A pharmaceutical executive based in Kentfield in Marin County, California, Alix Alderman has served as head of regulatory affairs/market access and vice president of PellePharm since 2017. Kentfield resident Alix Alderman’s drug development expertise include a pronounced focus on rare diseases and the orphan drugs used to treat them.
Briefly defined, the term “orphan drug” refers to a pharmaceutical developed for a rare disease and very small population; typically defined from a regulatory perspective as less than 200,000 patients in the US. Despite the small number of affected patients, these drugs are still be subject to clinical trials to determine a suitable benefit-risk assessment by FDA, compliance with orphan drug laws, and for reimbursement decisions by payers. The conditions that they treat are so rare that they are often simply not feasible to develop and commercialize under normal market conditions and can be priced highly, making reimbursement for a patient all the more critical.
Pharma’s Almanac Magazine lists technical challenges first on its list of problems in the world of orphan drug development. Because diseases that affect very small patient populations do not typically have a long history of scientific study, existing research provides limited assistance during the drug development process. This leads to informed risk-taking by developers to select trial endpoints and clinical outcomes assessments that are either novel or originally designed for studies in much larger patient populations, but nonetheless have a high probability of being informative and impactful based on very small numbers of trial participants. Close collaboration with FDA throughout the development process tends to be essential, as is early insight from payers. An understanding of the natural history of a rare disease and the spectrum of the disease can aid educating key stakeholders toward alignment on appropriate efficacy outcomes. Because orphan drugs are often complex in nature and require small production numbers, the manufacturing process can also present tremendous challenges.
