orphan drug

Pharmaceutical companies can no longer overlook the potential of orphan drug markets.

As pipelines contract and competition squeezes bottom lines, orphan drug markets—disease markets comprising less than 200,000 patients in the United States—have become an attractive means to expand portfolios.

There are many allures to orphan drug markets: Pharma companies entering orphan drug markets enjoy extended patent exclusivity and special tax benefits, while these drugs often fly under payers’ radar, making them less subject to reimbursement restrictions.

However, results haven’t always matched the potential in orphan drug markets. The "rules" for orphan drug commercialization are far different from those for treatments addressing a broad patient base.

Here are four challenges that we see holding back pharma companies in commercializing orphan drugs:

  1. Unlike mass markets, orphan drug markets offer only limited data, meaning companies must frequently make sales and marketing decisions without the assistance of deep insight.
  2. Because an orphan market comprises a limited number of patients, conducting market research for an orphan drug is fraught with difficulties.
  3. Sales models that work on a national scale break down in orphan markets.
  4. Small patient populations for orphan drugs mean advocacy groups have considerable influence on patient perceptions of treatment options, making advocacy support crucial.

The brief video below addresses these hurdles and outlines the ways pharma companies can overcome them.


Matt Gill abstractAbout the Author

Matt Gill is an Associate Principal at ZS Associates and based in the firm’s Boston office. Matt has extensive experience in orphan drug portfolios and has advised clients on a broad range of pharma marketing issues, including market insights, market opportunity assessment, customer segmentation strategy, go-to-market strategy, forecasting and product performance tracking.


Topics: Matt Gill, orphan drugs