Bridging the gap: Can International Consortium of Health Outcomes Measurement standard sets align outcomes accepted for regulatory and health technology assessment decision-making of oncology medicines.

Autor: Kalf RRJ; Zorginstituut Nederland, Diemen, the Netherlands.; Department of Pharmacoepidemiology and Clinical Pharmacology, University Utrecht, Utrecht, the Netherlands., Vreman RA; Zorginstituut Nederland, Diemen, the Netherlands.; Department of Pharmacoepidemiology and Clinical Pharmacology, University Utrecht, Utrecht, the Netherlands., Delnoij DMJ; Zorginstituut Nederland, Diemen, the Netherlands.; Erasmus School of Health Policy & Management, Erasmus University Rotterdam, Rotterdam, the Netherlands., Bouvy ML; Department of Pharmacoepidemiology and Clinical Pharmacology, University Utrecht, Utrecht, the Netherlands., Goettsch WG; Zorginstituut Nederland, Diemen, the Netherlands.; Department of Pharmacoepidemiology and Clinical Pharmacology, University Utrecht, Utrecht, the Netherlands.
Jazyk: angličtina
Zdroj: Pharmacology research & perspectives [Pharmacol Res Perspect] 2021 Apr; Vol. 9 (2), pp. e00742.
DOI: 10.1002/prp2.742
Abstrakt: Standard outcome sets developed by the International Consortium for Health Outcomes Measurement (ICHOM) facilitate value-based health care in healthcare practice and have gained traction from regulators and Health Technology Assessment (HTA) agencies that regularly assess the value of new medicines. We aimed to assess the extent to which the outcomes used by regulators and HTA agencies are patient-relevant, by comparing these to ICHOM standard sets. We conducted a cross-sectional comparative analysis of ICHOM standard sets, and publicly available regulatory and HTA assessment guidelines. We focused on oncology due to many new medicines being developed, which are accompanied by substantial uncertainty regarding the relevance of these treatments for patients. A comparison of regulatory and HTA assessment guidelines, and ICHOM standard sets showed that both ICHOM and regulators stress the importance of disease-specific outcomes. On the other hand, HTA agencies have a stronger focus on generic outcomes in order to allow comparisons across disease areas. Overall, similar outcomes are relevant for market access, reimbursement, and in ICHOM standard sets. However, some differences are apparent, such as the acceptability of intermediate outcomes. These are recommended in ICHOM standard sets, but regulators are more likely to accept intermediate outcomes than HTA agencies. A greater level of alignment in outcomes accepted may enhance the efficiency of regulatory and HTA processes, and increase timely access to new medicines. ICHOM standard sets may help align these outcomes. However, some differences in outcomes used may remain due to the different purposes of regulatory and HTA decision-making.
(© 2021 The Authors. Pharmacology Research & Perspectives published by John Wiley & Sons Ltd, British Pharmacological Society and American Society for Pharmacology and Experimental Therapeutics.)
Databáze: MEDLINE
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