Saturday, December 14, 2019

Orphan Drug Act – A Collosal Failure Considering Rare Disease Drug Statistics

May 23, 2010 by  
Filed under Featured Stories

For the past week, I have been obsessing over rare disease statistics ever since it was published that 80% of all rare diseases are ‘ultra orphans’ – affecting 6,000 people per disease state or less – and only 15% of all rare disease drug applications filed with the FDA are for ‘ultra orphans.’

Yesterday, I posted a blog with some extremely alarming statistics I worked out on paper. For example, if 80% of all rare diseases are ‘ultra orphans’ (affecting 6,000 people or less) and there are 7,000 rare diseases, approximately 5,600 different rare diseases would fall into the ‘ultra orphan’ category.

This morning, I went on the FDA Orphan Drug Database to run some other estimates. Of course, these numbers are estimates but I am sure I am not very far off with my projections.

Fact: Since the Orphan Drug Act was enacted in 1983 to present, there have been 2,182 orphan applications that have officially received the orphan drug designation from the FDA. Of all of the designations submitted, 347 have been approved as drugs by the FDA which works out to about 16%.

Estimate: If approximatley15% of all the rare disease applications submitted to the FDA are for ‘ultra orphans’, then approximately 327 of the total pool of 2,182 designations over the past 25 years would have been given to ‘ultra orphan” diseases.

Estimate: If we know that 16% of the applications eventually receive FDA drug approval, then of the estimated 327 ‘ultra orphan’ designations that have been in the pipeline over the past 25 years, approximately 52 would have moved forward to become FDA approved drugs.  This estimate is for all ‘ultra orphan’ diseases combined. Another way to look at this statistic — over the past 25 years, on average about 2 drugs per year have been approved by the FDA for 5,600 different ‘ultra orphan’ diseases.

Some people have actually tried to look at me with a straight face to tell me that the Orphan Drug Act of 1983 has been a success. A success?  How can anyone claim this drug development system for rare diseases is working for millions of Americans with ‘ultra orphan’ diseases?  The Orphan Drug Act of 1983 is a collosal failure on multiple dimensions. There are no novel incentives in place for Pharma or BioTech companies to develop products for thousands and thousands of ‘ultra orphan’ diseases.

I am planning to contact the National Organization of Rare Disorders (NORD) and the FDAs Office of Orphan Products Development (OOPD) to see if I can confirm if the ‘ultra orphan’ estimates I have worked out are accurate or not.  The last time I contacted NORD, they were unable able to provide me with any relevant statistics or facts on this drug development issue.

I recently found a very interesting budget document from the FDA’s Office of Orphan Products Development online which summarizes the budget program requirements that justify a $22 million request for FY 2011. We’re going to need a lot more than $22 million to deal with a healthcare crisis of this magnitude.  This is why I am in support of a new FDA Rare Disease division being proposed by the Kakkis Every Life Foundation to the U.S. House Appropriations Committee.

The statistic that continues to remain elusive is how many people fall into the  ‘ultra orphan’ category?  By using 500 people as an average for every ‘ultra orphan’ disease that exists, this works out to about 2.8 million people (500 people x 5600 ‘ultra orphan’ diseases).  The number of people could be much higher depending on what the average number turns out to be (or it could be lower), but this estimated number needs to be published to fully understand the scope of the problem.

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