rare disease

Rare diseases can be described in terms of incidence, etiology, morbidity, and survival.  Incidence can vary, however, from rare (e.g., Gaucher disease, cystic fibrosis) to epidemic proportions (e.g., HIV, malaria, cholera).  The incidence of less frequently occurring cancers (e.g., pancreatic and renal cell carcinoma, myeloma, and glioma), although more common in relation to other rare diseases, meet most regulatory requirements for orphan designation.

In a report of the top 200 brand drugs by retail dollars in 2007, 17 top brands (retailing from $144.7 million to $1.837 billion) had an approved orphan use.  Six of these 17 top drugs, in terms of retail dollars, were indicated solely (USA) for an orphan use: opioid dependence, organ transplant rejection, relapsing multiple sclerosis, and cystic fibrosis.  Eleven of the top 17 brands had an additional 2-9 approved uses, which likely factored favorably into their retail sales volume.

Blockbusters can get their start in rare diseases.  Botox (botulinum toxin type A) was first approved in 1989 as an orphan drug for a rare eye movement disorder (blepharospasm) associated with dystonia before seeking approval for cosmetic use.   ClinicalTrials.gov and the Pharmaceutical Research and Manufacturers of America (PhRMA) reported that over 80% of clinical research trials for rare diseases were not industry sponsored.

In regard to the misuse of the Orphan Drug Act, attempts to further subdivide diseases to achieve questionable subsets small enough to qualify for orphan designation seem unlikely to succeed given the requirement to provide support that a condition is a recognized disease with documented incidence.  There are valid and appropriate ways to target subset populations (e.g., pediatrics, refractory patients, severe forms of a more common disease, specific genotype).

The 2007 FDA Amendments Act (FDAAA) priority review (transferable) voucher incentive program and the 2008 common application form agreement between the USA and EU regulatory bodies demonstrate further global  support for rare diseases.  The outlook going forward is that orphan indication exclusivity can be one aspect of a profitable drug’s overall product life cycle, including non-orphan uses, and can contribute to its profitability.

Source:  Drug Information Journal

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FDA Encourages Applications for Orphan-Drug Status

Posted by cdavenport on Friday Mar 12, 2010 Under FDA, Personalized medicine

Getting an orphan-drug designation opens the door to incentives once the FDA approves a medicine for sale in the U.S., including 7 years’ marketing exclusivity and tax breaks.  Last year, just 250 requests for orphan-drug designation were filed, and 160 were successful.  There are roughly 350 orphan drugs approved, covering about 150 rare diseases.  Timothy Coté, director of the FDA’s Office of Orphan Products Development, said that his office is offering 2 workshops with on-the-spot regulatory advice to try to elicit more orphan drug applications.  Over the course of the 2-day workshop, each company met 4 times with FDA staffers who offered advice on 9 critical issues in filling out an application.  Orphan drug applications are 6-7 pages.  At the conclusion of the first workshop, only 14 of the 29 companies submitted applications, although they can still submit at any time.  It usually takes the FDA 60 days to determine whether the designation will be given.  Up to 50 more organizations can attend a second workshop to be held at the University of Minnesota in August 2010.  Dr. Coté said he is considering a workshop in Europe.  Next time, he wants only applicants who can file, without the delay of company approval, at the end of the 2 days to attend.

Source: Wall Street Journal (digital)

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