Rare Disease Trial Strategies

For many patients with rare diseases, finally receiving a diagnosis is a major milestone, but a lack of proven treatment options means that their prognosis is unchanged. Approved drugs are available for just 5% of the approximately 7,000 rare diseases identified so far¹, many of which are life-threatening or life-limiting. The pharmaceutical industry is making inroads through R&D despite the unique challenges facing clinical trials for rare diseases, not to mention often navigating without regulatory precedents and with an uncertain commercial outlook. Progress can therefore feel slow in spite of the priority that many drug companies place on these patients and their unmet needs.

Our white paper focuses on clinical trials, aligning the latest trends in rare disease R&D viewed through the lens of Citeline’s gold-standard clinical intelligence with unique patient insights uncovered in a survey conducted in partnership with Rare Patient Voice. From this, we can share a set of practical recommendations for study sponsors to adopt in their approach to clinical trial design, stakeholder engagement, and patient recruitment. This provides pharmaceutical companies and contract research organizations with a framework from which to accelerate their investments in rare diseases and better serve the needs of the millions of patients requiring new and better treatments.