The promise of accelerated approval of cancer drugs

KEY TAKEAWAY:  Few cancer drugs approved via the accelerated FDA approval pathway were judged to have verified benefits based on improvement in survival reported in confirmatory trials.

Many cancer patients depend on the accelerated drug approval program, which provides access to new cancer drugs faster but according to Jama the promise of these drugs may not be in line with reality.

The new study, published in the medical journal JAMA Internal Medicine on Tuesday, found that among cancer drugs that received accelerated approval from the FDA using surrogate endpoints or other measures, only one-fifth ended up improving overall survival rates in later confirmatory trials.

Let’s be clear: to cancer patients who have no hope the promise of a life-saving drug could be the only chance at extending life. However, who pays the cost for drugs that don’t work and can out healthcare sustem afford it?

As CNN wrote “yet these studies remind us that most new cancer drugs are of marginal benefit at best, despite their immense cost. Rigorous postmarketing studies with meaningful end points are expensive, requiring substantive infrastructure and oversight,” they wrote. “One might ask, ‘How can we afford to increase the rigor, and hence the cost, of assessing new cancer therapies after initial approval?’ The real question is: how can we afford not to?”

According to Evaluate Pharma “prescription drug sales expected to reach $1.18trn in 2024. Immuno-oncology line extensions significantly contribute to growth; emergence of novel technologies such as cell and gene therapy mark an inflection point in pharma’s evolution”.

Pharma is putting more money into Oncology than any other health condition. The reasons are quite simple; first, the current crop of drugs for other health conditions seem to work and are either generic or will soon be generic. Second, insurers are not likely to deny cancer patients new cancer drugs, so there is money to be made. Oh sure, we could say it’s about patients, but I don’t believe that fairytale anymore.

Accelerated approval of cancer drugs may be the only way to offer hope and promise to cancer patients but the FDA needs to balance these faster approvals with the science behind them.