Speeches & Testimony

UNIAMO MEETING

Abbey S. Meyers
President
National Organization for Rare Disorders (NORD)

Venice, Italy
September 1, 2002

Thank you for inviting me to speak to you today in this historical and very beautiful city. I come from the United States where rare disease patient organizations united together more than two decades ago to insure that new treatments would be developed for diseases no matter how rare they were. Drug companies felt that such treatments had little commercial value because they would not be profitable enough. When the Orphan Drug Act became law in 1983, the National Organization for Rare Disorders (NORD) became a charitable corporation composed of voluntary health agencies and individuals dedicated to the identification, treatment and cure of rare diseases through programs of education, research, advocacy, and services to patients and families.

In Europe our sister agency is EURORDIS, the European Organization for Rare Disorders, which just celebrated its 5th anniversary. EURORDIS has encouraged formation of consumer organizations in each European nation, and among them is UNIAMO, which is the Italian federation for rare disorders. Today, there are similar national federations in many countries of Europe, all working together to raise awareness about rare disorders, and to encourage progress in research, and access to treatments.

I salute you all for working together and ignoring your differences. You must continue to focus on the most important factors that we all have in common:

A. In all countries throughout the world, rare diseases go undiagnosed or misdiagnosed for a long time, primarily because physicians are not familiar with each ailment.

B. Education of the public and medical professionals is needed if we are to reduce the time lapse between onset of a rare disorder and proper diagnosis.

C. There is little research because the commercial sector is generally not interested, and academic scientists fear that it may be too hard to build a career in diseases that get very little funding.

D. Health care corporations must be enticed into developing diagnostic tests and treatments, and they must train their personnel so they can help families with rare disorders.

E. Wherever I go, senior scientists tell me they’re worried that there aren’t enough young researchers interested in building their careers in rare diseases. We have to do something to attract young scientists to this field.

F. Most importantly, there are no international agreements about controversial scientific and ethical issues such as gene patenting, stem cells, biotechnology, cloning, etc. Research that cannot be done in one country will certainly be done in another, and unethical research will certainly be pursued if we do not develop a global ethical pact that all nations agree to.

How can we solve these problems? Patient organizations raise money to fund research so they can find a cure for their disease. But there will be no cures unless we solve these problems and pave a road between the laboratory bench to the bedsides of patients. This dream brings to mind an ancient Chinese adage about “hope”. It says, “Hope is like a road in the country. There never was a road, but when many people walk together a road comes into existence”.

Our rare disease community is universal. Individually, each disease is rare, but together, we are a prevalent health problem. Individually, we are weak, but together we are politically powerful. Together we can pave a road that will carry the innovative ideas of rare disease scientists to the pharmaceutical and medical device companies that know how to develop them, and finally to the local pharmacies in our communities that make treatments available to patients who need them.

NORD in the United States, UNIAMO in Italy, and EURORDIS in Europe, are the United Nations for rare diseases. Here, you must forget the differences between individual diseases, and think about all of us together. If the road is not smooth, if there are barriers because some patients in some countries have no access to a treatment, then we must work together to remove these barriers.

A. We must reduce the time lapse between onset of a disease and proper diagnosis. Delays in diagnosis cause harm no matter which disease you have, no matter which country you live in.

B. We must increase research. Research breakthroughs come from an accumulation of knowledge over many decades. An important discovery in one disease will absolutely help other related diseases. But increasing research is not enough if scientists refuse to share their knowledge. It is our duty to promote collaboration and cooperation between researchers.

C. The European Union’s Orphan Drug Regulation is only the first step. It will help major discoveries to be translated into treatments, but treatments will not reach local pharmacies unless the pharmaceutical industry develops them and makes them commercially available.


1. Patient organizations still have to raise money to fund research grants on individual diseases, because if you don’t understand the basic cause of a disease, you cannot halt or delay its insidious progress.

2. And we still have to lobby our governments to spend more money on research because no matter how much consumer groups spend, it is miniscule compared to what a government can spend.

3. We have to encourage patients to participate in research, but we also have to improve human research protections.

4. We still have to encourage companies to focus on our diseases because if we don’t they will continue to focus on the most prevalent and profitable conditions.

5. And, we still have to convince scientists to build their careers in our rare diseases. But scientists need the security of knowing that funding will remain available in the future, and that assurance is our responsibility.


D. We still have to educate the public and medical professionals so they know what these diseases are. The public believes that rare disorders don’t matter because they’re confident that they won’t get one. But no one is immune from an orphan disease, and we have to remind them.

E. We have to tell the pharmaceutical industry that they ought to develop orphan drugs because it is the right thing to do, and because the laws we worked so hard to enact will ensure that they won’t lose money, and in fact, they deserve to earn a reasonable profit. But we must also draw an ethical line beyond which companies will not tread. They must understand that patient organizations represent the best interests of patients, and drug companies represent the best interests of stockholders. Sometimes these two realms will disagree, and sometimes they will work together cooperatively toward the same goal.

F. We have to work with national governments to ensure that they will pay for orphan drugs, and not make patients deteriorate while they wait for bureaucrats to make life-saving decisions. The price that patients have to pay while governments negotiate with manufacturers is too high.

G. Patient organizations must fund research fellowships, and encourage governments to fund them too, so that young scientists will see a future in rare disease research. The aging generation of senior scientists must train the younger ones so they will benefit from the lifetimes of work that the retiring scientists have created.

None of us can solve these problems alone. We must work together through national and international federations. We are all committed to the same goals – the alleviation of pain, avoidance of disability, and most importantly, saving the lives of people with rare disorders.

You have the best wishes of your American cousins at NORD, for your continuing efforts in Italy. We know you will succeed.