Professional Biography

Transcription

1 'MIND THE GAP!': HOW SHOULD WE MANAGE THE DIFFERENCE BETWEEN REGULATORY AND REIMBURSEMENT EVIDENCE REQUIREMENTS FOR MEDICAL DEVICES? Nneka Onwudiwe PharmD PhD MBA PRO/PE Regulatory Reviewer Professional Biography 1

2 Disclaimer The views expressed in this presentation are those of the speaker, and should not be construed to represent FDA s views or policies. 'MIND THE GAP!': HOW SHOULD WE MANAGE THE DIFFERENCE BETWEEN REGULATORY AND REIMBURSEMENT EVIDENCE REQUIREMENTS FOR MEDICAL DEVICES? Moderator: Bjoern Schwander, RN, BSc, MA, General Manager, Agency for Health Economic Assessment and Dissemination, AHEAD GmbH, Loerrach, Germany Panelists: Richard Charter, MSc, Head of Market Access & Pricing, EMEA, Becton Dickinson, Basel, Switzerland Karen Facey, PhD, Honorary Research Fellow and Evidence Based Healthcare Policy Consultant, University of Edinburgh, Edinburgh, UK Nneka C. Onwudiwe, PhD, PharmD, MBA, PRO/PE Regulatory Reviewer, US Government, Silver Spring MD 2

3 Issue For most medical devices, evidence requirements for regulatory approval are significantly less than evidence required for reimbursement approval. Regulatory agencies are re-examining the evidence required as demonstrated in US and in Europe with the new Medical Device Regulation. In contrast, medical devices are more likely to be developed by small/medium companies, who can struggle to finance large-scale clinical trials; moreover, short product life cycles make significant investments in lengthy evidence generation impractical. How can we balance the desire to increase the evidence required for regulatory approval without having a negative impact on device development, innovation and patient access? How can patient involvement in HTA/evidence facilitate the trade-off between ensuring quality, safety and having access to innovations? The core question is how should we 'mind the gap' in evidence requirements between medical device regulation and reimbursement requirements? Overview In April 2017, the new Medical Device Directive was approved in Europe: REGULATION (EU) 2017/745 OF THE EUROPEAN PARLIAMENT AND OF THE COUNCIL. This will impact patient access for medical devices. The regulatory/reimbursement perspective including evidence requirements and timelines to fulfil them will be debated. How payers plan to implement decisions based on changing requirements will also be discussed. The perspective of medical device manufacturers will highlight what this means to R&D and innovation of medical technologies, and how they will overcome the challenges in producing higher evidence, including the role of real world evidence (RWE) and risk sharing agreements. Last, the panel will include an often overlooked perspective on evidence and reimbursement: patient involvement in HTA. We will discuss what increased evidence means to patient involvement in HTA, as patients are the ones impacted by the changing access to medical devices and the goal of increased quality and safety of medical devices. Issue Panel Questions What can manufacturers to do to make sure their development programmes are more patient centered and reduce the evidence gap later on? There s lot of discussion about value-based healthcare does that imply we can bridge the evidence gap later? How do we make reimbursement conditions in Managed Entry Agreements manageable? 3

4 Product Lifecycle (PLC) Santos et al. The specificities of medical devices - opportunity for a dedicated product development methodology Devices Medical Product Development Process Discovery and Ideation Invention and Prototyping Preclinical Research Clinical Research FDA Review FDA Post- Market Safety Monitoring Product Launch 4

5 Safety Effectiveness Quality Regulatory Source: Adapted from Device Premarket Evaluation Unmet Need Concept Material Selection Feasibility Study Pivotal Study Functional Study design Safety Testing Design Manufacturing Methods Design Controls Validation Packaging Other Clinical Studies Nonclinical Studies 5

6 Device Postmarket Evaluation Unmet Need Postmarket Clinical Studies Postmarket Surveillance Studies Postmarket Approval Studies Medical Device Reporting Qualification of Medical Device Development Tools Medical device development tools (MDDT) is used to assess the effectiveness, safety, or performance of a medical device Can be qualified for use in device evaluation and to support regulatory decision-making (e.g., COA, biomarker) MDDT qualification can be initiated: 1) FDA identifies an area of need and/or calls for development activity in a specific area; 2) determined by individual or consortia of stakeholders; or 3) developer pursues qualification to foster broad use of the tool 6

7 Patient Preference Information Addresses only patient tolerance for risk and perspective on benefit Qualitative or quantitative assessments of the relative desirability or acceptability of attributes that differ among alternative diagnostic or therapeutic strategies (e.g., effectiveness, safety, means of implantation, etc.) Use of Real-World Evidence to Support Regulatory Decision-Making Generating the types of realworld evidence that can be used in FDA regulatory decision-making for medical devices Real-World Data (RWD)- data relating to patient health status and/or the delivery of health care (e.g., EHRs, claims data, disease registries, patientgenerated data, etc.) Real-World Evidence (RWE)-clinical evidence regarding the usage, and potential benefits or risks, of a medical product derived from analysis of RWD 7

8 Balancing Premarket and Postmarket Data Collection Role of postmarket information in determining the extent of data that should be collected in the premarket setting to support premarket approval Greater reliance on postmarket collection, including real-world data collection, can reduce the extent of premarket data collection Collection of certain data in the postmarket setting when there s uncertainty regarding certain benefits or risks of the device, but the degree of uncertainty is acceptable in the context of the overall benefit-risk profile of the device at the time of premarket approval Reimbursement/Access Procurement/Selection Safety Effectiveness Quality HTA/ Economic Analysis Training Regulatory Health Technology Assessment Source: Adapted from Health Technology Management 8

9 Device Premarket Evaluation Unmet Need e.g., MDDT: 23-item Kansas City Cardiomyopathy Questionnaire (KCCQ) e.g., Kaiser National Joint Replacement Registry Device Postmarket Evaluation Unmet Need Economic Studies Economic Studies 9

10 Ferguson M. Health Economics Outcomes Research and Evidence Strategies. Managing Medical Devices within a Regulatory Framework Example: Vertebroplasty Darryl T. Gray, MD, ScD; William Hollingworth, PhD; Nneka Onwudiwe, PharmD; et al. Thoracic and Lumbar Vertebroplasties Performed in US Medicare Enrollees,

11 Example: Vertebroplasty The Medicare program promulgated no national coverage policies for this procedure after reviewing the available nonrandomized evidence Local Medicare contractors have covered vertebroplasty for various indications since 2001 Vertebroplasty rates nearly doubled from 2001to 2005, increasing by 32.3% from 2001 to 2002 alone Most procedures were performed by diagnostic or interventional radiologists Most of the observed growth preceded the FDA s approval of polymethylmethacrylate cement use for vertebroplasty Example: Vertebroplasty 11

12 Commercial Viability Integration of premarket and post-market data 12