to treatments aimed at high blood pressure, headaches, arthritis or indigestion, etc. Because corporations did not deem rare diseases to be proftable enough, the unavailability of treatments (nicknamed “orphan drugs” in the medical literature) represented a gaping hole in the health care system. The “Free Market” mindset of business establishments measure potential success according to the rules of “supply and demand”; because rare diseases afect small potential markets, pharmaceutical manufacturers concluded that the “demand” side of the equation was too small to address the “supply”. Nonetheless, there are over 7,000 rare diseases afecting 25 to 30 million Americans and millions more throughout the world! Should they be left to sufer and die without hope simply because they represent minorities that are too small to make businesses rich? The ethical questions became as important as the fnancial issues. This is the story of how by working together, ordinary people were able to plug an enormous hole in the health care system when the 10 | Orphan Drugs: A Global Crusade “free market” did not address their needs. To accomplish this they needed to attract to their cause other patients, physicians, politicians, business leaders, government employees and millions of others throughout the world. They had to learn how the government functions, who the policy makers are, how to infuence them, and how to lure disinterested people to their cause. The passage of federal legislation, the Orphan Drug Act of 1983, was only the frst step. This event jump started decades of extraordinary scientifc advancements and development of miraculous new treatments for diseases once deemed to be hopeless. But it also kicked of a period of unimaginable price infation that has put the cost of some orphan drugs beyond the reach of many. There are many people to thank for their encouragement, their lending of knowledge and talents that went into this book. I started writing it a few years after I retired, and I never stopped tinkering with paragraphs and sentences. First and foremost I have to thank my family for standing by me during all the years that I missed their school plays, music concerts and football games (to name a few). My husband Jerry, and children David, Adam and Laura, got used to my mind being elsewhere even when we sat together at the dinner table or played a game of monopoly on a snowy day. My children are adults with their own families now, and I thank God every day that they are healthy, and they have health insurance which, thanks to ObamaCare, cannot be taken away from them. I also want to thank my good friends and colleagues who worked with me at NORD for many years: Jean Campbell and Maria Hardin, who were Vice Presidents at NORD, and to this day can still remember the names of people and their contact info long after my Rolodex became passé. Stephanie Putkowski was a nurse who worked tirelessly at NORD administering our research program and talking to distressed patients who needed comfort Orphan Drugs: A Global Crusade | 11 and hope. And I remain grateful to my former secretary, Audrey Ashley, who for decades saved me from embarrassing exposure as the world’s worse speller. Rob Tomaino deserves my greatest thanks for editing this book and rearranging the presentation of content. Rob is a medical writer who consulted for NORD on various projects, especially the “Rare Disease Database”. Without Rob’s editing I would have given up on this book long ago. Rob, Steph and several other of NORD’s staf worked under Mary Dunkle, NORD’s Vice President of Information Services, who translated complicated medical terms into understandable language so people could fnd out about the disease they or their loved one had, what its causes could be, possible treatments, and if investigational new treatments were being tested how they could fnd out about clinical trials. NORD’s “Rare Disease Database” can be accessed on NORD’s website (www.rarediseases.org) I join with Jean, Maria, Steph and Rob for lunch every few weeks so we can catch up on the things each of us is doing. They represent much of the success NORD had because I always knew they would make the right management decisions. They still remain involved with rare diseases even though they don’t work for NORD anymore. Eric Gervais and his wife, Carole Boyer, have been key to my completing this book and putting it on the Internet so people can read it. They will never know how much their encouragement has meant. Eric is the CEO of Médunik Canada, an orphan drug company that makes American and European orphan drugs available to Canadian patients. Canada has not yet enacted orphan drug legislation despite years of pressure from rare disease support groups in Canada. The parent company of Médunik is Duchesnay, which manufactures Diclegis, a drug to treat nausea during pregnancy. After you read in this book about Eric Gervais and his herculean efort to get Diclegis on the American market, you will understand his great 12 | Orphan Drugs: A Global Crusade accomplishment for another group of ignored and abandoned patients, pregnant women. Most pharmaceutical companies don’t want to test their drugs on women, no less pregnant women, because their perceived risk of liability is too high. Doris Zallen, Ph.D., also deserves my thanks for helping me in the early days of writing this book. Doris is a Professor at Virginia Tech and she has written several excellent books about genetic testing and bioethical issues. We served together on government advisory committees that were involved with gene therapy protocols. I always felt that judging the science of each research project was equally important to the bioethical issues that were inherent in each experiment, but too often the scientists didn’t feel the same way. Doris and I stood together on many of these issues. There are numerous others who deserve my thanks for helping me to bring this book to completion. Ultimately, anytime I was at wits end, I thought of the staf people who I worked with me at NORD, and was reminded of their devotion to NORD’s mission.

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