Capitalizing on Hope: Questionable Marketing Approval and Pricing of a New ALS Drug.

Regulatory agencies must balance patient demands to access new treatments for fatal diseases with limited treatment options while ensuring drug safety and efficacy. However, questionable U.S. regulatory actions resulted in the early approval of AMX0035 to treat amyotrophic lateral sclerosis (ALS) by reconvening advisory commissions to obtain positive decisions and designating the drug as a new molecular entity. Data from one randomized clinical trial suggests minimal delays in disease progression and longer survivability, but debate remains about the lack of confirmatory evidence of effectiveness owing to study limitations. A patient's decision-making process details the experience of using the drug, including perspectives on access, cost, effectiveness, and adverse effects. In line with the "nichebuster" business model, the drugmaker, Amylyx Pharmaceuticals, is charging US$158,000/year/patient and thus forecast to turn a profit on a drug with debatable clinical effectiveness prior to completing a Phase 3 trial. Early marketing approval, despite community demands, is unnecessary and may have reduced access because of the end of a compassionate use program, and the high price tag results in restricted coverage and high out-of-pocket costs. Also, the drug's key ingredients are available as a generic and a supplement.

Subtalar coalitions in the adult.

Tarsal coalitions, while relatively uncommon, are typically identified in adult patients during an evaluation for ankle instability, sinus tarsus pain, and/or pes planovalgus. The true incidence of tarsal coalition is unknown with estimates ranging from 1% to 12% of the overall population. The most common area of involvement of the subtalar joint is the middle facet, and heightened awareness should be present in adult patients with limited motion of the subtalar joint. Standard radiographic imaging, to include a Harris heel view, is recommended initially, although computerized tomography scan and MRI are often necessary to confirm the diagnosis.