Policy, BioPharma

Pence meets with “Right to Try” leaders at White House

Should unproven drugs be made available to terminally ill patients? Vice President Mike Pence looks to be preparing that discussion for the national stage, hosting “Right to Try” advocates at the White House.

By Juliet Preston on February 09, 2017
Vice President Mike Pence

U.S. Vice President Mike Pence

Vice President Mike Pence is well known for his “right to life” anti-abortion stance. But in a Tuesday meeting at the White House, Pence was focused on the other end of the spectrum — prolonging the lives of terminally ill patients.

It’s a movement known as “Right to Try.” Proponents are lobbying for legislation that can increase access to safe but unproven drugs when no other therapy is available for a terminal condition. In practice, this means the drug has passed Phase 1 safety and tolerability studies, without yet demonstrating efficacy and gaining FDA approval.

A select few patients can access these experimental drugs through formal clinical trials. However, the vast majority of patients are not eligible. Their disease may be too far advanced or there may be geographical barriers.

While serving as governor of Indiana in 2015, Pence signed legislation allowing in-state physicians to prescribe investigational drugs to these patients who have run out of options. Many other states have passed similar laws and Pence’s boss has also hinted at support for federal legislation.

On Jan. 31, President Donald Trump met with seven biopharma leaders at the White House and led a roaming discussion on manufacturing, FDA regulations, taxes and the need to curb what Trump called “astronomical” drug prices.

The president also touched on compassionate use, expressing dismay that terminally ill patients are currently being denied drugs that are still under review.

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“One thing that’s always disturbed me: they come up with a new drug for a patient who’s terminal, and the FDA says we can’t … approve the drug, because we don’t want to hurt the patient,” Trump told reporters. “But the patient is not going to live more than 4 weeks, [anyway]. So, we’re going to be changing a lot of the rules.”

Despite Pence and Trump’s support for compassionate use, there are few details on how such legislation could realistically be implemented — while they also work to bring healthcare costs down.

For decades, the FDA has had a program known as “compassionate use” or “expanded access,” which shares the Right to Try sentiment. It was updated in 2009. By 2015, some six thousand patients had applied, with just 33 applications being denied on a case-by-case basis.

Even with this program, many barriers remain. On its website, the American Cancer Society notes that patients often struggle to access the drugs.

Who supplies the actual medicine? It’s not readily available on pharmacy shelves and many therapies for terminal illnesses are increasingly personalized.

On a related note, who pays? For the drug, the administration, the care and the fallout from any potential side effects? Insurance companies are not required to foot the bill.

There are a lot of practical questions that need to be answered, alongside the ethical debates.

At Pence’s side in Indiana in 2015 was Jordan McLinn, a now eight-year-old boy from Indianapolis who became the face of the state’s Right to Try bill. He flew with his mom to Washington, D.C., to meet with Pence on Tuesday.

McLinn has Duchenne’s muscular dystrophy (DMD), an inherited disorder that affects 1 in every 7,250 males aged 5 – 24 years.

In September 2016, a disease-modifying drug from Sarepta was provisionally approved by the FDA, after a powerful advocacy movement by the DMD community. The therapy, dubbed Exondys 51, has been priced at $300,000 per year, despite controversially minimal improvements in patient outcomes.

Earlier that year, the agency rejected a DMD therapy developed by BioMarin Pharmaceuticals after a panel of independent advisers concluded that the drug, Kyndrisa, had not demonstrated any measurable efficacy.

Sarepta and BioMarin’s drugs were both in late-stage trials in 2015. They targeted a population of around 1800 patients in the United States and 5,000 patients overseas. There were no disease-modifying treatments available, which meant for every patient not in a clinical trial, the Right to Try law would be relevant.

Yet Kyndrisa didn’t prove effective and Exondys 51 demonstrates such minor gains relative to its price that it’s struggling to secure post-FDA approval reimbursement.

On the other hand, no significant safety issues were reported by either company. The drugs gave hope where there wasn’t any before. Who are we to deny them access, many would ask.

Photo: John Moore/Getty Images