Drug repurposing for rare: progress and opportunities for the rare disease community

Repurposing is one of the key opportunities to address the unmet rare diseases therapeutic need. Based on cases of drug repurposing in small population conditions, and previous work in drug repurposing, we analyzed the most important lessons learned, such as the sharing of clinical observations, reaching out to regulatory scientific advice at an early stage, and public-private collaboration. In addition, current upcoming trends in the field of drug repurposing in rare diseases were analyzed, including the role these trends could play in the rare diseases’ ecosystem. Specifically, we cover the opportunities of innovation platforms, the use of real-world data, the use of artificial intelligence, regulatory initiatives in repurposing, and patient engagement throughout the repurposing project. The outcomes from these emerging activities will help progress the field of drug repurposing for the benefit of patients, public health and medicines development

Drug repositioning for rare diseases: Knowledge-based success stories

International audienceWhile more than 7000 rare diseases have been identified, only about 5 percent benefit from a licensed treatment. As the majority of these diseases is life threatening, these facts underscore the need for new drugs. Drug repositioning is an alternative strategy in drug development, which represents an attractive opportunity for rare diseases. Drug repositioning (also called drug repurposing, drug reprofiling or drug re-tasking) consists in identifying for an already approved or investigational drug a new use outside the scope of the original medical indication. Drug repositioning is considered in the field of orphan drugs as being a faster and somehow less costly strategy than traditional new drug development for pharmaceutical companies. While several successful repositioning cases have been discovered by serendipity, most successes straightly derive from the molecular characterization of the concerned disease. This short commentary is mainly dedicated to these rationally-based success stories

Belgian cost-effectiveness analysis of hydroxocobalamin (Cyanokit) in known or suspected cyanide poisoning

Background: No published burden of illness or cost-effectiveness studies on cyanide (CN) poisoning exist. A health economic model has therefore been developed to determine the cost-effectiveness of hydroxocobalamin (Cyanokit) in suspected and known CN poisoning. Objectives: To assess the cost-effectiveness of hydroxocobalamin versus standard treatment in known or suspected CN poisoning in Belgium. Methods: Probabilities for neurological sequelae and mortality were applied based on phase III trials, literature data and the hydroxocobalamin preclinical trial. Since no cost and utility data for CN poisoning exist, costs and utilities of very comparable or related diseases derived from publicly available Belgian sources and literature were applied. Direct medical costs from the public healthcare payer's perspective were used. The time horizon was 1 year. Sensitivity analyses were performed to assess the robustness of the results. Results: Base-case analyses versus standard treatment revealed cost-effective results (incremental cost-effectiveness ratio= 9921(sic)/QALY) in suspected CN poisoning and dominance (more effective and cost-saving) in known CN poisoning. It was determined that 17 lives could be saved and one sequel prevented per year on a national level using hydroxocobalamin treatment. One-way sensitivity analyses varying efficacy, costs, utilities and time horizon demonstrated the robustness of the results. The results were most sensitive to the probability of death and neurological sequelae, but remained within acceptable limits of cost-effectiveness. Furthermore, it was shown that a longer time frame (5 or 10 years) leads to even more favourable cost-effective results. Conclusions: Hydroxocobalamin appears cost-effective to dominant compared with standard treatment in CN poisoning from a healthcare payer's perspective

Drug repurposing: From the discovery of a useful pharmacological effect to making the treatment available to the patient

International audienc