Orphan Drugs: Future opportunities and challenges
The orphan drug sector is booming. Record product approvals, robust pipelines and regulatory incentives. But will the good times last? As the market matures expect increasing competition, clinical research pressures and mounting payer scrutiny. It’s time to plan for the future.
The Future of Orphan Drug provides a 360-degree scan of the horizon for rare disease therapies. Through primary research with leading industry experts, the report identifies key strategic and tactical pressure points for companies seeking to consolidate or enter this high-growth space.
“You will see more companies getting into this space. We will see more novel treatments come out for diseases that never had a treatment. There will be more patient involvement in the process as we go forward.”
Tom Croce Head of Global Patient Advocacy, Shire
“The role of medical affairs is even more critical and more deep than in these larger indications. Part of it is that you're basically dealing with a disease that might affect a couple of hundred or 1000 patients across the globe. That means you have maybe a dozen of real experts in that field, so there is a much more individual collaboration with these world-class expert centres in a given disease that we need to engage with. It's a much closer relationship in the sense that we continue to support the research in these centres; we continue to support medical education and we continue to support disease awareness, which is very critical.”
Hartmann Wellhoefer
“I think a really important thing is the cultivation of creative ways to continuously generate data. The key challenge with an orphan condition is you have a small population. For any drug that comes out, there's lots of questions outstanding - even when it's approved - on long-term safety, real world safety, real world efficacy, efficacy in subpopulations, efficacy in combination with other drugs, efficacy looking at more of these real world endpoints that may or may not have been included in your pivotal trial.”
Vice President, Head of Medical Affairs, Biotechnology Company
“[You need to] get people with the right level of skills. You need to have people who can learn quickly where technical expertise is concerned but who are excellent communicators, who are having a good level of soft skills and the mental flexibility to deal with the fast pacing change in our industry. To attract and retain and develop those people will be the key challenge for medical affairs but not just medical affairs in the industry.”
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