HTA and Regulatory Perspectives and Interactions: bridging the gap

Transcription

1 HTA and Regulatory Perspectives and Interactions: bridging the gap For Colleagues at the 2014 CADTH Symposium April 7, 2014 Gatineau, Canada Lawrence Liberti, MS, RPh, RAC Executive Director Photo credit:

2 Landscape A Viewpoint Convergent Collaborative Enabling Complimentary evidence requirements from regulators and HTAs Tiered but convergent landscape between high, middle and low income countries Trust: Acceptance of a Collaborative/work sharing environment between agencies/regions Managing uncertainty: Innovative technologies for disease identification, treatment, management, adherence and vigilance monitoring Priorities: Patient and HCPs- facilitating access to safe, high quality and effective medicines globally Adoption of Risk-Based Facilitated Regulatory Pathways development, approval, inspections, life cycle management, vigilance

3 CIRS- Project Differences in priorities seen between functions In this example only two scenarios overlap between Regulatory and HEOR/Access SC04 Evidentiary standards used by HTA agencies will be integrated into licensing body requirements with separate, but parallel reviews SC19 Interaction between licensing bodies and HTA agencies will increase

4 HTA and Regulatory Alignment Bruening L: Regulatory and HTA Alignment. Presented at CIRS Workshop Is there a commonality across the structured decision frameworks used by HTA and regulatory agencies? which took place 1-2 October in Surrey UK.

5 How could the expectations of diverse stakeholders, including sponsors, regulators and HTA, be aligned in an international environment? Through Joint Scientific Advice Common or aligned evidentiary requirements Consistency in review package evidence

6 Number of consultations Timing of HTA-related scientific advice at a global level 12 Timing of scientific advice by the type of advice 10 2 Formal advice from multiple HTA agencies presented in the same meeting 8 2 Formal advice from a single HTA or payer agency KOL Panel with primary purpose of payer information Company sponsored payer advisory board 2 0 Ph II (7,5) Ph III (4,3) 1 1 Timing of development when advice was sought (Number of products, Number of companies)

7 The number of licensed products that incorporated HTA requirements into Phase 3 trials, by requirement type HTA acceptable primary endpoint Secondary endpoint(s) Inclusion of a comparator arm in the trial Comparator choice Use of surrogate outcomes Relative / Comparative efficacy evidence Specification of superiority margin Health-related quality of life Specification of non-inferiority margin Biomarkers used as a measure of efficacy Technical requirement Efficacy Safety Safety evidence Benefit risk evaluation Study design Trial duration Analysis methodology Trial follow-up and longer-term outcomes External validity of trial Use or applicability of patient crossover Use of adaptive trial design Trial design Patient selection PROs Patient selection Sub-group analysis methodology Patient enrichment (biomarkers) PRO methodology Use of PRO measure Place in treatment pathway Unmet need Cost effectiveness Budget impact / affordability issues Societal benefit Need for companion diagnostic Extra patient visits required (CIRS HTA Industry Survey 2013) Number of products Non-technical requirement Value to healthcare system Cost Non-price barriers to market

8 Percentage of Products HTA review package by jurisdiction (an Overlap with Regulatory Dossiers?) 100% 90% 80% 70% 60% 50% 40% 30% 20% 10% 0% Australia Canada England France Germany Italy Spain USA - Wellpoint Phase III pivotal study,phase IIIb pivotal study,real-world evidence Phase II study,phase III pivotal study,phase IV study,real-world evidence Phase II study,phase III pivotal study,real-world evidence Phase III pivotal study,phase IIIb pivotal study Phase IIIb pivotal study Phase III pivotal study Phase II study,phase IV study Phase II study,phase III pivotal study,phase IIIb pivotal study Phase II study,phase III pivotal study Phase II study (CIRS HTA Industry Survey 2013)

9 Evidence used for recommendation by HTA agency Total responses Input from Clinical Professionals combines data from the fields: Written submissions from clinical professionals and Personal statements from clinical experts presented at committee meeting Input from Patients combines data from the fields: Patient group submitted information, Written submissions from patient experts and Personal statements from patient experts presented at committee meeting 5 agencies provided data overall Total reviews = 18 (CIRS HTA Agency Survey 2013) [Q17]

10 The association between European Medicines Agency approval and Health Technology Assessment recommendations: HTA Assessment Outcomes (n= 86) The longer the approval time at EMA, the less beneficial the HTA recommendation was* There were negative correlations between approval time in HTA recommendations in England HTA recommendations in Netherlands total indicator of HTA recommendations * All of the correlations had marginally significant tendencies (Based on work conducted by Iga Lipska, Utrecht University 2014) 10

11 Regulatory and HTA interactions: considerations The independent regulatory and HTA systems are grounded in science and focused on meeting stakeholder needs. Scientific advice: provides insights into what is expected, practical, and achievable especially when provided jointly Opportunities to align regulatory and HTA requirements maximise process efficiency, consistency of outcomes. Sponsors: should address both regulatory and HTA data requirements from the earliest stages of development Heterogeneous HTA processes are moving towards alignment: integrating diverse needs (ie comparators, study designs) will be challenging Convergent-Collaborative-Enabling: How can industry, regulators and HTAs align to facilitate the evolution to these goals?

12 HTA and Regulatory Perspectives and Interactions: bridging the gap For Colleagues at the 2014 CADTH Symposium April 7, 2014 Gatineau, Canada Lawrence Liberti, MS, RPh, RAC Executive Director Photo credit: