No one is dying of cancer because of FDA foot-dragging. No one.
Nearly all experimental cancer drugs have no therapeutic value. A report from BIO, a biotech industry group, finds that only 5% of all new cancer drugs tested 2006–2011 were found to be safe and effective:
From Clinical Development Success Rates, 2006–2011
This means, to a first approximation, if twenty patients were allowed to freely access experimental drugs, nineteen would receive drugs that were either unsafe or ineffective.
At least 40% of new oncology drugs in this period failed because they were unsafe. So eight of our hypothetical twenty patients would have been actively harmed by untested drugs. Because cancer has such a high mortality rate, the bar for deeming a cancer drug safe is set very low. You really have to hurt someone with a cancer drug before deciding it is too dangerous to use.
Another eleven would receive no clinical benefit – but would suffer considerable exposure to side effects as well as the cost of care.
Only one patient would benefit. But the bar for benefit is also set very low. Oncology drugs are rarely approved any more on the basis of cure rates[1] , because it takes many years to evaluate cures. Instead, surrogates, such as “progression-free survival” or biomarker responses are used. The advantage of these measures is that they can be evaluated in months rather than years, speeding regulatory approval and patient access.
The disadvantage of surrogate responses is that they are not good indicators of treatment efficacy. One analysis indicates that for 55 cancer drugs approved by the FDA 2009–2014 on the basis of surrogate responses, only 16% of those responses show a high correlation with survival[2] .
If we have a problem with cancer drug approval by the FDA, it is that they approve too many drugs too quickly. Fewer than half of the cancer drugs approved 2006–2016 show meaningful clinical benefit[3] . Keep in mind that these new drugs routinely exceed $100K for a course of treatment[4] . We may be paying blockbuster prices but we are not getting blockbuster results.
Experimental cancer drugs are overwhelmingly unsafe and ineffective. Access to them is not a problem. Not for patients, anyway. So why the push for “right to try” legislation? Who benefits?
You might think the answer is “greedy drug companies” but you would be wrong. To its credit, Merck stated that “…’Right-to-Try’ legislation is not in the best interest of patients and is unlikely to help us bring forward innovative, safe, and effective medicines to all patients as quickly as possible”. Good for them for speaking out.
Right to Try is an idea hatched by right-wing libertarian ideologues in their never-ending quest to de-legitimize government in general and regulatory agencies in particular. What is particularly cynical about this ploy is that the FDA already has a “Compassionate Use” program which allows access to experimental drugs. About 99% of Compassionate Use requests are approved, usually within days for urgent cases.
No one is dying because of the FDA. But plenty of people are being scammed by the right-wingers. Although Merck and other pharma incumbents may oppose it (for now), others will not pass up the opportunity to sell poison for profit. Right to Try will become the Right to Rob.
Footnotes
[1] Surrogate endpoints in oncology: when are they acceptable for regulatory and clinical decisions, and are they currently overused?
[2] Strength of Validation for Surrogate End Points Used in the US Food and Drug Administration’s Approval of Oncology Drugs.
[3] Magnitude of Clinical Benefit of Cancer Drugs Approved by the US Food and Drug Administration | JNCI: Journal of the National Cancer Institute | Oxford Academic