Orphan drugs are at the forefront of medical science and offer a chance for the pharma industry to make a real difference to people’s lives; there’s also the lure of potentially lucrative commercial benefits for those that succeed. But finding the right balance between return on investment (ROI) and optimum patient access is proving difficult. And with only 415 orphan drugs in the marketplace, it’s still early days.
“The ROI of Orphan Drugs: Ensuring Optimal Returns” centers on the commercial perspective and provides the inside view from experts already wrestling with the issues arising in the emerging world of rare disease treatments
The orphan drugs market is lucrative and growing, however its particular challenges require “go to market” approaches that differ substantially from the norm – especially if there is any hope of ROI being achieved. This places the pharma industry at a crossroads on many issues. Should orphan drug prices reflect the level of unmet need, or be built from the bottom up to recoup R&D investment? What ethical stance should the industry be adopting? Are protracted legal battles about exclusivity setting the right tone? Launching an orphan drug and then repurposing it may make sense commercially but is it the right path in the longer term?