Commercializing medical innovation: Scaling access to neurological and rare disease therapies

While advances in biomedical innovations have greatly improved both the quality and quantity of life, new therapeutics designed for neurological and rare disorders still lag behind other disease areas. Conventional product development principles for commercializing medical therapies often do not apply to these areas, aggravated by the relative lack of incentives for private-sector investment in the absence of sufficient market potential. As a result, there is often a mismatch between product innovation and real-world clinical needs, resulting in either therapeutics that do not meet the demand or, conversely, tempering excitement around innovations that seem conceptually interesting but which undergo only the most cursory market analyses to inform product development (Alvarez et al., 2023; Kim et al., 2023; Martin et al., 2025). Neurological diseases have a profound impact on the lives of those affected throughout the world and can impose an undue burden on both individuals and society. With 81 million worldwide cases of debilitating disorders such as Alzheimer’s disease and stroke, the associated direct and indirect costs are estimated to be $1.5 trillion. With an increasingly aging population, these numbers are expected to continue to climb. Diseases such as cerebral palsy, muscular dystrophy, etc. are classified as rare, having a prevalence of less than 1 in 200,000 in the European Union and 200,000 people in the United States. Despite their relative rarity of occurrence, the overall global burden is substantial, as there are over 7000 known rare diseases affecting approximately 400 million people. Neurological diseases are by far the largest category of rare disorders, with a tremendous amount of heterogeneity. Neurological and rare diseases have long clinical development timelines and face high potential failure rates and restrictions that put additional burdens on formulators hoping to create drugs that would provide much-needed support for patients who do not have effective solutions (Singh et al., 2024; Thompson et al., 2024).