Getting the Right Evidence After Drug Approval

Vreman, Rick A; Leufkens, Hubert G. M.; Kesselheim, Aaron S.

doi:10.3389/fphar.2020.569535

Cited by 10 publications

(10 citation statements)

References 17 publications

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“…These initiatives could first include methodological work on RWD quality assessment, alternative trial designs or combining RCT with RWD sources as well as implementing methods into the decision making process ( Selker et al, 2014 ; Makady et al, 2017c ; Efthimiou et al, 2017 ; Selker et al, 2019 ; Chalkou et al, 2021 ). Second, the focus could be on consensus building on when and how to use RWD (methods) ( Berger et al, 2017 ; Vreman et al, 2020b ; Facey et al, 2020 ; Orsini et al, 2020 ). A third suggestion could be to invest more in generating and aligning the required data, such as patient registries, at an early stage, as is aimed by the European IMI EHDEN project as well as by national initiatives across the continent ( Ministerie van VolksgezondheidWen, 2019 ; IMI Innovative Medicines Initiative EHDEN, 2021 ; The National Patient Register, 2021 ).…”

Section: Discussionmentioning

confidence: 99%

Real World Data in Health Technology Assessment of Complex Health Technologies

et al. 2022

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The available evidence on relative effectiveness and risks of new health technologies is often limited at the time of health technology assessment (HTA). Additionally, a wide variety in real-world data (RWD) policies exist among HTA organizations. This study assessed which challenges, related to the increasingly complex nature of new health technologies, make the acceptance of RWD most likely. A questionnaire was disseminated among 33 EUnetHTA member HTA organizations. The questions focused on accepted data sources, circumstances that allowed for RWD acceptance and barriers to acceptance. The questionnaire was validated and tested for reliability by an expert panel, and pilot-tested before dissemination via LimeSurvey. Twenty-two HTA organizations completed the questionnaire (67%). All reported accepting randomized clinical trials. The most accepted RWD source were patient registries (19/22, 86%), the least accepted were editorials and expert opinions (8/22, 36%). With orphan treatments or companion diagnostics, organizations tended to be most likely to accept RWD sources, 4.3–3.2 on a 5-point Likert scale, respectively. Additional circumstances were reported to accept RWD (e.g., a high disease burden). The two most important barriers to accepting RWD were lacking necessary RWD sources and existing policy structures. European HTA organizations seem positive toward the (wider) use of RWD in HTA of complex therapies. Expanding the use of patient registries could be potentially useful, as a large share of the organizations already accepts this source. However, many barriers still exist to the widespread use of RWD. Our results can be used to prioritize circumstances in which RWD might be accepted.

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Section: Discussionmentioning

confidence: 99%

Real World Data in Health Technology Assessment of Complex Health Technologies

et al. 2022

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“…These costs can become so high that they outweigh any savings made by such a scheme [ 3 , 5 , 28 , 49 , 54 , 59 , 64 ]. The enforcement of additional studies to resolve the uncertainties regarding long-term effects could sometimes also be achieved by implementing a form of coverage with evidence development where it is agreed that the price will drop iteratively by a certain percentage or amount after predefined periods when the manufacturer does not produce the necessary evidence [ 89 ]. Such a reimbursement mechanism mitigates financial risks and enforces additional evidence generation while simultaneously preventing overly complicated outcome-based schemes.…”

Section: Discussionmentioning

confidence: 99%

Application of Managed Entry Agreements for Innovative Therapies in Different Settings and Combinations: A Feasibility Analysis

Vreman

Broekhoff

Leufkens

et al. 2020

IJERPH

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The reimbursement of expensive, innovative therapies poses a challenge to healthcare systems. This study investigated the feasibility of managed entry agreements (MEAs) for innovative therapies in different settings and combinations. First, a systematic literature review included studies describing used or conceptual agreements between payers and manufacturers (i.e., MEAs). Identical and similar MEAs were clustered and data were extracted on their benefits and limitations. A feasibility assessment was performed for each individual MEA based on how it could be applied (financial/outcome-based), on what level (individual patients/target population), in which payment setting (centralized pricing and reimbursement authority yes/no), for what type of therapies (one-time/chronic), within what payment structures, and whether combinations with other MEAs were feasible. The literature search ultimately included 82 papers describing 117 MEAs. After clustering, 15 unique MEAs remained, each describing one or multiple similar agreements. Four of those entailed payment structures, while eleven entailed agreements between payers and manufacturers regarding price, usage, and/or evidence generation. The feasibility assessment indicated that most agreements could be applied throughout the different settings that were assessed and could be applied in different payment structures and in combination with multiple other agreements. The potential to combine multiple agreements leads to a multitude of different reimbursement mechanisms that may manage the price, usage, payment structure, and additional conditions for an innovative therapy. This overview of the feasibility of combinations of MEAs can help decision-makers construct a reimbursement mechanism most suited to their preferences, the type of therapy under evaluation, and the applicable healthcare system.

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“…Expedited drug approval pathways require post-approval studies to address evidence gaps. These are often delayed, and if performed, tend to have the same inappropriate characteristics as the pre-approval trials [ 6 , 43 , 44 , 99 – 101 ]. A recent overview of 25 years of accelerated approvals of oncology products (all based on surrogate endpoints) showed 51 of 93 initial approvals to confirm benefit in the ensuing years.…”

Section: Regulatory Agenciesmentioning

confidence: 99%

An urgent call to raise the bar in oncology

et al. 2021

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Important breakthroughs in medical treatments have improved outcomes for patients suffering from several types of cancer. However, many oncological treatments approved by regulatory agencies are of low value and do not contribute significantly to cancer mortality reduction, but lead to unrealistic patient expectations and push even affluent societies to unsustainable health care costs. Several factors that contribute to approvals of low-value oncology treatments are addressed, including issues with clinical trials, bias in reporting, regulatory agency shortcomings and drug pricing. With the COVID-19 pandemic enforcing the elimination of low-value interventions in all fields of medicine, efforts should urgently be made by all involved in cancer care to select only high-value and sustainable interventions. Transformation of medical education, improvement in clinical trial design, quality, conduct and reporting, strict adherence to scientific norms by regulatory agencies and use of value-based scales can all contribute to raising the bar for oncology drug approvals and influence drug pricing and availability.

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Getting the Right Evidence After Drug Approval

Abstract: To generate comparative evidence in a timely fashion for drugs without restricting or delaying access, value-based pricing and reimbursement could be conditioned on a prospective, post-approval evidence generation plan.

Cited by 10 publications

References 17 publications

Real World Data in Health Technology Assessment of Complex Health Technologies

Real World Data in Health Technology Assessment of Complex Health Technologies

Application of Managed Entry Agreements for Innovative Therapies in Different Settings and Combinations: A Feasibility Analysis

An urgent call to raise the bar in oncology

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