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New FDA ‘Rare Disease’ Division Being Proposed to U.S. House Committee on Appropriations

May 22, 2010 by  
Filed under Featured Stories

The Kakkis Every Life Foundation is leading the effort to request $10 million in the fiscal year 2011 Ag-Rural Development-FDA Appropriations bill directing the U.S. Food and Drug Administration to establish a new review division for Biochemical and Genetic Diseases within the Center for Drug Evaluation Research (CDER), Office of New Drugs.

A letter from a number of rare disease foundations and advocates was sent to Chairman David Obey and Jerry Lewis and Chairwoman Rosa DeLauro, and Ranking Member Jack Kingston requesting the funds for the new FDA division. This would allow the Food and Drug Administration to build the human and scientific resources necessary to create a more specialized drug review by experts who understand rare diseases and the issues facing rare and ‘ultra orphan’ diseases.

Here is why a new FDA division is needed.  Have you ever tried to find some compelling statistics on Rare Disease on the Internet?  Try a Google or Bing search and see what comes up. It’s pretty frightening given that 30 million people, or 10% of the U.S. population, are living with rare disease.  Where’s the data for all these people?  It does not exist.

Since receiving our orphan drug designation for cyclodextrin last week from FDA, I have been thinking about the statistics that were published in the Wall Street Journal.  The Wall Street Journal referenced statistics from the Kakkis Every Life Foundation and BioMedical Insights in a blog post about our FDA approval:

Last year (2009), only 160 applications — out of 250 requests – received an orphan drug designation from the FDA. “Ultra orphans” make up less than 15% of orphan drug designations even though they represent more than 80% of identified rare diseases, according to data prepared by the Kakkis EveryLife Foundation and BioMedical Insights.

Ok, let’s do some math based on the data from the Journal and what we currently know about Rare Disease.  We’ll end up with even more shocking statistics!

Reasons Why New FDA Rare Disease Division is Needed Now

There are approximately 7000 identified rare diseases afflicting about 30 million American’s or 10% of the U.S. population. If 80% of identified rare diseases are ‘ultra orphans’ — affecting fewer than 6,000 patients in the U.S. per disease state — this means approximately 5,600 different rare diseases are ‘ultra orphans’. 5,600!

We know that in 2009, only 160 applications — out of 250 requests – received an orphan drug designation from the FDA. If only 15% of these designations are for ‘ultra orphan’ diseases, this means the FDA approved an application for approximately 24 ‘ultra orphan’ diseases out of 5,600 that exist.  It’s clear that pharmaceutical and biotech companies are simply not developing ‘ultra orphan’ drugs for people. If this is not a crisis situation, I don’t know what is.

According to the FDA, between 16-17% of drugs that receive an orphan drug designation will eventually make it through the pipeline and receive an approval at some point down the line. If you take 2009 as an example, of the 24 ‘ultra orphan’ designations handed out by the FDA, only about 4 or less will eventually become FDA approved drugs to treat people.  Less than 4 approved drugs a year on average to treat 5,600 different ‘ultra orphan’ diseases.

Statistics show that between 50%-75% of people afflicted with rare diseases are children. If 30 million American’s have rare disease,  between 15 and 22 million are children.  What we don’t know is how many of the 15-22 million children diagnosed with rare diseases are actually afflicted with ‘ultra orphan’ diseases where almost no drug development is happening.  The numbers must be in the  multi-millions.  Obviously, the Orphan Drug Act incentives are not working to encourage Pharma or Biotech companies to develop drugs for diseases that impact 6,000 people or less per disease state!

Given that most ‘ultra orphan’ diseases are fatal, life threatening or chronic, an economist needs to calculate the total financial burden of rare diseases on the U.S. healthcare system. This is what the savvy people in the Alzheimer’s community are finally doing to get the nation to pay attention to the dementia crisis.

With rare diseases and ‘ultra orphan’ rare diseases, we’re talking billions and billions of dollars in health care costs to care for children who have no treatments or drugs even entering the pipeline to help them. Very few people except for the folks at the Kakkis Every Life Foundation seem to understand the gravity of the situation facing the nation.

In my mind, establishing a new FDA division for rare diseases is a baby step but we must start somewhere because where things stand today is simply not acceptable for millions of families and children. It’s time the U.S. House Committee on Appropriations makes rare disease a priority and grants the money for this new FDA division.

It’s also time that the government passes new laws to give pharmaceutical companies and biotechs novel incentives to develop drugs for ‘ultra orphan’ rare diseases.


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  1. […] I posted a blog with some extremely alarming statistics I worked out on paper. For example, if 80% of all rare […]

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