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The Orphan Drug Act: Does it Help or Hurt?
Introduced with good intent, some of the provisions of the Orphan Drug Act may compel the FDA to rethink its strategy.
The Orphan Drug Act, introduced by the FDA in 1983 was intended to treat conditions affecting less than 200,000 patients per year. Since drug developers would not profit from investing in drug programs intended for such a small section of the population, the FDA hoped to provide incentives to the pharmaceutical industry through this Act. Although well-intended, the outcomes may not be those intended by the drug regulatory authority.
Orphan drugs can be very high priced, and they also come with a 7-year marketing exclusivity, which kills competition for the manufacturer. They are also exempt of the branded prescription drug fee levied through provisions of the Affordable Care Act. Taken together, these incentives may be distorting the original intent of the Act.
Read more at Pharmaceutical Online: http://bit.ly/1FDsLXD
Neurologists Share Tips for Securing Patient Access to Gene Therapies
March 19th 2025Tenacious efforts at every level, from the individual clinician to the hospital to the state to Congress, will be needed to make sure patients can access life-saving gene therapies for neuromuscular diseases.
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EMBARK Data Show Continued Improvements With DMD Gene Therapy
March 19th 2025Data from the EMBARK trial of delandistrogene moxeparvovec in patients with Duchenne muscular dystrophy (DMD) show that benefits in functional outcomes, gene expression, and muscle imaging persist 2 years after receiving the gene therapy.
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