Policy, Surveys

What does biotech think about ‘right-to-try’? It might surprise you

“Right-to-try” has made its way back to the headlines, filling our Twitter feeds with opinions on whether the House should reject or embrace a bill that would allow patients access to unapproved therapies. We know what politicians think, and what the public thinks. What does biotech think?

We asked ourselves that question here at Endpoints News, so we crafted a brief survey posing this question to our readers: Do you approve or disapprove of “right-to-try”?

We had expectations of what you might say. We were wrong.

The survey was enlightening on several accounts. The data were compelling, but — unsurprisingly — the jewels were in your comments. They were a far cry more thoughtful and nuanced than the comment feeds of many news sites. For that, we were proud. We were surprised, however, by how much the topic polarized you. Given an audience that often stands united on topics like these, we were instead intrigued to find “right-to-try” divided our readers.

Nearly 58% of our readers disapprove of the current legislation, while 42% approve of “right-to-try.”

The topic inspired a deluge of comments among those who responded. With 1,194 votes on the topic, nearly half — 544 —took the time to write out their thoughts on the issue. I read through hundreds of comments to find out what was behind your votes.

But first, the obligatory explainer for those of you who missed the debate.

In our survey, those who approved of the legislation shared overwhelmingly consistent reasoning. Many had a variation of the following response from an anonymous commenter:

Terminally ill patients who have no other treatment options should be allowed to try a treatment or therapy if they think it will extend their life. Why should we legislate treatments? Shouldn’t someone be able to make their own decision about their health?

Those who disapproved of the legislation, however, had several reasons why. Most common was concern for patient safety. This commenter put it most succinctly:

It may expose people without choice to useless and dangerous medicines leading to more suffering. There is an unfounded presumption that experimental drugs work. They don’t in most of the cases. Experimental drugs should always be tested under controlled conditions first.

That concern for patient safety was closely followed by worries that “right-to-try” would pose a risk to business. The fear is that pharma would somehow be held accountable for disastrous outcomes, either through litigation or the bad press following an experimental treatment gone wrong.

This may be suitable for some patients with terminal illness, however, the risks of expanding this policy are very dangerous and risk making a cautious industry even less confident (because of the risk of litigation), which will stifle future innovation and thus harm patients further in the long run.

Or more plainly:

We cannot claim any good anecdotal data but must live with all toxicity data.

Then there was the overwhelming agreement among dissenters that the bill was completely unnecessary, as the FDA’s Expanded Access policy (often called “compassionate use”) already gets experimental drugs to people who are out of other options. The FDA already approves 99% of these requests, but sometimes makes small safety-related changes such as dosing or frequency with which the patient takes the drug.

Current rules give patients wide access to experimental therapies. If the administration is concerned about the proliferation of regulations, it should avoid enacting unnecessary ones! — Bernard Munos

A mechanism already exists to make experimental drugs available to patients. Right-to-try is a thinly veiled attempt to reduce the regulatory authority of the government.

Perhaps most compelling were responses we received from physicians concerned that “right-to-try” would perpetuate snake oil marketing tactics that prey on vulnerable and desperate patients.

I have seen many patients desperate to do anything to help their loved ones in the most trying of times. I’ve seen a girl whose mother was in denial she had terminal (brain cancer), praying that the expensive moonshot homeopathic therapy she had found would cure her daughter. I’ve seen patients claim that they had found the cure to cancer but the rich were hiding it to make money off sick people. I’ve seen a man pleading to donate his organ to his brother thinking it could save him. These patients are vulnerable. They are desperate. They are in despair. They are grieving. Their worlds have been turned upside-down. If we allow ‘right-to-try,’ they WILL be exploited. They will be taken advantage of even by the most well-meaning business interests. You need not look further than the often value-destructive nutraceutical industry to recognize that. This cannot be allowed. It does not empower patients. It shackles them.

The general public is dramatically misinformed about right to try. It comes to a head during emotional times. The media sensationalism of the situation doesn’t help. Research organizations use the words ‘life saving clinical trial’ like they all work. This too is a disservice. That causes people to feel that they are being denied something instead of being protected from snake oil.

We put together the following word cloud that sums of the common topics discussed in comments.

Because they were too good to leave out, here are some more comments for your perusal:

Provided patients are well informed of the risks inherent in using yet-to-be-proven treatments (something we should be good at as part of the clinical trials process), and only for life-threatening conditions and ensuring there is no coercion, I see no reason why we should not allow patients the opportunity for a chance to survive. To do otherwise is at least patronizing, and at worst disempowering.

Right-to-try, as it is typically framed, is a cynical ploy by privileged Americans to subvert FDA’s authority and essentially buy a waiver of the rules designed to protect all Americans from dangerous or worthless drugs. Like insurance, our entire system of clinical research and regulated access to drugs only works if everyone participates, that includes those who can afford access to experimental drugs under ‘right-to-try.’ ‘Right-to-try’ framed properly would create incentives to drug sponsors and require third-party payers to participate to ensure limited, regulated availability of experimental drugs to Americans with serious medical conditions, regardless of financial means or status, when the options to treat with approved medications or participate in properly vetted clinical research studies are not feasible. The framework is in the law already; it just needs to be improved upon.

Only if we have the proper consent from patients. We can also look at novel Combinations that do not get executed due to IP corporate issues.

This would be a BIG step backward to the era prior to the 1962 [Kefauver-Harris] Drug Amendments to the 1938 Food, Drug, and Cosmetic Act. Desperately ill people should simply NOT have open access to investigational agents with unproven efficacy NOR unproven safety. Open label extension studies and early expanded access programs/compassionate use exist, seem to increase access to promising therapies for which there are some safety and efficacy data, and maybe should be expanded. Finally it is payor community (PBM’s and government bodies like NICE in the UK) that are responsible for restricting access to approved products to a much greater extent than is the case for the agents under discussion here. If the US Government is really interested in helping more sick people gain access to proven much less promising but unproven therapies they are looking down the wrong road.

We could be back to coffee enemas with ‘right-to-try.’ There should be easier access to drugs in development but blanket ‘right-to-try’ isn’t the answer.

Illustration: Shutterstock

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Research Scientist - Immunology
Recursion Pharmaceuticals Salt Lake City, UT
Director of Operations
Atlas Venture Cambridge, MA

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