More therapies for rare diseases

More therapies for rare diseases

The Washington Post reports:

People often criticize the brand-name drug industry for its focus on blockbuster drugs that can be sold to millions of people with common problems. What people may not have noticed is that drug companies have become increasingly successful in discovering and winning approval for medications that treat rare, or “orphan,” diseases.

The focus on rare diseases is certainly welcome; with new decision support tools –and no doubt support from industry– a higher percentage of people with rare diseases will be diagnosed. It’s good that effective treatments will be available for them.

However, implicit in the Post article is that pharmaceutical companies are turning away from high-selling “blockbuster” drugs to smaller, niche markets. Yet as the biotech companies have demonstrated, drugs for orphan diseases (like Cerezyme for Gaucher Disease) can be blockbusters in their own right, with annual sales in the high hundred millions or even reaching a billion dollars. At some point soon payers are going to find a way to ratchet down prices, which may limit the number of new drugs that ride the orphan/blockbuster gravy train.

April 26, 2006

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