In Defense Of “Me-Too” Drugs: Is Primary Care Losing Out To Rare Disease R&D?

Regulatory and financial incentives have sparked major investment in orphan drug development, but now some stakeholders are questioning whether the focus on orphans is orphaning another market: primary care conditions that are still not adequately treated. Are incentives needed?

In late February, the Food amd Drug Administration approved its fifth novel prescription drug or biologic for 2014: AstraZeneca PLC’s Myalept (metreleptin for injection) for the treatment of a very rare fat disorder called generalized lipodystrophy.

Patients with congenital or acquired general lipodystrophy (also known as Lawrence-Seip syndrome) have a near-absence of body fat in the...

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