Regulatory approval of a new therapy can bring much needed relief to patients—as long as they can access that therapy. Internationally, a diverse set of organizations ultimately facilitates or encumbers access to a novel pharmacotherapy based upon the estimated “value” of a new therapeutic within a system of clinical care. In healthcare systems with competing demands for resources, these assessments constitute a set of necessary steps preceding the addition of a new therapy to formulary with reimbursement for product acquisition at a level commensurate with its clinical utility. However, measures of disease severity or disease progression embedded within randomized controlled trials may create an incomplete framework for supporting such complex assessments. As a consequence, a product may become available under evidentiary standards for approval but remain inaccessible when stakeholders with different perspectives and points of emphasis review the portfolio of data.
Ensuring patient access to new ALS therapies requires more than regulatory approval of such therapies. It is access, after all, that brings much-needed relief to patients—but access is determined by many different forces within the broader healthcare environment. Programs focusing on ensuring access as well as product approval constitute a critical part of the drug development process, and these efforts are best addressed through integrated strategies attached to clinical research that commence at the earliest stages of drug development.