Speeches & Testimony

FDA TESTIMONY FABRY'S DISEASE

Abbey S. Meyers
President
National Organization for Rare Disorders (NORD)
55 Kenosia Avenue
Danbury, CT 06813


January 13 & 14, 2003

I am Abbey Meyers, President of the National Organization for Rare Disorders (NORD). NORD is the non-profit voluntary health agency that is the chief consumer advocate for the Orphan Drug Act of 1983. We continue to monitor implementation of the law, promote increased research on rare disorders, and to improve patients' access to orphan drugs.

For the sake of transparency, NORD has received donations from both Genzyme and Transkaryotic Therapies (TKT). In particular both companies have generously contributed to our research grant program for fellowships on lysosomal storage diseases. It is our hope that these "Roscoe Brady Fellowships" will attract young physician-scientists to this field, ultimately leading to new treatments for more than 40 lysosomal diseases.

Today and tomorrow, this advisory committee will review applications for two new orphan drugs that are enzyme replacement therapies for Fabry's disease. I'm here to explain the implication of the Orphan Drug Act on your discussion.

Fabry's is one of 42 known lysosomal storage diseases that, until recently, have been untreatable and deadly. Only a few hundred people in the United States have Fabry's disease, so it is beyond doubt an "orphan disease" that is in desperate need of therapy.

So why are two biotechnology companies racing to obtain marketing approval for such a very rare disease? First, the basic research breakthroughs on lysosomal storage diseases have mostly derived from the NIH, particularly from the laboratory of Dr. Roscoe Brady at the National Institute of Neurological Disorders and Stroke (NINDS). As a result, the understanding of the basic biochemical defect in Fabry's disease has been in the public domain, and two companies that have focused their product lines on orphan diseases decided to develop the same enzyme replacement therapy at the same time. Each company has invested millions of dollars in its race to marketing approval. But, because of the Orphan Drug Act, only one company can win this race, and it is a dead heat. Such contests are very unusual in orphan drug development.

The need for, and the success of, the Orphan Drug Act is obvious. Most companies have no interest in developing a drug for a very limited population because the potential market, and ultimately the potential profit, is so small. The pharmaceutical and biotechnology industry has made it very clear that the chief incentive of the Act is seven years of marketing exclusivity - that is, an absence of competition for seven years. In fact, most orphan drugs don't even have competitors after their exclusivity expires because other companies are simply not interested in such small markets. In the unique case of Fabry's disease, however, two companies began their development efforts at the same time, and they are racing each other to approval because it is a "winner-take-all" contest. One company will reach the market, but the other must wait seven years before it will be allowed on the market.

There are only three ways that more than one company can obtain FDA marketing approval for the same orphan drug, for the same indication, within the seven-year period of exclusivity: First if both companies voluntarily agree to share exclusivity, and secondly, if the follow-on manufacturer can prove that its drug is "different" from the first drug by proving it is "clinically superior" to the first drug, and thus a "major contribution to patient care." This is what FDA's regulations require. Thirdly, after marketing approval, if the first company cannot maintain an adequate supply of an orphan drug, another company may be allowed on the market.

I have personally asked both companies (Genzyme and TKT) to voluntarily agree to share exclusivity for the Fabry's enzyme because that would undoubtedly be the best remedy for the Fabry's disease patient community. However, they have not agreed to do so. Therefore, this problem is in the hands of this advisory committee today and tomorrow. But let me say definitively, it would be unacceptable for you to recommend that neither treatment should be approved because patients with Fabry's disease will not accept that. Patients demand that you recommend approval of at least one of these products because if you don't, it will be a death sentence for them.

We realize that you need the Wisdom of Solomon to determine which of these two products should reach the market, and you will indeed be cutting the baby in half. If you simply say both are fine and both should be approved, the law dictates that if one gets marketing approval even five minutes before the other, the second product will not reach the market for seven years. On the other hand, you could recommend approval of one and then determine that the other enzyme is "clinically superior". The fact is both products are on the European market, and Fabry's patients are benefiting from both of them. But FDA is challenged to protect the integrity of the Orphan Drug Act because without it we will never get treatments for other rare disorders.

The only unacceptable outcome of this advisory committee meeting is if you decide that neither drug should be approved. You will hear from patients today and tomorrow who would have been dead if they had no access to Fabrazyme or Replagal. We don't know which product is better than the other, but we do know that both products are on the market in the European Union, and patients are alive because they have access to them. The European Union's Orphan Medicinal Products Regulation was enacted in 2000, and these were among the first orphan drugs approved by the EMEA at the same exact time, on the same day.

To recommend approval of both of these orphan drugs, you would have to determine that they are "different" from each other, based on the FDA regulations that define "same" and "different" orphan drugs. The fate of people with Fabry's disease is in your hands. We wish you the best of luck in your deliberations.