Creating win-win-win situations with managed entry agreements? Prioritizing gene and cell therapies within the window of opportunity.

Autor: Callenbach MHE; Division of Pharmacoepidemiology and Clinical Pharmacology, Utrecht Institute for Pharmaceutical Sciences (UIPS), Utrecht University, Utrecht, the Netherlands., Goettsch WG; Division of Pharmacoepidemiology and Clinical Pharmacology, Utrecht Institute for Pharmaceutical Sciences (UIPS), Utrecht University, Utrecht, the Netherlands; National Health Care Institute (ZIN), Diemen, the Netherlands., Mantel-Teeuwisse AK; Division of Pharmacoepidemiology and Clinical Pharmacology, Utrecht Institute for Pharmaceutical Sciences (UIPS), Utrecht University, Utrecht, the Netherlands., Trusheim M; NEWDIGS, Tufts Medical Center, Boston, MA, USA. Electronic address: mark.trusheim@tuftsmedicine.org.
Jazyk: angličtina
Zdroj: Drug discovery today [Drug Discov Today] 2024 Jul; Vol. 29 (7), pp. 104048. Date of Electronic Publication: 2024 Jun 01.
DOI: 10.1016/j.drudis.2024.104048
Abstrakt: Outcome-based reimbursement models are gaining attention for managing the clinical uncertainties and financial impact of gene and cell therapies. Little guidance exists on how such models can create win-win-win situations, benefiting health-care payers, health-technology developers and patients. Our innovative approach prospectively prioritizes therapies for which a 'window of opportunity' might occur through the analysis of health-technology assessments and product characteristics. Within this window, one size does not fit all, and depending on the extent of clinical uncertainty and potential added benefit levels, different win-win-win situations exist in the United States, the United Kingdom and the Netherlands. Dutch Horizon scanning data prioritized etranacogene dezaparvovec (Hemgenix) and mozafancogene autotemcel for their potential to benefit from outcome-based reimbursement models. These insights extend beyond gene and cell therapies, and could help to provide sustainable health care and patient access to innovative therapies.
(Copyright © 2024. Published by Elsevier Ltd.)
Databáze: MEDLINE
Externí odkaz: