Speeches & Testimony

Testimony Submitted Regarding FY 2002 Funding for the Orphan Product Research Grants Program
April 2001

Mr. Chairman and members of the Senate Appropriations Subcommittee on Agriculture, Rural Development and Related Agencies, the National Organization for Rare Disorders (NORD), wishes to express its views regarding appropriations for the Orphan Products Research Grant Program administered by the Office of Orphan Product Development (OOPD) at the Food and Drug Administration (FDA).

NORD is a federation of approximately 140 voluntary health organizations and over 70,000 individual patients, healthcare providers and clinical researchers dedicated to helping the 25 million people in the United States suffering with rare "orphan" diseases. An orphan disease is defined by statute as any disease or condition impacting less than 200,000 Americans. It makes no difference whether you are male or female, rich or poor, young or old, white, African-American, Latino, Asian or American Indian. These diseases affect everyone.

On behalf of the rare disease community, we are respectfully requesting that just one dollar for each and every person suffering with a rare disease be appropriated by this Subcommittee for the FDA¹S Orphan Products Research Grant Program. Twenty-five million dollars would represent a minimal investment by the federal government in the development of lifesaving treatments that the private sector is not interested in. But the return on investment could be phenomenal if only a few new orphan drugs or devices are developed to reduce the burden of disease and death for thousands of patients with rare disorders.

As you can imagine, appropriating just one dollar for each rare disease patient in America, rather than the current funding level of a mere fifty cents per patient, is a win-win situation. Patients win when their symptoms are alleviated or cured. Families win when their loved ones no longer suffer. Society, as a whole, wins when patients are able to return to school or work to become productive tax-paying citizens. Pharmaceutical and biotechnology companies win when they are able to market new therapeutic products when part of the development costs are subsidized. The scientific community wins when the knowledge they gain can be applied to more prevalent diseases. And, finally, the government wins when the drain on healthcare dollars is minimized.


FDA Orphan Products Research Grants Program

Congress created the research grants program in FY 1983 to provide funding for pivotal clinical trials on new orphan drugs, medical devices, and medical foods for rare diseases. The funds have been made available to academic scientists and small companies. By definition, "orphan products" are treatments for rare conditions that have small potential markets and thus are not attractive to the commercial sector. Such treatments were not being developed for "orphan" diseases by the private sector until the Orphan Drug Act was enacted in 1983.

Since then, the FDA has approved over 218 orphan drugs for marketing, and more than 800 additional drugs are in the research pipeline. Of those products approved for marketing, 27 (23 drugs and 4 medical devices) were developed with funding from the orphan product grants. These 27 treatments would not be on the American market today saving the lives of thousands of Americans, enabling them to return to school or work, if Congress had not created this small but critically important pool of research funds.

Most of FDA¹s Orphan Products Research Grants support small clinical trials at academic institutions throughout the nation to develop the preliminary evidence that is necessary to attract commercial sponsors. It is the quintessential model for a successful government/industry partnership. There is no more appropriate program deserving of federal support because it fills a major gap between academic research and the private sector, and it creates lifesaving products that are needed throughout the world.

For example, children with Severe Combined Immune Deficiency (the "Bubble Boy Disease") no longer have to live in a plastic bubble because now their immune systems can fight off germs, thanks to an orphan drug developed with these grant funds. Children with urea cycle disorders no longer slip into a coma and die because an orphan drug enables their bodies to eliminate toxic levels of ammonia. Babies born without ribs no longer suffocate in infancy because an artificial rib (an orphan medical device) is being developed now with funds from the Orphan Products Research Grants Program that allows the children¹s lungs to expand and breathe. Cystic fibrosis, Crohn¹s disease, and multiple sclerosis drugs are on the market today only because these grants supported some of their research.

Unfortunately, there are many diseases and conditions that are too rare to attract private investment because the commercial sector is simply not interested in developing treatments for small markets. The investment necessary for research and development of new drugs and devices is too large in comparison to the size of the potential market for a rare disease. Case in point, there are only about 125 patients in the United States suffering with an orphan disease called fibrodysplasia ossificans progressiva (FOP), Only 15,000 with Huntington¹s disease or Duchenne Muscular Dystrophy, and only 30,000 with cystic fibrosis. Many of the genetic diseases each impact no more than 40,000 Americans. Whereas, drugs for cancer, arthritis or hypertension, for example, each affect many millions of Americans, representing several billion dollars in potential sales each year.

Given the fact that the Orphan Products Research Grant Program is attracting greater attention, more researchers are eager to participate each year. Therefore, it is very unfortunate that the annual appropriation for this program cannot begin to cover all of the meritorious grant requests for promising research projects. Today, about 100 grant applications are received annually, but many scientifically important applications are never funded simply because the appropriation is too small to meet the needs of the program. In fact, the appropriation now is less than it was in FY 1995, and has remained between $10 to $12 million for many years.

Mr. Chairman, if the government does not fund this research, who will? The private sector is simply not interested in rare diseases. If this Subcommittee does not meet the need of this unique sector of scientific research, people with rare diseases will be further victimized by the injustice of the supply and demand marketplace. For these diseases, no company wants to supply a treatment when the market demand is small.


Conclusion

In 1989 the HHS National Commission on Orphan Diseases estimated that only 30 percent of the 25 million patients suffering with rare diseases receive a diagnosis in three to five years after the onset of symptoms. That works out to about 7.5 million patients who are shuffled from specialist to specialist, year after year. Fifteen percent, or 3.7 million people, wait seven years or more. And even after diagnosis, they can only hope that someone, somewhere, will conduct research to develop a treatment for their disease.

And so, on behalf of those medically disenfranchised Americans and their families, we respectfully request that the members of this Subcommittee appropriate no less than $25 million dollars to the FDA Orphan Products Research Grant Program for FY 2002. We are relying on the members of this Subcommittee to fill the void between government and the private sector, and propel these treatments forward from academic laboratories to our local pharmacies. Ultimately, your compassion and insight will put new orphan drugs and devices into the waiting hands of critically ill patients. If you don¹t provide adequate resources for the Orphan Products Research Grants Program, unfortunately no one else will.

Thank you.

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