Bypassing Ethics For Politics

The “Right To Try” law allows terminally ill patients to obtain experimental drugs without getting federal approval. It’s a proposal being advanced in several states by patient advocates who are frustrated by the yearslong federal approval process for experimental drugs in the pipeline.

States Move To Expand Experimental Drugs For Some : NPR.

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I first heard about this on Sunday listening to NPR – Colorado’s move to bypass the FDA and allow free access for patients to experimental drugs.

While I definitely support and sympathize with the motivation, after working now nearly 5 years in a cancer center where we do clinical research, I do feel I’m in a position to criticize it as well.

First off, I’ll say that yes, there is good reason to want patients to have access to experimental drugs and other non-FDA-approved treatment options.  However, that should be done in a highly organized fashion which ensures full disclosure and noted acceptance by the patient, and also in a way so as the data from the patient’s care can be further used to help other patients.

Yes, the process for FDA approval takes years – often even decades – meaning a lot of drugs actually never make it to the market in the US. For example, one study we’re participating with now which is not groundbreaking but is just designed to get a competing drug onto the market (and potentially reduce costs to patients), is not expected to actually gain approval and be for sale until the mid-2020’s.

And often, a drug is approved to treat X and Y, but it may also be good at treating Z and A, but just not have FDA approval because another drug already is in that arena which makes getting that approval harder.

But really, what this seems to be, is a way to simply side-step GCP – Good Clinical Practice – the backbone of clinical research in the U.S.

GCP is designed with 2 objectives in mind. One is to protect the integrity of the data being collected which leads to better development of treatments and better safety for the public. And the second – the priority, actually – is to protect the safety of the study subject.

It means no experimenting on vulnerable populations – children, pregnant women, prisoners, people who speak a different language, etc.  It means following a protocol so that the data received is actually able to be compared to data from another patient, even if that patient may be on the far side of the world.

It does not mean allowing the patient to willy-nilly choose a treatment out of desperation or misguided knowledge.

Imagine a world where the drug companies no longer care to go through a clinical trial because all they have to do is market strongly and strategically to get patients to beg their doctors for it?

Outcomes, be damned!  Data available for physicians to make informed care decisions?  Who needs that?

Of course, the crux here is that drug companies could be too scared to even offer the drugs if they aren’t FDA-approved.  They have enough of a hard time when they do have an approved drug and run into pricing scandals or long-term survival issues, and more.

And, if I were a physician in this litigious society, I would never been inclined to provide these non-FDA-approved treatments without at least reliable data to back-up my choice – of which there will be none without GCP and properly designed clinical research.

And don’t get me started on the insurance issues here…

So, what will come of this legislation remains to be seen, but I think it’s clear that – like when laws regarding teaching science and literature get passed – this is being done by politicians with their hand open for a vote or a handout – whichever comes first.

NOTE: If you’re interested in learning about the history of why clinical research is designed the way it is – including experiments done before by Nazi’s and even our great US military – there is a free online training run by the NIH on Good Clinical Practice.

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