Thanks to the breakthroughs in cell and gene therapies, more pharmaceutical and biotechnology companies began ramping up investments in research and development of treatment options for rare diseases. Now, hundreds of potential therapies — far more than ever before — are in the approval process with many set to launch in the near term. While this is an exciting time for potential patients and the industry, without carefully plotted paths to access, the patients who need these novel therapies may encounter many roadblocks, and manufacturers may be putting their innovation at risk.
Manufacturers will need to adjust their patient services — with consideration to reimbursement support, adherence, financial assistance, patient assistance, data aggregation, and other services — to become even higher touch to help patients navigate the terrain, or to establish a safety net to catch patients who fall through the cracks of the healthcare system. Furthermore, because the population sizes for these diseases are relatively small, manufacturers may have more insights about individual patients and their lives, which means robust compliance programs must also be instituted along with the fine-tuned services.