Digital Theses Archive


Tesi etd-03262018-113327

Thesis type
Incorporating Health Technology Assessment (HTA) into Medical Device R&D: the costs and benefits of being an early bird
Scientific disciplinary sector
Corso di studi
Membro Prof.ssa NUTI, SABINA
Membro Dott.ssa SEGHIERI, CHIARA
Membro Prof. Leandro Pecchia
Parole chiave
  • early
  • early cost-utility analysis
  • early economic evaluation
  • Health Technology Assessment (HTA)
  • innovation
  • medical device
  • Research and Development (R&D)
Data inizio appello
Riassunto analitico
The Research and Development (R&amp;D) process in Medical Device (MD) industry is complex, expensive, and plenty of pitfalls. This process is more multifaceted than the innovation continuum in other industries. In particular, the R&amp;D process may require a considerable amount of resources, but resulting in limited benefits for companies, governments, and the whole society. The combination of health-care budget constraints and multifactorial barriers to successful market access (e.g. regulatory constraints, evidence-based reimbursement, user acceptability and adoption) has led the key stakeholders (e.g. R&amp;D investors, third-party payers) to start incorporating HTA into R&amp;D. Nevertheless, many questions remain unanswered. The main aim of this thesis was to understand the implication that early HTA may have on MD market access and to identify methodological gaps.<br><br>In the first chapter, we performed a systematic literature review to describe and assess techniques for conducting case studies in early economic evaluation (EEE). Out of 2,826 publications identified (time period: 2006-2017), 69 studies were included with a total of 88 case studies in MDs and/or In Vitro Diagnostics. Except for some countries (i.e. The Netherlands and UK), the results of the systematic review showed that the use of EEE is still not a common practice, especially in industry. Indeed, only 18.8% of the publications reviewed were the results of academia-industry collaborations. Our findings led us to develop a framework to assess the quality of studies in EEE and to explore which drivers might influence the choice of a specific methodology for conducting an EEE. <br><br>The second chapter further investigated the industry perspective to shed light on the scarce evidence discovered in chapter I. This chapter assessed to what extent the overall early HTA strategy (i.e. EEE combined with other perspectives, such as early usability) might influence MD success. We, therefore, developed and field-tested an evidence-based framework that explained MD success of Small and Large Enterprises (SEs and LEs). We applied a multi-method approach utilising 25 international experts and 22 European and US selected companies. Our results showed that early HTA plays a pivotal role in MD industry success and that it could have different implications based on enterprises size. On one hand, the adoption of early HTA in SEs setting could be vital for the companies’ survival. On the other hand, investing the proper amount of funds in early HTA by LEs can increase R&amp;D efficiency; however, the allocation of additional funds after a certain threshold could not guarantee MD success.<br><br>In the third chapter, we applied the results from the previous chapters to a case study. An EEE was conducted via an early cost-utility analysis to assess an innovative exoskeleton able to prevent patients’ falls. We employed a Markov model simulating an UK-based cohort of 13,906 hip fractured patients to model the impact of the exoskeleton preventing a second hip fracture on costs and Health Related Quality of Life (HRQOL). In particular, our analyses focused on patients at high-risk of hip fracture (e.g. dementia and cardiovascular disease) comparing the use of the exoskeleton to standard care (i.e. no intervention). The results suggested the importance of fall-prevention and HRQOL will vary accordingly the subpopulation assessed. HRQOL will play a considerable role in CVD patients, whilst fall-prevention will be probably more important in dementia cohort. Nevertheless, the leasing cost of the exoskeleton will drive the success probability in both dementia and CVD cohorts.