The theme of this year’s World Orphan Drug Congress (WODC) Europe was ‘strategy, advocacy and partnering for the world of orphan drugs and rare diseases’, which offered a great opportunity for Oxford PharmaGenesis to showcase our experience in the area of patient engagement. Patients are more informed and more empowered than ever before to manage their health and quality of life. Engaging and communicating effectively with patients are key objectives for all healthcare stakeholders.
The importance of the perspectives of patients and carers in drug development for rare diseases was foregrounded throughout the congress. This was exemplified by presentations and round-table discussions on patient-centric models for repurposed drug development; the need for effective communication with patients to improve gene therapy research; and the opportunities and challenges of patient involvement to improve patient access to licensed medicines.
Patient advocacy groups had a large presence at the congress. Alan Thomas gave an insightful presentation on his own journey from initial diagnosis to becoming the founder of the patient support group Atacsia a Fi – Ataxia and Me. Kay Parkinson, CEO and founder of Alström Syndrome UK and CEO of Cambridge Rare Disease Network, presented her personal story as a mother of two children with Alström syndrome, highlighting the lack of medical understanding, research and financial support available for such a rare disease. Flóra Raffai, Executive Director of Findacure, brought home the importance of patient involvement in optimizing trial design and driving research forward by emphasizing that the patient is the expert in a rare disease.
Other topics that were discussed at the congress included innovative ways to facilitate accelerated access to and approval of medicines for rare diseases; EU rare disease policies; and reimbursement strategies and market access for drugs targeting rare cancers and other non-cancer conditions.
“It was great to see such a mix of participants at the meeting, from pharmaceutical company representatives to payers and patients with rare diseases” said Dr Laura Pearce, Senior Medical Writer with Oxford PharmaGenesis. “The need to engage with patients as early as possible in the drug development process was highlighted during the meeting and the collaboration of all stakeholders should help to make a real difference in the future.”
Considering the core position of patient engagement in our practices at Oxford PharmaGenesis, we were thrilled to see the drive toward understanding and incorporating the perspectives of patients and carers in all aspects of drug development, approval and market access, and we look forward to the next WODC in 2017.[vc_row][vc_column][vc_images_carousel images=”5380,5379,5378,5377″ img_size=”large” autoplay=”yes” wrap=”yes”][/vc_column][/vc_row]
