Cardiovascular Clinical Trialists (CVCT) Forum – Paris 2012 - Lunch Session 1 : Non randomized and/or non-blinded trials (Stuart POCOCK)

MODIGLIANI Lunch Debate Session 1 - Friday November 30, 2012
THE DEVICE THERAPY TRIALISTS WORKSHOP

Chairpersons: Gaetano DE FERRARI, Pavia, ITA - Ileana PIÑA, New York, USA
Webcast: Tariq AHMAD, Durham, USA

Device trial methodology, regulatory and implementation issues
Advances in interventional medical devices are increasingly affecting cardiovascular therapy, just as pharmacological innovation did the generation before. Yet, designing and conducting a device trial is challenging and drug trial designs may not necessarily be applied fully to device trials.
➢ Although there is increasing recognition that this development process substantially differs from that for drugs, how much device trial methodology may deviate from drug trial methodology is a matter of discussion.
➢ Regulation works differently for drugs, devices, and procedures, and there are wide international variations. Although progressively moving toward some alignment, currently, in Europe, industry needs only to fulfill the (light) criteria of “CE” mark before approval.
General sale of devices may be permitted on the basis of proof of safety, rather than of efficacy or effectiveness. In any case, reimbursement claims may require collecting evidence in outcome cost-effectiveness trials. Regulators and commercial bodies should seek consensus.
➢ Innovation is led by device industry which is facing the economic challenge of bringing innovation to the market in a very competitive environment. Multi sponsored trials and the cooperation with public funders may be instrumental in improving knowledge production for a better device therapy.
➢ Interpretation of trial results, and consequently therapy adoption, is another challenge. The strength of evidence is not necessarily the main driver for adoption. Coronary angioplasty in stable CAD is widely adopted while, despite evidence for a beneficial effect of device therapy in heart failure, only a minority of eligible patients is currently offered these options.
➢ Beyond trials aimed at evaluating safety, effectiveness and approval, trials that establish the value of a therapy and hence support utilization in clinical practice are most needed.

The aim of this session is to contribute to identifying and promoting innovative, cooperative and practical solutions that may help filling the gaps between device and drug trials, between CE mark and FDA regulations and between generating evidence and practical implementations.

Session program:
How much one could deviate from “randomized - controlled” trials?
• Non randomized and/or non-blinded trials: When can they be trusted, what can help them to be “acceptable”?
Speaker: Stuart POCOCK, London, GBR
• Options of and alternatives to the “control group” in device trials
Speaker: William T. ABRAHAM, Columbus, USA
• Industry perspective
Speakers: Rob KIEVAL, CVRx, USA - Holger WOEHRLE, ResMed, GER
Approvability issues: Pathway to a more global device approval process
Speaker: Ileana PIÑA, New York, USA
Post approval and registry studies. Advantages and limitations in complementing trial evidence base and improving therapy adoption
Speaker: Ileana PIÑA, New York, USA
Discussant: Roxana MEHRAN, New York, USA
Comparative effectiveness studies. How they may help decision makers and support utilization in clinical practice?
Speaker: Rita REDBERG, San Francisco, USA
Discussant: Kenneth STEIN, Boston Scientific, USA