Health Technology Assessment Challenges in Oncology: 20 Years of Value in Health

Abstract

Background: Oncology treatments have changed from chemotherapies to targeted therapies and more recently immunooncology. This has posed special challenges in the field of health technology assessment (HTA): capturing quality of life (QOL) associated with toxicity due to chemotherapy, crossover upon progression in targeted therapy trials, and survival extrapolation for immuno-oncology drugs.Objectives: To showcase 20 years of Value in Health (ViH) publications in oncology.Methods: A review was undertaken of oncology articles published in ViH from May 1998 to August 2018. Full-length articles published in ViH with the keywords "oncology," "cancer," "h(a)ematology," and "malignancy" were included for review. Conference abstracts were excluded.Results: Four major themes were identified: (1) QOL and the development of multiple functional assessment of cancer therapy tools and mapping instruments; (2) analysis of clinical evidence using indirect comparisons, network analyses, and adjustment for crossovers; (3) modeling, Markov models, partitioned survival models, and extrapolation methods; and (4) financial implications and how to deal with uncertainty, introduction of conditional reimbursement, managed entry, and risk share agreements.Discussion: This review article highlights the important role ViH has played in disseminating HTA research in oncology. A few key issues loom on the horizon: precision medicine, further development and practical application of new QOL measures, methods for translating clinical evidence, and exploration of modeling techniques. For a better understanding of the complex interplay between access and financial risk management, ViH will no doubt continue to promote pioneering research in HTA and oncology.

Barriers and Opportunities for Implementation of Outcome-Based Spread Payments for High-Cost, One-Shot Curative Therapies

“…Maes et al propose two possible solutions to comply with the ESA: the payer pays for the service of the treatment delivering long-term health outcomes or the payer pays for data services as a delivered data package per year instead of paying for a single treatment administration (Maes et al, 2019). Furthermore, healthcare systems wishing to implement spread payments may face national legal barriers which may require adjustment of country-specific regulations (Carlson et al, 2009;Carlson et al, 2011;Espin et al, 2011;Ferrario and Kanavos, 2013;Tuna et al, 2014;Kleinke and McGee, 2015;Barlas, 2016b;Proach et al, 2016;Montazerhodjat et al, 2016;Kanavos et al, 2017;Nazareth et al, 2017;NEHI, 2017;PWC Health Research Institute, 2017;Spark et al, 2017;Goncalves et al, 2018;Salzman et al, 2018;Infante et al, 2019;Lorente et al, 2019;Mahendraratnam et al, 2019). For instance, the current legislation in Spain only allows long-term spending for certain investments which excludes medicines and Sweden does not allow payments for more than three years for non-investment consumables (that are not purchased to deliver financial return) (Alliance for Regenerative Medicine, 2019).…”

“…These conditions could either be a pre-agreed adjustment of the payment in expectation of competition, as proposed by Towse et al investigating the implications of paying for one-shot cures vs. repeat administrations. Contrarily, Schaffer et al propose the addition of re-opener clauses that foresee provisions for payment adjustment whereas an evaluation of Belgian MEAs suggested the complete termination of the agreement when exclusivity rights expire or a competitive product enters the market (Kanavos et al, 2017;Nazareth et al, 2017;AMCP, 2019;Infante et al, 2019).…”

Cost‐effectiveness and financial risks associated with immune checkpoint inhibitor therapy

“…Choice of extrapolation of survival data is a common source of uncertainty in cost‐effectiveness assessments of ICI drugs 86 . For example, in guidance document TA519 when considering pembrolizumab, NICE found that using the Gompertz curve for extrapolation assumed that no patient could reside in the postprogressive health state after year 6 17 .…”

The Potential for Early Health Economic Modelling in Health Technology Assessment and Reimbursement DecisionMaking Comment on "Problems and Promises of Health Technologies: The Role of Early Health Economic Modeling"

“…21 Moreover, there seems to be a tendency for payers to be to prepared to enter into 'coverage with evidence' development schemes or risk share agreements. 4,22 This is typically done in order to acquire further data to support the evidence for the incremental costeffectiveness ratio. Unfortunately, while these schemes and agreements offer a solution to early funding as reimbursement is granted even though evidence such as mature survival data is not yet available, the risks are often not well-understood.…”

“…For example, the advent of immuno-oncology agents, which have unique biological mechanisms of action and clinical effects, has necessitated new approaches to economic evaluation, especially with regards to extrapolating and translating data beyond the pivotal clinical trials. 4 In countries without mandatory health economic modelling, like South Korea and Taiwan, the utility of early modelling is less clear. However, this strategy can still be used to characterise both the clinical and economic environments within which the new health technology will be assessed for reimbursement.…”