Mona Sabharwal

Informing Canada's cancer drug funding decisions with scientific evidence and patient perspectives: the Pan-Canadian Oncology Drug Review.

MS: In 2007, many of Canada’s provinces participated in the interim Joint Oncology Drug Review. The Review provided evidence-based recommendations for cancer treatments and demonstrated the value that a national collaborative platform can provide to cancer care decision-making. pcodr succeeded the interim Joint Oncology Drug Review and includes the provinces and territories (with the exception of Quebec). In addition, pcodr partners with the provincial cancer agencies, the Canadian Partnership Against Cancer, and the Canadian Agency for Drugs and Technology in Health.

Do the American Society of Clinical Oncology Value Framework and the European Society of Medical Oncology Magnitude of Clinical Benefit Scale Measure the Same Construct of Clinical Benefit

Purpose Whether the ASCO Value Framework and the European Society for Medical Oncology (ESMO) Magnitude of Clinical Benefit Scale (MCBS) measure similar constructs of clinical benefit is unclear. It is also unclear how they relate to quality-adjusted life-years (QALYs) and funding recommendations in the United Kingdom and Canada. Methods Randomized clinical trials of oncology drug approvals by the US Food and Drug Administration, European Medicines Agency, and Health Canada between 2006 and August 2015 were identified and scored using the ASCO version 1 (v1) framework, ASCO version 2 (v2) framework, and ESMO-MCBS by at least two independent reviewers. Spearman correlation coefficients were calculated to assess construct (between frameworks) and criterion validity (against QALYs from the National Institute for Health and Care Excellence [NICE] and the pan-Canadian Oncology Drug Review [pCODR]). Associations between scores and NICE/pCODR recommendations were examined. Inter-rater reliability was assessed using intraclass correlation coefficients. Results From 109 included randomized clinical trials, 108 ASCOv1, 111 ASCOv2, and 83 ESMO scores were determined. Correlation coefficients for ASCOv1 versus ESMO, ASCOv2 versus ESMO, and ASCOv1 versus ASCOv2 were 0.36 (95% CI, 0.15 to 0.54), 0.17 (95% CI, -0.06 to 0.37), and 0.50 (95% CI, 0.35 to 0.63), respectively. Compared with NICE QALYs, correlation coefficients were 0.45 (ASCOv1), 0.53 (ASCOv2), and 0.46 (ESMO); with pCODR QALYs, coefficients were 0.19 (ASCOv1), 0.20 (ASCOv2), and 0.36 (ESMO). None of the frameworks were significantly associated with NICE/pCODR recommendations. Inter-rater reliability was good for all frameworks. Conclusion The weak-to-moderate correlations of the ASCO frameworks with the ESMO-MCBS, as well as their correlations with QALYs and with NICE/pCODR funding recommendations, suggest different constructs of clinical benefit measured. Construct convergent validity with the ESMO-MCBS did not increase with the updated ASCO framework.

Does it Matter Whether Canada’s Separate Health Technology Assessment Process for Cancer Drugs has an Economic Rationale?

PharmacoEconomics DOI 10.1007/s40273-015-0278-7 LETTER TO THE EDITOR Does it Matter Whether Canada’s Separate Health Technology Assessment Process for Cancer Drugs has an Economic Rationale? Jeffrey S. Hoch 1,2,3,4 • Jaclyn Beca 1,2,3 • Mona Sabharwal 5 • Scott W. Livingstone 6 Anthony L. A. Fields 7 The Author(s) 2015. This article is published with open access at Springerlink.com ‘‘What our health-care systems need to do with their limited budgets is maximize value and no dimensions of health … will be a good measure of value because people consider other aspects in their evaluations … We must back out of the dead end that is pursuing overall efficiency’’ [1]. 1 Introduction We are grateful for the opportunity to comment on the results of McDonald et al. [2]. In this note, we summarize their findings, build on their interpretations, and suggest future directions for researchers interested in improving a health technology assessment (HTA) process. 2 Summary of the Findings McDonald et al. searched for reasons why Canada and other countries might separate their cancer drug HTA process. They appraised whether any of the rationales they & Jeffrey S. Hoch jeffrey.hoch@utoronto.ca Pharmacoeconomics Research Unit, Cancer Care Ontario, Toronto, ON, Canada Jaclyn Beca becaj@smh.ca Canadian Centre for Applied Research in Cancer Control (ARCC), Toronto, ON, Canada Mona Sabharwal monas@cadth.ca Institute for Health Policy, Management and Evaluation, University of Toronto, Toronto, ON, Canada Scott W. Livingstone scott.livingstone@saskcancer.ca Pan-Canadian Oncology Drug Review (pCODR), Canadian Agency for Drugs and Technologies in Health (CADTH), Toronto, ON, Canada Saskatchewan Cancer Agency, Saskatoon, SK, Canada University of Alberta, 11560 University Ave NW, Edmonton, AB T6G 1Z2, Canada Anthony L. A. Fields afields@ualberta.ca found were derived from the assumption commonly made by health economists that ‘‘the goal of society or decision makers is to maximize the total aggregate health benefit conferred to a population for a given level of resources’’. They found that this rationale was not used by Canada or any country to support the use of a separate HTA process for cancer drugs. The researchers were also not able to find another country besides Canada that had a separate cancer drug HTA process. These findings may seem surprising: Canada is the only country in the world that has a separate cancer drug HTA process; and McDonald et al. could find no justification based in economic theory for a separate process. To be clear, the authors reported that other countries (e.g. Den- mark, Belgium, the UK) set aside a separate budget for cancer drugs, and the authors found several ‘‘non-eco- nomic’’ rationales for Canada’s separate HTA process. There are two potential interpretations of these search re- sults: (1) the search missed something or (2) there really is no economic rationale in print for a separate cancer drug HTA process. Although their study might not be Centre for Excellence in Economic Analysis Research (CLEAR), Li Ka Shing Knowledge Institute, St. Michael’s Hospital, Toronto, ON, Canada

