Right-to-try: Cause for Hope or Just Hype?

By Jason Poquette via Multibriefs

A 2010 movie entitled "Extraordinary Measures" starred Harrison Ford and was based on the real life story of the parents of two children with a rare, terminal disorder known as Pompe’s disease.

It was a good movie and an amazing story. The kids got the medicine during the trial phase and it worked.

I wonder if the supporters of right-to-try expect that newly signed legislation will produce many more stories like this. Maybe it will.

On May 30, 2018, President Trump signed the "Right-to-Try Bill" into law. The bill, sponsored by Sen. Ron Johnson, R-Wis., was created to give terminally ill patients the opportunity to try unapproved medications which could cure them or extend their lives. "Today I am proud to keep another promise to the American people as I sign the right to try legislation into law,” said the President, “and a lot of that trying is going to be successful. I really believe that."

Senator Johnson was equally optimistic. After the passing vote he remarked, "Patients with terminal diseases ought to have a right to access treatments that have demonstrated a level of safety and could potentially save their lives."

The legislation provides the right for terminally ill patients the right to try, under medical supervision, medications that have successful completed Phase I of the FDA approval process and are ready for human trials.

Under normal circumstances it could be months or years before such medication would be commercially available. The new law gives a glimmer of hope to patients who have exhausted available treatments and my ineligible for clinical trials.

But is it really hope...or just political hype?

The legislation isn’t without its critics. It has been pointed out that historically only about "13.8% of drugs that enter a Phase 1 study ever get approved." And it is worse for oncology drugs.

"The success rate for cancer drugs, which will likely be a key focus of the right-to-try program, is 3.6%." This means that, theoretically, the vast majority of drugs that a terminally ill patient will get access to will do no good. In fact, they could be positively harmful.

Ellen V. Sigal, chairwoman of an advocacy group called Friends of Cancer Research, said, "This bill endangers patients and will offer them no value, just false hope."

As a pharmacist I do believe there are some significant risks involved in trying medications that haven’t been thoroughly tested. But these patients will generally understand the risks.

In fact, terminally ill patients have already been obtaining access to medications prior to FDA approval via “compassionate use” or “expanded access” programs already in existence.

I’m not really convinced the law offers "false hope" either. The reality is that even "proven" treatments for many chronic and terminal illnesses are far from perfect. There are no guarantees in medicine.

Even the best treatments are not 100 percent effective. Patients generally know this. Sadly, those with chronic and terminal conditions know it better than most of us do.

One concern that hasn’t been addressed is the cost of treatment. Who will pay for it? I’m doubtful that health plans will cover such experimental treatments.

In fact, I’m not sure that they should. But that leaves patients paying for it out of their own pockets, which could get very expensive.

Are patients prepared to exhaust what little may be left of their life savings on a long-shot treatment that hasn’t been proven yet? These are hard questions.

Another concern is whether the patient’s "right-to-try" means manufacturers have an obligation to supply. There are risks for the manufacturer as well.

Patients enrolled in clinical trials are carefully selected to control variables and minimize risks that may be related to co-morbid conditions. Patients who receive treatment via right-to-try may experience side effects for treatment failure that reflects poorly on the manufacturer, even though it may not be relevant to most patients who would try the new medication.

So...rght-to-try...hope or hype?

I’d say it’s a little of both.

But for me, some hope is always better than none.

Jason Poquette, BPharm, R.Ph., earned his pharmaceutical degree from the University of Connecticut in 1993. In addition to his 20 years of practice, Jason writes his own blog, The Honest Apothecary, a pharmaceutical news and information site dedicated both to patients and to fellow pharmacists.