Friday, December 19, 2014

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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7 Responses to “Orphan Drug Act – A Collosal Failure Considering Rare Disease Drug Statistics”
  1. Forget about your flawed numbers. Over 50% of new drug discovery occurs in academic medical centers, often as Public Private Partnerships. The new National Center for Advancing Translational Science (NCATS) at the NIH has a large program for funding research into “Therapeutics for Rare and Neglected Diseases.” Remember, the NIH funds $40B in scientific research year – the TRND program is much more than $22M on an annual basis. Don’t look towards the FDA, look towards the NIH.

    • Chris Hempel says:

      Hi Gerald:

      Thanks for your comment. I really would like to understand how these numbers are flawed — here is a recent video by the Myelin Repair Foundation.

      According to the video, we’re spending 139 billion each year, 800,000 research papers are getting published each year and we get about 22 new drugs for all diseases (including all the rare ones too). 21 new drugs for all people? Anyway you look at this, it does not sound like a good return on investment!

      The rare disease drugs would be part of the 21 total — so how many are we getting for rare diseases? Maybe I am off a little since I am doing estimates with publicly available info — maybe we’re getting 5 of the 21? Is that sufficient to deal with 30 million people suffering from rare diseases? There are between 6,000 and 7,000 rare diseases. Are 21 new drugs a year sufficient for everyone? I read it’s costing close to 1 billion dollars now and takes over 10 years to develop a new drug — who is going to spend that for 500 kids with an ultra rare disease? Who is even going to spend 10 million?

      I think my numbers are probably fairly accurate. If they are not, I would expect the NIH, NORD, someone to provide me with the “real numbers.”

      I know about the recently formed NCATS and also TRND — in fact, Niemann Pick Type C is one of the pilot projects for TRND. I am a lucky to be a beneficiary of the program. And even if you are, it’s difficult to get a compound through the Valley of Death. I am experiencing this first hand with trying to develop cyclodextrin as a drug for NPC.

      The bottom line Gerald is all the money we’re spending at the NIH is NOT translating into fast enough treatments and cures for people.

      • w hoh says:

        Cystic Fibrosis affects 30,000 americans and pulmozyme (orphan drug) has extended lives by 15-30 years. That would be a success.

        • Chris Hempel says:

          W Hoh — cystic fibrosis is 1 rare disease out of 7,000+ rare diseases that impact 30 million Americans, or 350 million WW.

          This may be a success for YOU, or if you have a child with CF or are are a researcher studying CF. But for the millions of others, the Orphan Drug Act is a JOKE.

          To try and defend the atrocities that have been committed against the millions of people WW suffering from rare diseases is incomprehensible.

          This is about MONEY, not health.


  2. Jeff says:


    Your correlations are flawed from the beginning. You are basing the efficacy of the Orphan Drug Act on the number of drug applications approved or rejected by the FDA. Have you looked at the overall number of drug applications which are rejected by the FDA? The number is staggering. Also, you need to look at the reasons for the orphan drug rejections. If a drug is rejected due to safety concerns, failed efficacy, etc., why does that reflect poorly on the ODA? How does the FDA rejecting an application relate to the ODA when the ODA primarily allows a company to enter the market for 7 years without competition as well as give tax breaks for R&D? Furthermore, you failed to look at the statistics of orphan drugs for rare disorders prior to the ODA. How can you draw a conclusion without looking at the BEFORE and after? If you did, you would see that in the decade leading up to 1983, only 10 orphan drugs entered the market. From 1983-2007 there were been 269 marketed. That’s a huge difference and why it is considered a success. Why the change? Because big pharma ignored this market due to the limited number of users and inability to recoup their costs. The ODA allowed small biotech firms to enter the market, provide a valuable resource and be protected from competition for 7 years in order to recoup their entry-costs. Because of that you have seen numerous new drugs enter the market which would have otherwise never been developed. I’m usually against government interference in the private sector and yes you could take away the “government control” here but the result would be no one wanting to take the risk with the likelihood that they would never be able to even recoup their entry costs. Remember, it takes 10-14 years and an average cost of $800 million to perform the research, run the clinical trials, and get FDA approval before bringing a drug to market. Trying to do that with only a few thousand potential patients is hard enough. Doing that with multiple other companies vying for the same patient population is not sustainable or attractive to a company as history has proven.



  3. Goodmorning Chris,

    With great interest I read your blog on rare disease statistics (but also the other ones). I think your estimates are not far off from the real numbers. You already know that just like you I am obsessing with these rare disease statistics. In 2008 I presented some EU rare disease statistics at a conference (here is the hyperlink ->

    Please have a look at slide 5: you will find confirmation of your estimates, but then based on EU numbers (1/100.000 = ~5000 patients in de EU)

    Please also take a look at slide 10: Scientific output of the last 40 years for three diseases classes (blood (D), metabolic (E) & nervous system (G); Y= OD in development or approved; N= no OD in development or approved).

    For many ultrarare disorders there is hardly any research being performed to better understand the disorder, diagnostics, identification of potential drug targets. The point I want to make is that the problem for many ultrarare disorders is not just lack of drug development, but also lack of disease research. Yet there are one or two examples of very ultrarare disorders (<500 in EU), eg tyrosinemia type I, with a low scientific output, but already a product on the market. On the other hand we know so much about cystic fibrosis, but a real curative therapy is not yet available. Although life expectancy for CF patients has increased over time as a result of improved healthcare

    It is not realistic that for all ultrarare disorders sufficient knowledge has been gathered or a curative therapy will be developed in the near future (take for example congenital malformations).

    However, I truly believe that there are more "tyrosinemia type I" ultrarare disorders, for which sufficient disease knowledge has been gathered to initiate the development of a therapy. The question is how can we identify these disorders, and who is going to initiate the development. We are currently working on the first question, which perhaps will fuel the interest of pharma companies or initiate the start of a new kind of public-private partnerships.




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