Assessment of Coverage in England of Cancer Drugs Qualifying for US Food and Drug Administration Accelerated Approval
The study evaluates the acceptance and coverage of FDA-accelerated approved cancer drugs by the National Health Service (NHS) in England, focusing on decisions made by the National Institute for Health and Care Excellence (NICE).
- A total of 93 cancer drug indications received accelerated approval from the FDA between 1992 and 2017.
- Out of these, 6 drug indications were withdrawn from the US market.
- 42 drug indications were not routinely available through the NHS in England.
- 12 of these 42 were not recommended by European regulators or NICE due to concerns about safety, efficacy, or cost-effectiveness.
- 30 drug indications were not reviewed by NICE or the European Medicines Agency (EMA).
- Among those recommended by NICE, 86% required additional negotiations such as confidential discounts, restricted use to specific subgroups, or additional evidence of efficacy.
- The study highlights a discrepancy between FDA accelerated approval and European regulatory standards, often due to reliance on surrogate measures for clinical benefits.
- NICE's recommendations were heavily influenced by clinical benefits and cost-effectiveness evaluations, often requiring further data or conditions for approval.
The US Food and Drug Administration’s (FDA) accelerated approval process allows drugs to be approved based on clinical trial findings that would otherwise not be acceptable for use in the traditional FDA approval process (ie, indicating changes based on surrogate measures that are only reasonably likely to estimate actual clinical benefit). Surrogate measures include biomarkers and other measurable physical properties that may be able to estimate the way a patient feels, functions, or survives. When surrogate measures are validated as being clinically meaningful, they can be substituted for traditional clinical outcomes as end points for clinical trials used for FDA approval, allowing those clinical trials to be conducted more quickly or among fewer patients. The accelerated approval process was developed to facilitate the approval of drugs that address unmet medical needs by allowing regulatory approval based on invalidated surrogate measures; this process requires that the manufacturer commit to conducting confirmatory clinical trials after approval is granted. Accelerated approval is thus comparable to conditional approval; however, the drugs are formally designated as fully approved from the time of their first approval.
Cancer drugs comprise the largest category of drugs that are granted accelerated approval. From 1992 to 2019, more than one-half of drug indication pairs with FDA accelerated approval have been for the treatment of cancer. In 2018, 83% of drugs approved via this process were indicated for the treatment of solid tumors and hematological cancers. Previous studies of cancer drugs undergoing accelerated approval indicated that most of these drugs are eventually granted traditional approval. However, confirmatory clinical trials are also likely to use surrogate measures of tumor response or disease progression and, in some cases, to use the same surrogate measures as those used to support the original accelerated approval decision. Surrogate measures of disease response are controversial in the context of cancer drugs, as some widely used surrogate measures (ie, response rate and progression-free survival) do not have a clear association with improvements in clinically meaningful outcomes, such as overall survival or quality of life.
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