Recent breakthroughs in the space of cardiovascular diseases, oncology and some rare diseases have revolutionized the treatment paradigm and improved outcomes for many patients. Bringing this into the context of regulatory approval and health technology assessments (HTA) across Europe has meant that proving a significant level of efficacy against existing comparators rather than placebo is increasingly hard. Thus, pharmaceutical companies increasingly face difficulties designing their clinical trials in a way that corresponds to the needs of regulators, while at the same time optimizing the overall commercial success of products and minimizing costs associated with their clinical trial programs. There is therefore a growing need for pharmaceutical companies to re-evaluate their trial design strategies.
In light of these considerations, the study aims to assess if a broad, investment-heavy clinical trial strategy results in broader market access and improved commercial success, and to provide a set of recommendations on possible winning trial design strategies.