Rare Diseases
Chair: Dr Tony Lockett
Approximately 7,000 distinct rare diseases affect 350 million people worldwide, but only 5 to 10 percent of rare diseases have an FDA-approved treatment. There is thus an imperative for continued advancements in Rare Disease drug development, including gene replacement and genome editing approaches. The recent increase in regulatory approval of Gene Therapies offers tangible hope for sustained disease-modifying approaches, with approximately 80 percent of rare diseases having a known monogenic cause.
These remain challenging; however, listening to patients’ experiences, perspectives and challenges and exchanging insights can help shape the future of rare disease management to address the patient’s unmet needs better. Only through open and transparent dialogue between industry, researchers, academics, regulators and patients can we ensure that the patient perspective becomes an integral part of how medicines are developed and delivered to patients.
The FPM Rare Diseases Expert Group aims to provide a platform to help foster open dialogue, education and collaboration with all relevant stakeholders in the rare diseases community. This group seeks to support drug discovery across rare diseases, including orphan drugs, advanced therapies and gene therapies. The group also hopes to engage with and educate clinical healthcare professionals and patients to increase understanding of rare conditions and encourage participation in research.
