Unintended Consequences of Coverage Laws Targeting Cancer Drugs

Salcher‐Konrad, Maximilian; Naci, Huseyin

doi:10.1177/1073110520958880

Cited by 4 publications

(6 citation statements)

References 15 publications

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“…Under the proposed approach to impose terms and conditions on drug approvals more broadly, it can be expected that post market confirmatory trials will continue to be relied upon to address uncertainties that exist at the time of approval or that arise post-market. The examples discussed above confirm concerns expressed previously in other jurisdictions that post market confirmatory trials should not be relied upon to address uncertainties under existing regulatory structures (66).…”

Section: Discussionsupporting

confidence: 72%

“…Similar findings have been found for drug approvals more generally; one study found that that the proportion of approvals supported by only single-group pivotal trials has increased from 1995 to 2017 (74). Furthermore, several studies have found that approving drugs with limited clinical evidence, once intended to be the exception has instead become the new norm (66,75,76). Notwithstanding a literal reading of the new regulatory approach, it seems unlikely that Canada will stop exercising flexibility to approve drugs with promising evidence and start requiring strict adherence to regulatory requirements requiring substantial evidence of clinical effectiveness.…”

Section: How Can the Current Noc/c Pathway Inform The Proposed Agile ...supporting

confidence: 66%

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Conditional Drug Approval as a Path to Market for Oncology Drugs in Canada: Challenges and Recommendations for Assessing Eligibility and Regulatory Responsiveness

2022

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International drug regulators use conditional drug approval mechanisms to facilitate faster patient access to drugs based on a lower evidentiary standard typically required of drug approvals. Faster and earlier access is justified by limiting eligibility to drugs intended for serious and life-threatening diseases and by requiring post-market evidence collection to confirm clinical benefit. One such mechanism in Canada, the Notice of Compliance with Conditions (NOC/c) policy, was introduced in 1998. Today, most of the drugs approved under the NOC/c policy are for oncology indications. We analyze oncology drugs approvals under the NOC/c policy to inform discussions of two tradeoffs applied to conditional drug approvals, eligibility criteria and post-market evidence. Our analysis informs recommendations for Canada's proposed regulatory reforms approach to conditional approvals pathways. Our analysis demonstrates that under the current policy, eligibility criteria are insufficiently defined, resulting in their inconsistent application by Health Canada. Regulatory responsiveness to post-market evidence from post-market clinical trial and foreign jurisdiction regulatory decisions is slow and insufficient. In the absence of sufficient regulatory responsiveness, physicians and patients must make clinical decisions without the benefit of the best available evidence. Together, our analysis of the two core tradeoffs in Canada's conditional drug approval provides insight to inform the further development of Canada's proposed agile regulatory approach to drugs and devices that will expand the use of terms and conditions.

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Section: Discussionsupporting

confidence: 72%

Section: How Can the Current Noc/c Pathway Inform The Proposed Agile ...supporting

confidence: 66%

Conditional Drug Approval as a Path to Market for Oncology Drugs in Canada: Challenges and Recommendations for Assessing Eligibility and Regulatory Responsiveness

2022

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“…21 That the public and other regulators are privy only to the end result without visibility or knowledge about the deliberations regarding the benefit-risk evaluation that leads up to the final result is unsatisfactory from a transparency and confidence perspective. This is particularly important for products for which decision making is a challenge, 22 as in this study in which alectinib hydrochloride was approved as conditional by the EMA but given accelerated approval by the FDA and cangrelor initially received a complete response letter from the FDA. Transparency on the factors behind the decision may become a challenge in cases in which the application of formal weightings to the various components of the benefit-risk matrix is not clearly documented.…”

Section: Discussionmentioning

confidence: 99%

“…These factors are especially important for innovative or technologically challenging products for which experience in evaluating similar products worldwide is limited. 22 This transparency is becoming increasingly important as agencies are striving to build effective and efficient regulatory processes by leveraging assessments by trusted reference agencies through approaches such as work sharing and reliance pathways. Transparent communication and documentation of the decisions in the PAR are central and should continue to evolve as a best practice that should be adopted by agencies worldwide.…”

mentioning

confidence: 99%

Transparency in European Medicines Agency and US Food and Drug Administration Decision Making: Is It Possible to Identify the Rationale for Divergences in Approved Indication From Public Assessment Reports?

