Beyond the Bench to Bedside: How Health Technology Assessment Impacts Canadian Patients

TMED Blog

| 31 January 2022

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Alyssa Burrows, MSc Candidate (Translational Medicine)

In 2019, 32% of Ontario’s spending went towards healthcare [1]. Given the finite healthcare budget, it is crucial that the therapeutics, tests, and procedures paid for by the provincial governments are cost-effective and beneficial to as many patients as possible. Dr. Nicole Mittmann M.Sc., Ph.D., the Chief Scientist and Vice-President of the Canadian Agency for Drugs and Technologies in Health (CADTH), presented on the role of CADTH and health technology assessment (HTA) in translational medicine. Understanding how a therapy moves from a limited clinical trial patient population to the broader patient population and who will pay for it is an under-discussed but integral aspect of translational medicine [2]. CADTH plays a vital role in this process, undertaking HTAs that provide crucial evidence-based recommendations to healthcare payers, which can help make therapeutics widely available to patients [3].

Dr. Mittmann explained that the current internationally accepted definition of HTA is “…a multidisciplinary process that uses explicit methods to determine the value of a health technology at different points in its lifecycle. The purpose is to inform decision-making in order to promote an equitable, efficient, and high-quality health system [4].” CADTH is an independent, not-for-profit organization that provides Canadian healthcare decision-makers with objective evidence to inform their decisions [5]. When undertaking a HTA, CADTH engages in a deliberative process that has three core principles: transparency, impartiality, and inclusivity [6]. This deliberative process follows the Input, Throughput, and Output (ITO) model [6]. During the Input phase, CADTH collects evidence, information, and perspectives from clinicians, patients, and other stakeholders [6, 7]. Dr. Mittmann stated that she constantly thinks about how to actively engage stakeholders purposefully and equitably which are two of the several guiding principles considered when involving patients and the public in HTAs [8, 9]. Critically evaluating and appraising information occurs during the Throughput stage, this is where the hierarchy of evidence is considered, and real-world evidence (RWE) can be integrated as necessary [6, 10, 11]. Communicating a decision to the public and collecting reflective feedback from stakeholders is part of the Output phase [6]. CADTH can recommend three possible outcomes of a HTA: list, conditional list, and refusal to list [12]. To ensure CADTH’s recommendations are transparent they are made publicly available which is one of the three core principles of the deliberative process that Dr. Mittmann highlighted.

Dr. Mittmann shared that in 2021, the Federal Drug Administration and Health Canada (HC) approved 50 and 43 new molecular entities, respectively, many of which are first-in-class therapeutics capable of meeting the unmet needs of patients [13, 14]. During her presentation Dr. Mittmann elucidated four key groups involved in the decision-making process to allow therapies to become available to patients. HC is a regulatory body that reviews the evidence submitted by the manufacturer and approves drugs that are safe and effective [15]. CADTH completes HTAs and makes a recommendation to payers, the Patented Medicine Prices Review Board regulates the prices of patented medications and compares prices internationally, and lastly, the pan-Canadian Pharmaceutical Alliance negotiates drug prices for the province’s and territory’s public plans ensuring affordability and accessibility [16–18]. Given the evolving nature of health technologies, these groups must continuously adapt to ensure that Canadian patients and clinicians have access to safe, effective, and valuable therapeutics, ultimately transforming healthcare.

Dr. Mittmann discussed that the current HTA process in Canada is not without criticism. Bayoui & Laupacis state that a current challenge is that funding recommendations are not updated as new evidence, including RWE, becomes available. Very few opportunities exist to change the decision once it has been made [19]. The authors recommend that a more active approach in reviewing evidence is taken throughout the product’s lifecycle, such as following the Guidelines from the National Institute for Health and Care Excellence, which suggest a standard check for new data every 5 years and allowing other stakeholders such as clinical societies and patient advocacy groups to initiate HTA reviews [19, 20]. Although these proposed solutions are resource-intensive, it would allow for CADTH to change their recommendations as new information becomes available. This change would potentially provide more patients with access to life-changing therapies, as the provinces and territories (e.g. payers) generally make funding decisions concordant to CADTH’s recommendations.

After Dr. Mittann’s invigorating TIME lectures, the Translational Medicine (TMED) graduate students were fortunate to continue the discussion with her. Throughout, Dr. Mittmann highlighted that CADTH always anticipates novel technologies, identifies unmet patient needs, and continuously adapts to integrate various forms of evidence and stakeholder perspectives into their HTA recommendations. Regarding Equity, Diversity, Inclusion and Indigeneity, CADTH is making strong efforts to engage Indigenous leaders through their Indigenous working groups, identify conscious and unconscious bias in the available evidence, and integrate in RWE.