Multiple Dimensions of Value: Evaluative Frameworks for New Cancer Therapies

TO THE EDITOR: The increasing burden of costs associated with novel cancer therapies has become untenable. In the United States, the annual cost of a new cancer therapy routinely exceeds

Comparing assessment frameworks for cancer drugs between Canada and Europe: What can we learn from the differences?

100,000; such costs may prevent patients from receiving effective options or only enable access under risk of significant financial duress or bankruptcy. In this context, we applaud the recent development of the ASCO Value Framework, a tool that enables physicians and patients to discuss the relative value of a new cancer therapy compared with the standard of care. The framework assesses benefit and toxicity of a cancer therapy through a net health benefit score while highlighting the direct drug costs borne by patients. A core tenet in ASCO’s process was to ensure that the framework development was transparent and malleable. Some criticisms already have targeted the arbitrariness of net health benefit scoring and limited perspective on costs as well as the inability of the framework to evaluate the methodological strength of evidence, consider noncomparative studies despite the increasing propensity for regulatory drug approvals based on earlier-phase trials, and evaluate scenarios in which multiple comparators represent current standards or for which nontrial (real-world) data are available. Over time, these limitations in the initial process may be addressed or proven of minimal consequence; certainly, we now have a much-needed starting point for a dialogue with patients on relative clinical benefit and costs. We highlight that additional dimensions may also warrant consideration to create the most fulsome assessment of value. In particular, such factors as patient preferences, burden of illness, feasibility of adoption, and formal cost-effectiveness analysis (CEA) may broaden the perspective of value from the individual level to society at large. Since 2007, the provinces and territories of Canada (with the exception of Quebec) have participated in the pan-Canadian Oncology Drug Review (pCODR). pCODR provides evidencebased cancer drug funding recommendations and evolved from iterative processes for formal evaluation of cancer therapies at a national level that have spanned more than a decade. The Canadian experience suggests that although clinical and economic evidence are fundamental building blocks for evaluation, additional factors often come into play when funding decisions are implemented. As such, pCODR follows an established deliberative framework (Table 1) in making recommendations by systematically assessing clinical and economic evidence in addition to patient perspectives and the feasibility of drug adoptionwithin the provincial context and budget. The framework aspires to ensure that no single element overrides another and that no single threshold must be met for any element in the review. Can these additional elements enhance our understanding of value beyond the traditional pillars of benefit and drug costs already considered by ASCO? In its consideration of benefit, pCODR expands its scope beyond efficacy and safety to consider quality of life, burden of illness on society, and availability of

The impact of cancer drug wastage on economic evaluations

The increasing burden of costs associated with novel cancer therapies is becoming untenable. In Europe and Canada, assessment frameworks have been developed to attribute value to novel therapies and ultimately facilitate access to cancer drug funding. A review of the two frameworks has not previously been undertaken. This review provides insight into the relative strengths and benefits of each approach, the various perspectives of value (patient, physician and societal) and how the frameworks relate to their unique context and core principles. Both frameworks assess the clinical benefit of a new cancer therapy. The European framework considers effectiveness, quality of life, and toxicity in its determination of benefit and has the advantage of providing a simple summary score to facilitate priority setting. The Canadian framework considers other elements including cost-effectiveness, patient preferences and adoption feasibility; its deliberative framework precludes a simple summative presentation of value but can address complex and nuanced drug funding considerations with flexibility. Both frameworks have evolved to meet the needs unique to their jurisdictions and offer potentially complementary tools in the assessment of new cancer drugs. Lessons learnt in both systems can be applied to future iterations of the frameworks, which remain works in progress.

Engaging specialist oncologists in the assessment of oncology drugs in Canada

The objective of this study was to determine the impact of modeling cancer drug wastage in economic evaluations because wastage can result from single‐dose vials on account of body surface area– or weight‐based dosing.

Public engagement in priority-setting: Results from a pan-Canadian survey of decision-makers in cancer control

www.thelancet.com/oncology Vol 18 May 2017 573 5 Chi KN, Spratlin J, Kollmannsberger C, et al. Food effects on abiraterone pharmacokinetics in healthy subjects and patients with metastatic castration-resistant prostate cancer. J Clin Pharmacol 2015; 55: 1406–14. 6 Inoue K, Shishido A, Vaccaro N, et al. Pharmacokinetics of abiraterone in healthy Japanese men: dose-proportionality and effect of food timing. Cancer Chemother Pharmacol 2015; 75: 49–58. 7 US Food and Drug Administration. Zytiga (abiraterone acetate) product label. http://www.accessdata.fda.gov/drugsatfda_docs/ label/2011/202379lbl.pdf (accessed Sep 20, 2016). 8 US Food and Drug Administration. Tykerb (lapatinib) product label. http://www.accessdata.fda.gov/drugsatfda_docs/label/2007/022059lbl. pdf (accessed 15 Feb 2017).