Rapid access to innovative medicinal products while ensuring relevant health technology assessment. Position of the French National Authority for Health

Vanier, Antoine; Fernandez, Judith; Kelley, Sophie; Alter, Lise; Semenzato, Patrick; Alberti, Corinne; Chevret, Sylvie; Costagliola, Dominique; Cucherat, Michel; Falissard, Bruno; Gueyffier, François; Lambert, Jérôme; Lengliné, Étienne; Locher, Clara; Naudet, Florian; Porcher, Raphaël; Thiébaut, Rodolphe; Vray, Muriel; Zohar, Sarah; Cochat, Pierre; Guludec, Dominique Le

doi:10.1136/bmjebm-2022-112091

Cited by 25 publications

(9 citation statements)

References 22 publications

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“…However, the use of single-arm basket trials to support this recommendation in such a rare indication 7 , 8 , 9 presents a challenge when requesting reimbursement, as randomized controlled trials (RCTs) are considered to be the gold standard by health technology assessment bodies. 10 , 11 , 12 …”

Section: Introductionmentioning

confidence: 99%

Therapeutic benefit of larotrectinib over the historical standard of care in patients with locally advanced or metastatic infantile fibrosarcoma (EPI VITRAKVI study)

Orbach,

Carton,

Khadir

et al. 2024

ESMO Open

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Section: Introductionmentioning

confidence: 99%

Therapeutic benefit of larotrectinib over the historical standard of care in patients with locally advanced or metastatic infantile fibrosarcoma (EPI VITRAKVI study)

Orbach,

Carton,

Khadir

et al. 2024

ESMO Open

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“…At the time of initial assessment and appraisal, which typically follows the marketing authorization, pivotal clinical trials are the primary source of submitted data. Randomized controlled trials (RCTs) remain the gold standard for establishing efficacy (5); however, RCTs require strict implementation conditions and an optimal management setting that deviate from routine care (6;7). To mitigate potential biases that could compromise internal validity and to accommodate pragmatic and ethical constraints, RCTs are conducted in selected and homogeneous populations that only partially mirror the characteristics of patients treated in routine care.…”

Section: Introductionmentioning

confidence: 99%

Can requests for real-world evidence by the French HTA body be planned? An exhaustive retrospective case–control study of medicinal products appraisals from 2016 to 2021

Fernandez,

Babin,

Thomassin

et al. 2024

Int J Technol Assess Health Care

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Objectives: In France, decisions for pricing and reimbursement for medicinal products are based on appraisals performed by the National authority for health (Haute Autorité de Santé (HAS)). During the appraisal process, additional real-world evidence can be requested as "Post-Registration Studies" (PRS) when there are uncertainties in evidence that could be resolved by additional data collection. To facilitate PRS planning, a retrospective exploratory analysis was conducted to identify the characteristics of medicinal products associated with a PRS request. Methods: This analysis encompassed all appraisals finalized between January 1, 2016 and December 31, 2021 and compared products for which the appraisal led to a PRS request with those that did not. Results: Six hundred positive opinions for reimbursement were identified, with a PRS request present in 17 percent (n = 103) of cases. The independent characteristics associated with a PRS request were a mild or moderate clinical benefit score, a major to moderate or minor clinical added value score, previous availability under an early access program, and certain therapeutic areas (neurology, pulmonology, and endocrinology). These findings suggest two different profiles of PRS requests: (i) products for which there is uncertainty in the size of the clinical benefit and (ii) innovative products for which a substantial benefit is expected but uncertainties persist. Conclusions: These results will assist health technology developers to better anticipate data generation to promptly address uncertainties identified by HAS. It may also help HAS and other assessment agencies to work together to improve postlaunch evidence generation according to the characteristics of the medicinal products.

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“…Although there are several approaches that can be used to mitigate the impact of bias on a nonrandomized comparison, such as target trial emulation, matching adjusted indirect comparison, simulated treatment comparison, and propensity score methods such as the inverse probability of treatment weighting, the use of an EC and correspondingly the nonrandomized treatment effect often still results in a substantial risk of residual bias and treatment effects from NRS have received criticism due to such biases (6)(7)(8)(9)(10). This poses significant challenges for decision-makers in interpreting the results of such comparisons.…”

Section: Introductionmentioning

confidence: 99%

The role of quantitative bias analysis for nonrandomized comparisons in health technology assessment: recommendations from an expert workshop

Leahy,

Durand-Zaleski,

Sampietro-Colom

et al. 2023

Int J Technol Assess Health Care

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The use of treatment effects derived from nonrandomized studies (NRS) in health technology assessment (HTA) is growing. NRS carry an inherently greater risk of bias than randomized controlled trials (RCTs). Although bias can be mitigated to some extent through appropriate approaches to study design and analysis, concerns around data availability and quality and the absence of randomization mean residual biases typically render the interpretation of NRS challenging. Quantitative bias analysis (QBA) methods are a range of methods that use additional, typically external, data to understand the potential impact that unmeasured confounding and other biases including selection bias and time biases can have on the results (i.e., treatment effects) from an NRS. QBA has the potential to support HTA bodies in using NRS to support decision-making by quantifying the magnitude, direction, and uncertainty of biases. However, there are a number of key aspects of the use of QBA in HTA which have received limited discussion. This paper presents recommendations for the use of QBA in HTA developed using a multi-stakeholder workshop of experts in HTA with a focus on QBA for unmeasured confounding.

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Rapid access to innovative medicinal products while ensuring relevant health technology assessment. Position of the French National Authority for Health

Abstract: Accelerating rapid access to innovative treatments by adapting the traditional RCT designWhile RCTs are the gold standard for producing clinical evidence, HAS acknowledges that the strict

Cited by 25 publications

References 22 publications

Therapeutic benefit of larotrectinib over the historical standard of care in patients with locally advanced or metastatic infantile fibrosarcoma (EPI VITRAKVI study)

Therapeutic benefit of larotrectinib over the historical standard of care in patients with locally advanced or metastatic infantile fibrosarcoma (EPI VITRAKVI study)

Can requests for real-world evidence by the French HTA body be planned? An exhaustive retrospective case–control study of medicinal products appraisals from 2016 to 2021

The role of quantitative bias analysis for nonrandomized comparisons in health technology assessment: recommendations from an expert workshop

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