Report from American Society of Clinical Oncology Symposium 2020 and American Society of Clinical Oncology Gastrointestinal Cancer Symposium 2021
- Six out of 18 cancer drugs with accelerated approval still have indications on labeling and are recommended in guidelines despite not showing improvement in primary endpoints in post-approval trials.
- This issue highlights the imbalance between speed and evidence in the accelerated approval pathway.
- Recently, the FDA has taken more regulatory actions to address these situations.
- There is a need for additional guidance and reforms to ensure FDA-approved drugs are proven to be safe and effective for patients.
Patients with cancer need timely access to drugs that meaningfully improve how they feel, function, or survive. However, measuring clinical endpoints such as improvement in overall survival can take time, and patients may be willing to tolerate uncertainty about such benefits in exchange for early access to promising cancer drugs, particularly for cancers that lack other treatment options.
The accelerated approval pathway of the US Food and Drug Administration (FDA) covers this situation. The FDA may grant approval to any drug—although it is most often used in cancer—that has shown improvements in surrogate measures in clinical testing that are only reasonably likely to predict actual clinical benefit (that is, improved survival or quality of life). Cancer related surrogate measures include changes in tumour size or time to progression of cancer. These surrogate measures may ultimately be shown to predict meaningful clinical benefit, but this is often not the case...
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