Should regulators insist on robust evidence that a new drug shows clear benefit to patients as a condition of approval, or are demands for such levels of certainty unrealistic, or even unethical? Marc Beishon reports.
- two studies from the US and Europe that show that a majority of drugs enter the market without showing Overall Survival or Quality of Life, and only about 15% of these have since done so. The majority of cancer drugs enter the market without showing evidence of benefit on overall survival or quality of life.
- Other studies have shown no relationship between price and clinical benefit of FDA-approved drugs.
- There are just not many new cancer drugs that qualify as real game changers, particularly for solid tumours, although some are certainly huge money spinners for the pharmaceutical companies, owing to eye-watering price-tags.
- A recent and “ridiculous” example, he says, is FDA approval for using adjuvant sunitinib for renal cancer. “Of the two trials, a larger one of 2,000 or so patients was totally negative, and a smaller one of 600 was only positive for progression-free survival but not for overall survival, and it has substantial toxicity.”
- “We do have some great new drugs,” says Tannock. “But I am concerned for patients who have little idea how to judge which ones are effective and end up selling everything to get them.”
- He argues that the progression-free survival (PFS) findings from trials may be biased, citing the BOLERO-2 trial, which showed that adding everolimus … to exemestane, doubled PFS in patients with advanced HER2+ breast cancer. “But toxicity was such that 25% of patients left the trial – and while the PFS was impressive, longer-term survival was negative.
- If you have an agent that improves PFS with minimal toxicity, such as aromatase inhibitors, that’s fine, but for those with high toxicity such as everolimus or sunitinib it is misguided to approve them.”