The FDA should not require efficacy testing. If it’s safe let patients and their doctors do what they want. In practice that’s what they do anyway, see off label prescribing which is around one fifth of all prescriptions in the US. Removing efficacy testing would double or more the number of new drugs available.
Fraud is already illegal. Doctors can already be sued for malpractice. Testing for efficacy makes no sense in a world in which off label prescribing is routine.
> Assessing the FDA via the
Anomaly of Off-Label Drug
Prescribing
—————— ——————
> Once a drug has been approved for some use, the FDA has almost no control over how that drug is actually prescribed. The prescribing of drugs for non-FDA-approved uses, called “off-label prescribing,” is widespread. Drugs prescribed off-label have not met the FDA’s requirements for proving efficacy in the off-label applications. The practice of off-label prescribing therefore raises interesting questions. Why does the FDA, in effect, require that some drugs be tested for efficacy but not others?
If there are good reasons for the FDA to have strong pre-approval powers regarding efficacy, shouldn’t FDA post-approval powers be commensurate? Alternatively, if there is good reason for widespread off-label prescribing, doesn’t this call into question the FDA’s pre-approval powers?
> There are also scientific reasons to replace Phase 3. The reasoning behind the Phase 3 requirement — that the average efficacy of a drug is relevant to an individual patient — flies in the face of what we now know about drug responsiveness. Very few drugs are effective in all individuals. In fact, most are not effective in large portions of the population, for reasons that we are just beginning to understand.
> It’s much easier to get approval for drugs that are marginally effective in, say, half the population than drugs that are very effective in a small fraction of patients. This statistical barrier discourages the pharmaceutical industry from even beginning to attack diseases, such as Parkinson’s, that are likely to have several subtypes, each of which may respond to a different drug. These drugs are the underappreciated casualties of the Phase 3 requirement; they will never be developed because the risk of failure at Phase 3 is simply too great.
Off label use is allowed. Marketing for off label use is absolutely not allowed. Claims of off label efficacy are not remotely allowed. And the FDA has post approval powers to revoke approval for drugs not found effective. Doctor's autonomy leads to off label being allowed, and some of the legal arguments for it involve freedom of speech, not medical optimality.
Phase 3s already are no longer required, it looks like the article you linked was written in 2003? That was literally almost 17 years ago when we'd just barely sequenced the first human genome. There are entire fields of medicine - precision medicine - that literally address the whole subgroup question. Companion diagnostics are routine, rare disease approvals are up, plenty of Huntington trials in progress - that article might as well be about an alien civilization with how different things are today. Value based pricing, redefining cancer into separate genetically defined baubgroups and developing drugs for each, Gene therapy, ... It's no longer easier to get approval of a drug that's marginally successful in a large population, smaller populations with large effects make it way easier to see responses. And the 21st century cures act in 2016 evolved the regulatory landscape even more - it's like comparing Sputnik with the ISS. Yeah, they're both sattelites, but the field has come a long way in the time between the two.
Fraud is already illegal. Doctors can already be sued for malpractice. Testing for efficacy makes no sense in a world in which off label prescribing is routine.
> Assessing the FDA via the Anomaly of Off-Label Drug Prescribing —————— ——————
> Once a drug has been approved for some use, the FDA has almost no control over how that drug is actually prescribed. The prescribing of drugs for non-FDA-approved uses, called “off-label prescribing,” is widespread. Drugs prescribed off-label have not met the FDA’s requirements for proving efficacy in the off-label applications. The practice of off-label prescribing therefore raises interesting questions. Why does the FDA, in effect, require that some drugs be tested for efficacy but not others? If there are good reasons for the FDA to have strong pre-approval powers regarding efficacy, shouldn’t FDA post-approval powers be commensurate? Alternatively, if there is good reason for widespread off-label prescribing, doesn’t this call into question the FDA’s pre-approval powers?
https://www.independent.org/publications/tir/article.asp?id=...
> There are also scientific reasons to replace Phase 3. The reasoning behind the Phase 3 requirement — that the average efficacy of a drug is relevant to an individual patient — flies in the face of what we now know about drug responsiveness. Very few drugs are effective in all individuals. In fact, most are not effective in large portions of the population, for reasons that we are just beginning to understand.
> It’s much easier to get approval for drugs that are marginally effective in, say, half the population than drugs that are very effective in a small fraction of patients. This statistical barrier discourages the pharmaceutical industry from even beginning to attack diseases, such as Parkinson’s, that are likely to have several subtypes, each of which may respond to a different drug. These drugs are the underappreciated casualties of the Phase 3 requirement; they will never be developed because the risk of failure at Phase 3 is simply too great.
http://www.washingtonpost.com/wp-dyn/articles/A43257-2003Nov...