Acceptance of Evidence Transfer Within German Early Benefit Assessment of New Drugs for Pediatric and Adolescents Target Populations

• DOI: 10.1007/s40273-024-01467-8
• Publication Date: 2025-01-17T06:42:59Z
• Authors: Georgia Pick; Dirk Müller; Charalabos-Markos...
• Keywords: Health Economics; Health administration; Pharmacoeconomics

In Germany, all new drugs undergo an early benefit assessment (EBA) by the decision-making body (G-BA). Due to limited access clinical data in pediatric healthcare since 2017, evidence transfer has allowed for from adult studies be used EBA of drugs. This study examines acceptance transfer, aiming understand its correlation with granted added benefit. By searching G-BA database, relevant EBAs were identified. addition descriptive statistics, agreement statistics regarding binary and ordinal extent logistic regression without intercept performed investigate juxtaposing it manufacturers' claims, evaluate impact identified factors on transfer. 14 36 EBAs, was accepted G-BA. They referred four therapeutic areas, received a non-quantifiable subject investigation program. Non-quantifiable implies value itself which can range minor major is considering genuine uncertainty mainly induced theese due not allowing quantification The between claims G-BA's appraisals less than chance [kappa - 0.054 (- 0.158 0.050)] whereas became fair 0.333 (0.261-0.406)]. Congruence mechanism action, alignment disease pattern, transferability efficacy safety, same comparator fundamental Additionally, supportive evidence, breakthroughs, small-scale approval enhanced models yielded similar results showing different model fit explained variance. Evidence hinges upon fulfilling various minimum criteria additional evidence. Availability or older patients group under evaluation crucial.