With challenges surrounding clinical trials in rare diseases, such as small sample sizes and heterogenous populations, it is difficult to achieve the comparative effectiveness and safety of a novel product. In its new paper, BresMed, a company specialising in health economics and Medilink member, explores these challenges in more depth and proposes a new framework for overcoming these challenges.
Many rare diseases have no existing disease-altering treatments. In these cases, current treatment is often symptomatic, variable, and unknown. Working closely with clinical and patient experts through formal processes can create consensus and define these pathways, as well as clinically distinct populations.
Lack of head-to-head comparison trial data is another challenge in the field of drugs for rare diseases, which can be addressed through early consideration of factors, such as current treatments and likely comparators, techniques for achieving the best comparison, as well as limitations of the empirical evidence.
In order to overcome week surrogacy relationships between trial outcomes and how patients feel, function, and survive, it is important to identify measures for inclusion in a trial that balance clinical validity with external data availability and can be linked to product value.
Manufacturers should consider how they will capture quality-of-life evidence early in their process of planning and whether to capture the effects on caregivers as well as patients. Literature reviews as well as alliances with patient groups, clinical key opinion leaders and academics can play crucial role here.
Finally, preparing for reimbursement early with a clear value case in mind is a crucial step in the successful demonstration of the value of drugs for rare diseases. it is necessary to take a considered and creative approach to evidence generation beyond the clinical programme and invest in building relations with key opinion leaders.
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