Orphan Drugs: Getting Arms around Rare Diseases

Finding ways to bring new therapies for rare diseases to patients in a timely manner, effectively and affordably is an important public health challenge. The key concern for decision makers in the health authorities for all medicinal products including Orphan Drugs is that the treatment demonstrates efficacy through “substantial evidence” from adequate, well-planned, well-controlled clinical trials A successful clinical development programs in rare diseases starts with a tailored approach to ensure the right methodology is employed for the target rare disease therapy. The research methodology needs to be evaluated specifically for each rare disease and the target therapy in the light of all available scientific knowledge by all experts acting in all stakeholders.