Dr. Mittmann kindly shared her academic and curiosity driven professional journey with us, which equipped her with the skills and experiences to lead her current team at CADTH. Dr. Mittmann strongly recommends that students consider volunteering on committees such as their local Research Ethic’s Boards or Hospital Formularies to further develop their collaboration, communication, and interdisciplinary healthcare knowledge. On behalf of the TMED graduate students, I would like to thank Dr. Mittmann for her time and for providing a unique perspective to the field of translational medicine.

Acronyms

CADTH	Canadian Agency for Drugs and Technologies in Health
EDII	Equity, Diversity, Inclusion and Indigeneity
HC	Health Canada
HTA	Health Technology Assessment
ITO	Input, Throughput, and Output
RWE	Real-World Evidence
TIME	Translational Institute of Medicine
TMED	Translational Medicine

References

1. Government Of Canada SC (2020) Share of consolidated provincial, territorial and local government expenses by function, 2019. https://www150.statcan.gc.ca/n1/daily-quotidien/201127/cg-a002-eng.htm. Accessed 27 Jan 2022

2. Rogowski WH, Hartz SC, John JH (2008) Clearing up the hazy road from bench to bedside: A framework for integrating the fourth hurdle into translational medicine. BMC Health Serv Res 8:1–12

3. Lindenberg M (2020) Health technology assessments alongside the translational pathway of innovations in oncology. https://doi.org/10.3990/1.9789036550680

4. O’Rourke B, Oortwijn W, Schuller T, the International Joint Task Group (2020) The new definition of health technology assessment: A milestone in international collaboration. Int J Technol Assess Health Care 36:187–190

5. Who We Are | CADTH. https://www.cadth.ca/who-we-are. Accessed 27 Jan 2022

6. Bond K, Stiffell R, Ollendorf DA (2020) Principles for deliberative processes in health technology assessment. Int J Technol Assess Health Care 36:445–452

7. Jansen MPM, Baltussen R, Bærøe K (2018) Stakeholder Participation for Legitimate Priority Setting: A Checklist. Int J Health Policy Manag 7:973–976

8. Street J, Stafinski T, Lopes E, Menon D (2020) Defining the role of the public in Health Technology Assessment (HTA) and HTA-informed decision-making processes. Int J Technol Assess Health Care 36:87–95

9. Abelson J, Wagner F, DeJean D, et al (2016) PUBLIC AND PATIENT INVOLVEMENT IN HEALTH TECHNOLOGY ASSESSMENT: A FRAMEWORK FOR ACTION. Int J Technol Assess Health Care 32:256–264

10. Murad MH, Asi N, Alsawas M, Alahdab F (2016) New evidence pyramid. BMJ Evidence-Based Medicine 21:125–127

11. Tadrous M, Ahuja T, Ghosh B, Kropp R (2020) Developing a Canadian Real-World Evidence Action Plan across the Drug Life Cycle. Healthc Policy 15:42–47

12. Richardson M, Mittmann N, Kaunelis D (2021) Deliberative Appraisal Processes in Health Technology Assessment. Canadian Journal of Health Technologies. https://doi.org/10.51731/cjht.2021.64

13. Research C for DE and (2022) Novel Drug Approvals for 2021. FDA

14. Government of Canada HC (2014) Search Page - Drug and Health Product Register. https://hpr-rps.hres.ca/reg-content/regulatory-decision-summary-result…. Accessed 27 Jan 2022

15. Canada H (2016) Health Canada’s mandate. https://www.canada.ca/en/health-canada/corporate/mandate.html. Accessed 27 Jan 2022

16. Vision, Mission, and Values | CADTH. https://www.cadth.ca/vision-mission-and-values. Accessed 27 Jan 2022

17. Home Page | pCPA. https://www.pcpacanada.ca/node/30. Accessed 27 Jan 2022

18. Canada PMPRB (2020) Patented Medicine Prices Review Board. https://www.canada.ca/en/patented-medicine-prices-review.html. Accessed 27 Jan 2022

19. Bayoumi A, Laupacis A (2021) Outdated criteria for drug plan reimbursement obstruct evidence-based care. CMAJ 193:E1573–E1574

20. 13 Ensuring that published guidelines are current and accurate | Developing NICE guidelines: the manual | Guidance | NICE. https://www.nice.org.uk/process/pmg20/chapter/ensuring-that-published-g…. Accessed 27 Jan 2022

Beyond the Bench to Bedside: How Health Technology Assessment Impacts Canadian Patients

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Beyond the Bench to Bedside: How Health Technology Assessment Impacts Canadian Patients