Fw: To All Volunteers in Wisconsin and Mississippi

[Guest guest]

URGENT CONGRESSIONAL ALERT

Dear Volunteer in Public Policy:

Senator J. Durbin (D-IL) needs your help. He wants to include an additional $2 million in the Food and Drug Administration appropriations bill for the Orphan Drug Research Grant Program, but he can’t do it without your support!

Funding for the Orphan Drug Research Grant Program has remained static for many years and additional funds are urgently needed to encourage academic scientists and small companies to conduct clinical trials at research institutions throughout the nation to develop the preliminary evidence necessary to attract commercial sponsors. For small unprofitable pharmaceutical companies that cannot afford to pay all the costs of clinical research, these grants underwrite some of their research expenses.

I urge you to call Senator Herb Kohl (D-WI), Chairman of the Senate Appropriations Subcommittee on Agriculture, Rural Development, and Related Agencies and Senator Thad Cochran (R-MS), Ranking Member of the same Subcommittee today. Tell them why it is so important that they support efforts to increase the funding for the Orphan Drug Research Grant Program by $2 million. Let them know why this is so important to you, your family and the entire rare disease community.

Below are some talking points you may find helpful, as well as the local phone numbers of your Senators. If you need additional guidance please call me or e-mail me at dorman@....

Senator Herb Kohl State Offices

Appleton

Eau Claire

La Crosse

Madison

Milwaukee

Senator Thad Cochran (R-MS) State Offices

Oxford

FUNDING FOR FDA ORPHAN RESEARCH GRANTS PROGRAM INADEQUATE

FY 2002

Office of Orphan Product Development

Food and Drug Administration (FDA)

What is the Orphan Product Research Grant Program?

Because there are many diseases and conditions too rare to attract private investment by large pharmaceutical companies, Congress created the unique research grants program in 1983 to provide funding to academic investigators and small companies for pivotal clinical trials on new orphan drugs, medical devices, and medical foods for rare diseases. The investment necessary for research and development of new drugs and devices is too large in comparison to the size of the small potential market for a rare disease.

The funds made available through the FDA’s Orphan Products Research Grants Program encourage academic scientists and small companies to conduct clinical trials at research institutions throughout the nation to develop the preliminary evidence necessary to attract commercial sponsors. For small unprofitable pharmaceutical companies that cannot afford to pay all the costs of clinical research, these grants underwrite some of their research expenses.

Since 1983, the FDA has approved 220 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 FDA Orphan Products Research Grants Program. 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.

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 children’s lungs to expand and breathe. Cystic fibrosis, Crohn’s disease, and Multiple Sclerosis drugs are on the market today only because these grants supported some of their research.

FDA’s Budget Request for FY 2002

According to the Administration’s budget request, the FDA will receive a total of $1.414 billion for FY 2002, an increase of $123 million or 9.5% over FY 2001. Unfortunately, the Orphan Product Research Grants Program will receive absolutely no increase in funding –

“$317,547,000 shall be for the Center for Drug Evaluation and Research and related field

activities in the Office of Regulatory Affairs, of which no less than $12,534,000 shall be

available for grants and contracts awarded under section 5 of the Orphan Drug Act.”

Diane E. Dorman, Sr. Dir. for Public PolicyNational Organization For Rare Disorders - Washington Office3907 Blackburn Lane, #22, Burtonsville, MD 20866Office/, Fax/, Cell/ddorman@...