CRT Plenary Meeting Proposed topics for future workshops and workstreams

Transcription

1 CRT Plenary Meeting Proposed topics for future workshops and workstreams S. Schroeder

2 Surrogate / Composite Endpoints Regulatory workshop Co-chairs: S. Anker / S. Schroeder Postponed to Q4 / 2014 Workshop objectives: To discuss the use and acceptance of surrogate/composite endpoints in drug development and the regulatory approval process. Background: Regulatory approval of new medicines is based on demonstrating beneficial outcomes in studies of the new treatment, and an overall favorable benefit/risk ratio. The workshop will focus on two main themes: Composite endpoints: Mortality & Hospitalization / MACE & Responder analysis / Recurrent events Surrogate endpoints: View of EMA: the case of Heart Failure / Blood Lipids / HbA1c / Natriuretic peptides / 6min Walktest & pvo2 Expected outcome: The expected outcome would be 1 or 2 papers discussing the benefits and potential risks of using surrogate and composite endpoints to bring new treatments to patients.

3 Electronic Health Records: enhancing clinical research and patient care Proposal from GSK Regulatory workshop with clinical trialists, industry, representatives of national/regional healthcare systems and regulators. When: 1-2Q2015 Proposed co-chairs: Andrew Zalewski (GSK), Andrew Morris (Univ of Dundee and Chief Scientific Officer, Scottish Healthcare System), Lars Walentin (Uppsala, Sweden). Objective and scope: The purpose of the workshop will be to engage clinical trialists, industry, the representatives of national/regional healthcare systems (who are also interested in improving the utility of clinical data), and regulators (EMA and FDA) in the discussion on EHR applicability in large randomized clinical trials. After the workshop and the position paper written by its participants the following issues will be highlighted: Current status of EHR in healthcare systems and the nature of any barriers in national/regional healthcare systems affecting participation in clinical research (infrastructure, designated IT resources, privacy issues) What clinical trials or interventions or observational studies are appropriate for application of EHR What CV outcomes of interest can be reliably collected via EHR (efficacy endpoint and monitoring safety), What level of validation will be required by the regulators to rely on information derived from EHR (e.g., traditional adjudication of events vs information derived from EHR vs hybrid model) What novel approaches to study design can be entertained with use of EHR/healthcare systems (cluster randomization, no placebo use, use of registries to test new interventions). Timelines : If accepted by ESC CRT, the scientific program will be developed by 2Q2014 with final agenda and speakers agreed by 4Q2014. Format: plenary sessions with ample time for discussion in the afternoon of the first day and breakout small group discussions during first half of the next day. The paper submission of EHJ will be planned within 6 months of workshop.

4 Different standards of care in ACS (Suggestion from EMA Cardiovascular Working Party) With involvement of the CVS WP members Rapporteurs for the revisions of 2 EMA ACS Guidelines (Guideline for STEMI / Guideline for NON-STEMI) Workshop linked to the Concept paper to be published in May, for consultation, regarding the update of the 2 EMA ACS Guidelines, with a plan to merge them.

5 Workshop topics in relation to EMA Cardiovascular Working Party workplan Rev. 4 of the Guideline on clinical investigation of medicinal products in the treatment of hypertension (EMA/CHMP/29947/2013/Rev. 4) Draft Guideline released for 6M public consultation by 31 January Estimated finalization time: 3Q2014 Rev. 2 of the Guideline on clinical investigation of medicinal products for the treatment of cardiac failure (CPMP/EWP/235/95, Rev. 1) Draft Guideline to be released for 6M public consultation in 3Q2014. Rev. 2 of the Guideline on clinical evaluation of medicinal products used in weight control (CPMP/EWP/281/96 Rev.1) Draft Guideline to be released for 6M public consultation in 2Q2014. CRT Workshop Obesity and CV impact held in October Manuscript under development by Prof. Geneviève Derumeaux and Dr Lata Kler (co-chairs)

6 Development of scientific guidance on post-authorisation efficacy studies In relation to EMA workshop October 2013 on Methods for efficacy in the Everyday Practice Risk Management Plans and Assessment of their Effectiveness Also of interest for the EMA Pharmacovigilance Risk Assessment Committee Role of nephropathy & CVD (renal disease) Surrogate endpoints and imaging modalities in cardiovascular trials (including regulatory acceptability) Larger and larger studies are needed to demonstrate any benefit and suitable surrogates and imaging modalities could define success or non-success at an early stage making investments more efficient. Registry-trials A new way of pragmatic clinical research joining Industry and Academy More synergy/coordination between US FDA and Europe EMA approval processes Methodological platform for multimorbid research. Comorbidity is the norm (in elderly) but clinical research is mostly disease-specific. Risk-based approach to monitoring Responsibility, accountability and insurability of health providers and medical industry

7 Topics for future workstreams Work on awareness, strategies or activities on how to keep patients in a clinical trial In cardiology, long term trials are often needed as they are event driven. On top of that mortality is an important outcome parameter and low in frequency (lost to follow up can bias this outcome) Therefore it is important to keep the patients in the trial (and at best on the randomized treatment) This is a topic where patients, physicians and industry can work together.