The rare disease industry is building up steam, breaking new ground, and beginning to find its feet as a viable business model. It is a field that is hugely rewarding, as developers reach out to patients who previously had no hope of a cure. But addressing severe unmet medical needs in an industry where every patient counts requires a whole new medical paradigm. Looking to the future, we can see that there are a great many questions that must be asked. How can orphan drug developers get a drug all the way to patients, and deliver great value at the end? How should developers tailor their reimbursement strategy? Where do the unique opportunities lie?
We asked 14 industry experts for their perspectives about the issues faced along the whole orphan drug development journey. Read the following pages for their insights into:
• How (and why) biotechs can partner with pharma
• How to deliver value to patients
• How to get reimbursement and surmount the barriers to patient access
• The perspectives of patient advocates
• The unique opportunities available for orphan drug developers
We hope you enjoy reading these interviews and find them of value, and would like to thank the experts for their input.