Working in rare disease presents some very unique marketing challenges. Communications often need to be quite personal and require innovative solutions to reach the small, dispersed patient populations. One of the best ways to stay on top of the trends is by attending conferences such as the annual World Orphan Drug Congress (WODC) 2017, which draws the attendance of representatives from multiple sectors of the rare disease world. These include industry leaders, regulators, patient advocacy groups, government institutions, academics and payers. Here, I’ve captured three trends in the industry that continually surfaced over the 2-day event.
- Patient-centricity is paramount
- Gene therapy continues to be the area of focus
- Digital solutions are expanding into new areas of rare disease management
Patient-centricity is paramount
It comes as no surprise that those working in the rare disease space care about the patients for which they are developing, approving, and providing access to therapies. In fact, I think it’s crucial for one working in this industry to have a vested interest in providing better outcomes for patients with orphan disease. What was pleasantly surprising to me as I listened to multiple speakers from diverse areas of the industry present, was the high level of importance they place on the patient-oriented focus of their business decisions and the increasing effort to include the patients in several steps of the drug development process. From Francois Nader, the former President & CEO of NPS Pharma, who specifically cited the patient as the primary filter to help guide decision-making as he lifted his company from near collapse to launching two rare disease products and selling to Shire for $5.2B, all the way to the Richard Moscicki, Deputy Center Director for Science Operations at the FDA, who discussed the multiple ways in which the government regulatory organization is working with patients and advocacy groups to ensure the patient voice is heard when discussing drug development guidance. Time and again, the patient was at the center of almost all conversations and presentations, revealing the increased intention to bring focus back to why we all started working in the rare disease industry to begin with—to bring hope to the patients with the greatest unmet needs in rare disease.
Gene therapy continues to be the area of focus
Many industry leaders are making a purposeful effort to amplify their efforts in the area of gene therapy for orphan diseases. From the large, well-established pharmaceutical companies, such as GSK and Pfizer, to the younger specialty biotech companies, like Spark Therapeutics and uniQure, gene therapy is an exciting clinical development opportunity—but, it’s not without its challenges. Bluebird Bio shared some of its challenges in relation to clinical trial design, immunogenicity, and the selection of the best primary endpoint to progress clinical development. Meanwhile, Barbara Sosnowski unveiled Pfizer’s efforts for creative partnerships and collaboration in order to refocus their clinical development programs and include gene therapy in the pipeline.
Digital solutions are expanding into new areas of rare disease management
Coming from the agency background, digital innovation is a topic that often comes up among the marketing and sales team members from industry leaders. What was surprising and different at the congress this year, was the way that digital solutions have infiltrated multiple new areas in the management of rare diseases. For example, Margaret Collins, Professor of Pediatrics at the Cincinnati Children’s Hospital, presented an insightful case study of how she and her team have revolutionized the review process for a current clinical trial by utilizing cloud-based technology and digital image scanning to send whole biopsy samples across the country for pathology review. This solution has not only proven to be effective, it’s also made the process more streamlined, expeditious, convenient, and cost-effective. Similarly, on the convention floor, I talked to multiple vendors who provided information on their unique application of digital platforms to aid in patient disease awareness and education, as well as compliance and adherence—all at a very personal, patient level.
From the podium to the booths on the conference floor, the WODC proved to be a successful event and opportunity to engage and interact with some of the orphan drug industry’s top leaders and true pioneers. With the future looking big and bright for the potential to expand digital innovation and continued development of gene therapy solutions, it remains evident that the industry will stay true to the mission to continually progress, but always put the patient first.