Ting Wang1, Neil McAuslane2, Lawrence Liberti3, Hubert Leufkens4, Anke Hövels4. 1. Centre for Innovation in Regulatory Science, London, UK; Division of Pharmacoepidemiology and Clinical Pharmacology, Utrecht Institute for Pharmaceutical Sciences, Utrecht University, Utrecht, The Netherlands. Electronic address: twang@cirsci.org. 2. Centre for Innovation in Regulatory Science, London, UK. 3. Centre for Innovation in Regulatory Science, London, UK; Division of Pharmacoepidemiology and Clinical Pharmacology, Utrecht Institute for Pharmaceutical Sciences, Utrecht University, Utrecht, The Netherlands. 4. Division of Pharmacoepidemiology and Clinical Pharmacology, Utrecht Institute for Pharmaceutical Sciences, Utrecht University, Utrecht, The Netherlands.
Abstract
OBJECTIVES: To evaluate the current practice of companies and agencies to assess the changes made in aligning regulatory and health technology assessment (HTA) stakeholders; to identify areas of commonality of evidentiary requirements that could occur; and to identify strategic issues and trends of regulatory and HTA synergy. METHODS: Two separate questionnaires were developed to assess stakeholders' perceptions on regulatory and HTA alignment, one for pharmaceutical companies and the other for regulatory and HTA agencies. The responses were analyzed using descriptive statistics. RESULTS: Seven regulatory and 8 HTA agencies from Australia, Canada, and Europe and 19 international companies developing innovative medicine responded to the survey. This study provided a snapshot of the current regulatory and HTA landscape. Changes made over the past 5 years were reflected in three main areas: there is an increasing interaction between regulatory and HTA agencies; current conditional regulatory approvals are not always linked with flexible HTA approaches; and companies are more supportive of joint scientific advice. Four types of evidentiary requirements were identified as building blocks for better alignment: acceptable primary end points, inclusion of an active comparator, use of patient-reported outcomes, and choice and use of surrogate end point. CONCLUSIONS: The study showed that the gap between regulatory and HTA requirements has narrowed over the past 5 years. All respondents supported synergy between regulatory and HTA stakeholders, and the study provided several recommendations on how to further improve evidentiary alignment including the provision of joint scientific advice, which was rated as a key strategy by both agencies and companies.
OBJECTIVES: To evaluate the current practice of companies and agencies to assess the changes made in aligning regulatory and health technology assessment (HTA) stakeholders; to identify areas of commonality of evidentiary requirements that could occur; and to identify strategic issues and trends of regulatory and HTA synergy. METHODS: Two separate questionnaires were developed to assess stakeholders' perceptions on regulatory and HTA alignment, one for pharmaceutical companies and the other for regulatory and HTA agencies. The responses were analyzed using descriptive statistics. RESULTS: Seven regulatory and 8 HTA agencies from Australia, Canada, and Europe and 19 international companies developing innovative medicine responded to the survey. This study provided a snapshot of the current regulatory and HTA landscape. Changes made over the past 5 years were reflected in three main areas: there is an increasing interaction between regulatory and HTA agencies; current conditional regulatory approvals are not always linked with flexible HTA approaches; and companies are more supportive of joint scientific advice. Four types of evidentiary requirements were identified as building blocks for better alignment: acceptable primary end points, inclusion of an active comparator, use of patient-reported outcomes, and choice and use of surrogate end point. CONCLUSIONS: The study showed that the gap between regulatory and HTA requirements has narrowed over the past 5 years. All respondents supported synergy between regulatory and HTA stakeholders, and the study provided several recommendations on how to further improve evidentiary alignment including the provision of joint scientific advice, which was rated as a key strategy by both agencies and companies.
Keywords:
HTA-regulatory synergy; drug development; early scientific advice; evidence generation; health technology assessment (HTA); review and reimbursement
Authors: Ting Wang; Neil McAuslane; Wim G Goettsch; Hubert G M Leufkens; Marie L De Bruin Journal: Front Pharmacol Date: 2022-07-18 Impact factor: 5.988
Authors: Lourens T Bloem; Rick A Vreman; Niels W L Peeters; Jarno Hoekman; Menno E van der Elst; Hubert G M Leufkens; Olaf H Klungel; Wim G Goettsch; Aukje K Mantel-Teeuwisse Journal: Clin Transl Sci Date: 2021-05-01 Impact factor: 4.689
Authors: Rick A Vreman; Jacoline C Bouvy; Lourens T Bloem; Anke M Hövels; Aukje K Mantel-Teeuwisse; Hubert G M Leufkens; Wim G Goettsch Journal: Clin Pharmacol Ther Date: 2018-11-08 Impact factor: 6.875