The Origin of First-in-Class Drugs: Innovation Versus Clinical Benefit.

First-in-class (FIC) designation became a hallmark of innovation, however, even at the marketing authorization stage, little is known about the clinical benefits these products deliver. We identified the provenance of the FIC drugs that entered the French market from 2008 to 2018 and matched these medicines to the clinical benefit grading by Haute Autorité de Santé (HAS) and Prescrire. Analyses were performed using descriptive statistics to present our findings by drug origin and therapeutic area and to establish the degree of concordance between HAS and Prescrire. Of the 135 FIC drugs identified, 71.1% (n = 96) originated from the industry, 16.3% (n = 22) from academia, and 12.6% (n = 17) from joint partnerships. Three therapeutic areas accounted for most FIC medications: antineoplastic (25.9%, N = 35), anti-infective (14.1%, N = 19), and metabolic (11.1%, N = 15) agents. HAS and Prescrire agreed on 60.74% of clinical benefit gradings. According to HAS, only 5% of all FIC drugs had substantial added benefit, and only 3%, according to Prescrire. HAS and Prescrire graded 45.9% and 68.2%, respectively, of FIC drugs as no clinical benefit and 48.9% and 28.9%, respectively, as some clinical benefit. FIC-designated drugs are primarily of industry (> 70%) rather than academic origin. We found that 55% of FIC medicines that entered the French market over the 10-year period deliver no additional clinical benefit. Whereas FIC medicines may represent important scientific advancements in drug development, in > 50% of cases, the new mode of action does not translate into additional clinical benefits for patients.

Industry funding of patient organisations in the UK: a retrospective study of commercial determinants, funding concentration and disease prevalence.

OBJECTIVES: To assess the relationship between UK-based patient organisation funding and companies' commercial interests in rare and non-rare diseases in 2020. DESIGN: Retrospective analysis of the value and volume of payments from pharmaceutical companies to patient organisations in the UK matched with data on the conditions supported by patient organisations and drugs in companies' approved portfolios and research and development pipelines. SETTING: UK. PARTICIPANTS: 74 pharmaceutical companies making payments to 341 UK-based patient organisations. MAIN OUTCOME MEASURES: Alignment between the commercial interests of pharmaceutical companies and the disease area focus of patient organisations; difference in the volume and value of payments to patient organisations broken down by prevalence of conditions; industry funding concentration, measured as the number of companies funding each patient organisation, the share of overall industry funding coming from each contributing company and the share of industry funding of each organisation comprised by the single highest payments. RESULTS: 1422 payments were made by 74 companies to 341 patient organisations. Almost all funds (90%) from pharmaceutical companies were directed to patient organisations that are aligned with companies' approved drug portfolios and research and development pipelines. Despite rare diseases affecting less than 5% of the UK population, more than 20% of all payments were directed to patient organisations which target such conditions. Patient organisations focusing on rare diseases relied on payments from fewer companies (p value=0.0031) compared to organisations focusing on non-rare diseases. CONCLUSIONS: Companies predominantly funded patient organisations operating in therapeutic areas relevant to companies' portfolio or drug development pipeline. Patient organisations focusing on rare diseases received more funding relative to the number of patients affected by these conditions and relied more heavily on payments from fewer companies compared to organisations targeting non-rare diseases. Increased independence of patient organisations could help avoid conflicts of interest.