Bujar

Ferragu

McAuslane

et al. 2021

Clinical Therapeutics

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Although it cannot be expected that different medicines' regulatory agencies always reach the same review outcome, it is important that decision making is documented and communicated to ensure transparency. This study examines whether justification for divergences between the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA) regarding approved indications could be identified from the agencies' public assessment reports (PARs). We focused on 9 products previously identified to have been submitted simultaneously to both agencies with the same indication but had a different indication approved; there were 15 differences in indications. Our analysis confirms that the rationale for observed divergent indication decisions was predominantly found in the benefit-risk section of the PAR (9 of 15 cases for the FDA and 10 of 15 for the EMA). If not found in the benefit-risk section, the rationale for these decisions was found in other PAR sections (eg, labeling or clinical efficacy section) or not at all. Our study found a small number of inconsistencies or gaps in how, where, and whether regulatory decision making on approved indications are documented by the FDA and the EMA. We believe it is important for regulators to standardize their approach and systematically and transparently document their rationale for the approved indication, using a structured benefit-risk assessment format within the PAR. This process is especially important for innovative products for which experience in evaluating similar products worldwide is limited, particularly as agencies are striving to build effective regulatory processes by leveraging assessments by trusted reference agencies through approaches such as reliance. Clear and systematic communication and documentation of the decisions in the PAR are central and should continue to evolve as a best practice; an enabling step toward this would be a harmonized PAR template for use by agencies globally.

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“…Finally, we must ask whether cancer is somehow morally special, such that it justifiably commands unlimited resources while all other health care needs are subject to cost constraints. This has recently been referred to as “onco-exceptionalism,” and is regarded as being ethically problematic (Salcher-Konrad and Naci 2020 ; Fu et al 2021 ).…”

Section: Solidarity: the Challenge Of Limited Resourcesmentioning

confidence: 99%

Precision medicine and the fragmentation of solidarity (and justice)

Fleck

2022

Med Health Care and Philos

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Solidarity is a fundamental social value in many European countries, though its precise practical and theoretical meaning is disputed. In a health care context, I agree with European writers who take solidarity normatively to mean roughly equal access to effective health care for all. That is, solidarity includes a sense of justice. Given that, I will argue that precision medicine represents a potential weakening of solidarity, albeit not a unique weakening. Precision medicine includes 150 targeted cancer therapies (mostly for metastatic cancer), all of which are extraordinarily expensive. Our critical question: Must a commitment to solidarity as defined mean that all these targeted cancer therapies should be guaranteed to all within each country in the European Union, no matter the cost, no matter the degree of effectiveness? Such a commitment would imply that cancer was ethically special, rightfully commandeering unlimited resources. That in itself would undermine solidarity. I offer multiple examples of how current and future dissemination of these targeted cancer drugs threaten a commitment to solidarity. An alternative is to fund more cancer prevention efforts. However, that too proves a threat to solidarity. Solidarity, with or without a sense of justice, is too abstract a notion to address these challenges. Further, we need to accept that we can only hope to achieve “rough justice” and “supple solidarity.” The precise practical meaning of these notions needs to be worked out through a fair and inclusive process of rational democratic deliberation, which is the real and practical foundation of just solidarity.

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Unintended Consequences of Coverage Laws Targeting Cancer Drugs

Cited by 4 publications

References 15 publications

Conditional Drug Approval as a Path to Market for Oncology Drugs in Canada: Challenges and Recommendations for Assessing Eligibility and Regulatory Responsiveness

Conditional Drug Approval as a Path to Market for Oncology Drugs in Canada: Challenges and Recommendations for Assessing Eligibility and Regulatory Responsiveness

Transparency in European Medicines Agency and US Food and Drug Administration Decision Making: Is It Possible to Identify the Rationale for Divergences in Approved Indication From Public Assessment Reports?

Precision medicine and the fragmentation of solidarity (and justice)

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