Payer and Implementation Challenges with Advanced Therapy Medicinal Products (ATMPs)

Spoors, John; Miners, Alec; Cairns, John; Palnoch, Danny; Summerfield, Ash; McEntee, Joanne; Vithlani, Sheena

doi:10.1007/s40259-020-00457-4

Cited by 7 publications

(6 citation statements)

References 5 publications

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“…Although ATMPs offer potentially important benefits for patients, their families and society overall, their high upfront and often one-off costs can pose challenges related to affordability and implementation in the NHS [ 39 ]. Many of the mitigation measures are commercial in nature (e.g., patient access schemes) and not directly in the remit of NICE technology evaluations.…”

Section: Discussionmentioning

confidence: 99%

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Evaluation of Advanced Therapy Medicinal Products by the National Institute for Health and Care Excellence (NICE): An Updated Review

Pinho-Gomes

Cairns

2021

PharmacoEconomics Open

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This review discusses the methodological challenges to the evaluation of advanced therapy medicinal products (ATMPs) by the UK National Institute for Health and Care Excellence (NICE). We analysed the health technology appraisals (both published and in development) of ATMPs conducted by NICE in England until July 2021. A total of 14 health technology appraisals of ATMPs were included, of which two were in development and 12 had been published. There were ten gene therapy products (talimogene laherparepvec [TA410], strimvelis [HST7], tisagenlecleucel [TA554 and TA567], axicabtagene ciloleucel [TA559], voretigene neparvovec [HST11], autologous anti-CD19-transduced CD3+ cells [TA677], betibeglogene autotemcel [ID968], onasemnogene abeparvovec [HST15] and OTL-200 [ID1666]), one tissue engineered product (holoclar [TA467]) and three somatic cell therapy products (darvadstrocel [TA556] and autologous chondrocyte implantation [ACI][TA477 and TA508]). Only three of these technologies were not recommended by NICE, although four were only recommended within the Cancer Drugs Fund. There was large uncertainty in the assessment of clinical effectiveness, as evidence relied mostly on small, single-arm, open-label studies. There were also several concerns in the cost-effectiveness evaluations, such as limited information on health-related quality of life, immature survival data due to short follow-up, and unclear curative potential. Substantial risk may be incurred with ATMPs, which have high upfront and possibly irrecoverable costs but uncertain long-term benefits. In conclusion, the challenges raised by the economic appraisal of ATMPs, albeit not unique, may be exacerbated by the uncertainty related to the often scant evidence base. Adaptations of the conventional decisionmaking process rather than completely new methods may improve appraisals of ATMPs.

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Section: Discussionmentioning

confidence: 99%

“…Second, ATMPs are associated with a larger financial risk for the NHS than many other technologies [ 39 ]. They often have high upfront and, depending on contractual arrangements, potentially irrecoverable costs, yet the full benefits may take many years to accrue or may not be permanent.…”

Section: Discussionmentioning

confidence: 99%

Evaluation of Advanced Therapy Medicinal Products by the National Institute for Health and Care Excellence (NICE): An Updated Review

Pinho-Gomes

Cairns

2021

PharmacoEconomics Open

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“…DAF considerations : With higher payer willingness to pay thresholds comes the possibility that developers will be motivated to inflate the price of GTs to meet the ICER threshold ( 52 , 53 ), in other words, the developers will charge the price as high as the payer are able to endure ( 26 ). It is unlikely that payers will accept innovative value-pricing methods, considering that they must be implemented with a radical modification of current policy.…”

Section: Economic Analysismentioning

confidence: 99%

Gene Therapy Evidence Generation and Economic Analysis: Pragmatic Considerations to Facilitate Fit-for-Purpose Health Technology Assessment

Qiu

Pochopien²,

Liang

et al. 2022

Front. Public Health

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Gene therapies (GTs) are considered to be a paradigm-shifting class of treatments with the potential to treat previously incurable diseases or those with significant unmet treatment needs. However, considerable challenges remain in their health technology assessment (HTA), mainly stemming from the inability to perform robust clinical trials to convince decision-makers to pay the high prices for the potential long-term treatment benefits provided. This article aims to review the recommendations that have been published for evidence generation and economic analysis for GTs against the feasibility of their implementation within current HTA decision analysis frameworks. After reviewing the systematically identified literature, we found that questions remain on the appropriateness of GT evidence generation, considering that additional, broader values brought by GTs seem insufficiently incorporated within proposed analytic methods. In cases where innovative methods are proposed, HTA organizations remain highly conservative and resistant to change their reference case and decision analysis framework. Such resistances are largely attributed to the substantial evidence uncertainty, resource-consuming administration process, and the absence of consensus on the optimized methodology to balance all the advantages and potential pitfalls of GTs.

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“…Moreover, despite no confirmatory evidence is available to demonstrate the “curative” benefits of ATMPs, manufacturers charged high prices with intentions to rapidly recoup the development investment ( 11 ). This made payers raise skepticism about the justification of the high prices of ATMPs in relation to the untransparent disclosure of manufacturing cost and often, paucity of clinical evidence ( 12 ).…”

Section: Introductionmentioning

confidence: 99%

Reinforcing Collaboration and Harmonization to Unlock the Potentials of Advanced Therapy Medical Products: Future Efforts Are Awaited From Manufacturers and Decision-Makers

Qiu

Liang

Wang

et al. 2021

Front. Public Health

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Some advanced therapy medicinal products (ATMPs) hold great promises for life-threatening diseases with high unmet needs. However, ATMPs are also associated with significant challenges in market access, which necessitates the joint efforts between all relevant stakeholders to navigate. In this review, we will elaborate on the importance of collaborations and harmonization across different stakeholders, to expedite the market access of promising ATMPs. Manufacturers of ATMPs should proactively establish collaborations with other stakeholders throughout the whole lifecycle of ATMPs, from early research to post-market activities. This covered engagements with (1) external developers (i.e., not-for-profit organizations and commercial players) to obtain complementary knowledge, technology, or infrastructures, (2) patient groups and healthcare providers to highlight their roles as active contributors, and (3) decision-makers, such as regulators, health technology assessment (HTA) agencies, and payers, to communicate the uncertainties in evidence package, where parallel consultation will be a powerful strategy. Harmonization between decision-makers is desired at (1) regulatory level, in terms of strengthening the international standardization of regulatory framework to minimize discrepancies in evidence requirements for market authorization, and (2) HTA level, in terms of enhancing alignments between regional and national HTA agencies to narrow inequity in patient access, and cross-border HTA cooperation to improve the quality and efficiency of HTA process. In conclusion, manufacturers and decision-makers shared the common goals to safeguard timely patient access to ATMPs. Collaboration and harmonization will be increasingly leveraged to enable the value delivery of ATMPs to all stakeholders.

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Payer and Implementation Challenges with Advanced Therapy Medicinal Products (ATMPs)

Cited by 7 publications

References 5 publications

Evaluation of Advanced Therapy Medicinal Products by the National Institute for Health and Care Excellence (NICE): An Updated Review

Evaluation of Advanced Therapy Medicinal Products by the National Institute for Health and Care Excellence (NICE): An Updated Review

Gene Therapy Evidence Generation and Economic Analysis: Pragmatic Considerations to Facilitate Fit-for-Purpose Health Technology Assessment

Reinforcing Collaboration and Harmonization to Unlock the Potentials of Advanced Therapy Medical Products: Future Efforts Are Awaited From Manufacturers and Decision-Makers

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