With regard to HTA and reimbursement discussions, the Health Technology Assess- ment International (HTAi) Policy Forum supports and promotes ongoing discussions and scientific advances in this field. The most recent deliberations (2013) deal with HTA and value, but definitions of value vary depending on the stakeholder view (patient, societal, health system, industry).50 However, all participating HTA experts agree that patient health and benefits are central, and that patient health improvement 46 Cartistem has become the world’s first allogenic, off-the-shelf MSC-based product (umbili- cal cord blood (UCB)-derived MSCs for the treatment of traumatic and degenerative osteoarthri- tis). In 2011 the Korean company FCB PharmiCell received Korean FDA approval for commercial sale of HeartiCellgram (autologous bone marrow-derived MSC) indicated for post-acute myocar- dial infarction treatment.
47 The Osiris Therapeutics Inc. product Prochymal consists of allogenic MSCs. The company was granted an authorisation for the treatment of acute graft-vs-host disease (GvHD) in children under Health Canada’s Notice of Compliance with conditions (NOC/c) in May 2012.
48 Wang S., Qu X., Zhao R. C. (2012) Clinical applications of mesenchymal stem cells. J. Hema- tol. Oncol. 5: 19.
49 Eichler H. G., Oye K., Baird L. G., Abadie E., Brown J., Drum C. L., Ferguson J., Garner S., Ho- nig P., Hukkelhoven M., Lim J. C., Lim R., Lumpkin M. M., Neil G., O’Rourke B., Pezalla E., Shoda D., Seyfert-Margolis v., Sigal E. v., Sobotka J., Tan D., Unger T. F., Hirsch G. (2012). Adaptive licensing: taking the next step in the evolution of drug approval. Clin. Pharmacol. Ther. 91 426–437. 50 Chris Henshall and Tara Schuller, on behalf of the HTAi Policy Forum.HEALTH TECH- NOLOGY ASSESSMENT, vALUE-BASED DECISION MAKING, AND INNOvATION. International Journal of Technology Assessment in Health Care, 29:3 (2013), Cambridge University Press 2013 doi:10.1017/S0266462313000378.
Background Paper: HTA and Value: Assessing value, making value-based decisions, and sustaining innovation. HTAi Policy Forum Meeting 2013, 3-5 February 2013, Barcelona, Spain.
is considered a core element of value. Wider elements of value include other benefits for patients (e.g. less burdensome, improved time to diagnosis, early detection), bene- fits for caregivers, and benefits for health care and society in general. Although value is often defined in economic terms, there are divergent views about whether or not value is proportional to cost, i.e. cost-effectiveness thresholds. value measurements (both core and wider) can be quantitative and qualitative. There are different approaches that health systems take to value-based decision-making.
The Dutch IMDI (Innovative Medical Devices Initiative, part of the Dutch Life Sciences & Health key economic sector) offers another way to approach the HTA of medical devices. IMDI is investigating whether it is possible to establish potential clin- ical use and added benefit in a very early stage of device development. It has defined added value along three lines, which takes a system approach instead of an individual patient approach:
• Added value in health care/public health, which is further defined as health gains, quality of life, safety, self-reliance, and effect on the number of people working in health care.
• Added value in industry/economy. The Ministry of Health in the Netherlands does not want to limit entrepreneurship in the health care industry by imposing stricter controls. Its view is that innovation in health care would benefit from social inno- vation (such as self-management, informal care, community welfare services) or innovation in the organisation of health care, and not so much from regulation. • Added value in research.
In the UK, the National Institute for Health and Care Excellence (NICE) provides guid- ance to the government on the clinical benefits and cost-effectiveness of selected new and established technologies. The Institute provided a guidance document with an overview of the principles and methods of health technology assessment, describing key principles of appraisal methodology.51 However, despite continuous development of technology appraisal methodology, there remain many areas of controversy and uncertainty. See also Chapter 5.
Summary
Based on this field survey, it is the opinion of the committee that, in every phase of a medical device’s life cycle, it would be of major value to provide more explicit guid- ance on how to perform studies to assess the benefit or added benefit of the device and which type of studies are possible and needed given the type of device and the intended context and users.
51 Guide to the methods of technology appraisal 2013, National Institute for Health and Care Excellence (NICE).http://publications.nice.org.uk/guide-to-the-methods-of-technology-ap- praisal-2013-pmg9.
Chapter 4 therefore offers an overview of research approaches for collecting evidence that is not limited to clinical investigations needed for CE marking, but also include guidance on conducting clinical research with CE-certified devices. Chapter 4 aims to provide detailed guidance on investigating the different clinical benefits of medical device use while acknowledging the specific nature and market of medical devices. Improved guidance in assessing the clinical benefit or added benefit of medi- cal devices will lead to more clinical and statistically relevant data that can be used to inform CE-marking, uptake and reimbursement discussions.