Cooking with the Genzyme Recipe: New Players Funding Rare Disease Drugs in Boston

4/12/10

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inside of a pharma company.”

There are also economic advantages to the rare or orphan drug business, despite the relatively small markets for the treatments. For one, the number of patients that need to be treated with an orphan drug during clinical trials can be way fewer than a trial for, say, a heart drug. The FDA may grant orphan drug developers an expedited approval process for their products, shortening the time it takes to bring their drugs to market. (The agency grants “orphan” status to drugs that treat patients with diseases that affect fewer than 200,000 patients in the U.S.) These and other perks can reduce development costs by millions of dollars.

In many cases, rare disease drugs can provide life-changing benefits for patients. This makes the treatments valuable both to the patients and their health insurers who have a vested interest in keeping them healthy. The ability to show such benefits helps pharmaceutical companies convince healthcare payers to provide reimbursements for their drugs. That’s not expected to be the case in large markets where there are a number of “me-too” drugs that work in similar ways, or in markets with generic competitors.

“It’s almost unanimous that all the big pharmas are paying much more attention to rare diseases than they have in the past,” says Mark Miller, a vice president of corporate development at the Indianapolis-based drug powerhouse Eli Lilly (NYSE:LLY). He is expected to travel to Cambridge this week for the Boston Biotech Business Development Conference 2010, where he plans to connect with local developers of drugs for rare disorders and other diseases of interest to his company.

“Clearly there’s a growing opportunity for biotech companies that might be focused in” the rare disease field, Miller says. “I think people look at the Genzyme model and think that that is a reasonable model to try to emulate.”

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  • Jerry Jeff

    Well, here’s hoping for an end to these captive markets. All those billions come out of our health insurance premiums, after all. And what’s up with that picture?