353P Applying a decision-making framework for expanded access to Duchenne muscular dystrophy

• Published in: Neuromuscular disorders : NMD 2024-10, Vol.43, p.104441, Article 104441.684
• Main Authors: Rouet, J., Duro-Ocana, P., Lawrence, C., Zanker, S.
• Format: Article
• Language: English
• ISSN: 0960-8966
• DOI: 10.1016/j.nmd.2024.07.693

Expanded access (EA) to investigational medicines offers a potential lifeline for patients suffering from a serious or life-threatening disease, who have exhausted other comparable or satisfactory alternative therapeutic options and are not eligible to enter a clinical trial. The 21st Century Cures Act requires that a company developing new medicines shall make a policy regarding EA public. Bionical Emas have developed a patient centric decision-making framework for EA starting from a position of “yes, EA will be provided if specific conditions are met”. This framework takes the burden away from patients and families by allowing pharmaceutical companies to work through internal and external barriers backwards to provide reasoning, transparent rationale, and reflection on whether to provide EA to eligible patients. We present a case study in which we applied this framework to Duchenne muscular dystrophy (DMD) and consider disease specific challenges. There are licensed drugs available however, as these therapies are mutation specific, there remains an unmet need for the majority of the DMD population. There is no cure for DMD and most treatment options available are to help manage symptoms and improve the quality of life. As a result, EA programs are considered by DMD patients, families and physicians as a possible route to access potentially effective treatments. There are novel therapies in the pipeline which may be able to address the unmet need however, the pharmaceutical company must assess factors including benefit: risk ratio, the impact on clinical development activities, supply and manufacturing constraints, commercial sustainability, and the needs of the patient community through engagement with Patient Advocacy Groups and Key Opinion Leaders. This decision-making framework enables a pharmaceutical company to assess EA through a clear patient centric approach which is especially crucial for debilitating and life-threatening conditions such as DMD.