Oxford PharmaGenesis recently attended the 8th annual World Orphan Drug Congress (WODC) in Barcelona, which highlighted the importance of early and effective collaboration and communication between patients and other healthcare stakeholders, particularly in the rare disease space.
“It was a privilege to meet with so many stakeholders prioritizing the unmet needs of patients with rare diseases,” said Dr Kim Wager, Senior Medical Writer. “It’s impossible not to be moved by the stories of patients and their families, and excited by the potentially disruptive technologies under development.”
Four themes provided the focus of the congress: gene therapy, science and strategy, clinical development and approval, and access. Underpinning these themes was the recognition that the development of therapies for rare diseases, while challenging, shows tremendous potential. Both the presentations and the round-table discussions provided an excellent forum for discussion, with representation from pharma, biotech, regulators, health technology assessment bodies, payers, patient advocacy groups and patients.
Rapid progress is being made, necessitating adaptation of approaches to drug development and commercialization. Innovative treatments, such as gene correction or RNA interference, present areas in which establishing and communicating value may be difficult. Current reimbursement paradigms were not designed to accommodate either patients with rare diseases or therapies that may provide a cure with a single treatment. Key to this is a patient-centric approach to understanding clinical outcomes and assessment of value, and a sophisticated mechanism of pricing and reimbursement.
“Rarely have I attended a congress that provides such opportunity for patients with rare diseases to interact directly with the pharmaceutical industry and its suppliers,” said Laura Schmidt, Senior Medical Writer. “The round tables facilitated open and frank discussions between the two parties, and will hopefully support the development of valuable collaborations in the future.”
Oxford PharmaGenesis is committed to making a difference to patient health. Through its specialist services, including rare and orphan disease, patient engagement and value demonstration, the company is well placed to continue driving progress in these exciting times.
