Right-to-try drug bill could needlessly raise patients' hopes, experts say

- Maret 12, 2018

Congress may be on a speedy path to lifting the hopes of terminally ill patients. Whether it’s anything more than a feel-good exercise is an open question.

The House of Representatives is expected to deliver the deciding vote for a right-to-try bill Tuesday that President Donald Trump touted in his State of the Union address and would give terminally ill patients — or those likely to die prematurely — access to experimental medicines without the FDA’s blessing. The Senate, which already passed its own bill, is considered likely to adopt the changes.

The right-to-try idea, conceived by a little-known libertarian think tank, has an instantaneous appeal. But FDA, drug industry and health policy experts say it’s unlikely to significantly increase the number of patients who get unapproved drugs. And the few who do could be exposing themselves to unnecessary risks while compromising medical research.

“I think this is feel good legislation for legislators, rather than meaningful legislation for people in need,” said Kenneth Moch, president and CEO of Cognition Therapeutics, which is trying to develop treatments for neurological conditions like Alzheimer’s disease.

Former FDA Commissioner Robert Califf said he worries any right-to-try law will set patients up for disappointment. “What it may do is to raise expectations of vulnerable people and their families that there is some miracle cure that is going to be made available.”

Even if more companies release products under right-to-try — a prospect Califf thinks is doubtful — the odds for helping patients are long. That’s because most promising new drugs end up being too dangerous or simply don't work.

FDA estimates that only five to seven of every 100 drugs that enter the first phase of human testing eventually get approved. The rest have risks that outweigh their stated benefit or don't deliver on those promises.

Critics say the problem with right-to-try is it mistakenly presumes the FDA is the barrier to patients accessing unapproved medicines outside clinical trials.

FDA’s compassionate use program already provides experimental drugs to very sick patients, approving 99 percent of the requests it receives each year. It’s undergone changes in recent years to make it easier for doctors and patients to navigate the process.

The hurdles to accessing experimental medicines largely rest with pharmaceutical companies that have a variety of reasons for turning patients down. New drugs are expensive to make and companies need enough product for clinical trials — the only way to get FDA approval. They also need patients to be incentivized to participate in studies to get a sufficient cross-section to evaluate the product. Obtaining an experimental drug outside of a trial carries the risk of disrupting the costly and time-consuming process, especially if a person outside of a trial is harmed by the medicine.

“The right-to-try approach blames an innocent entity for access problems, and by pushing legislation that targets the FDA, those who advocate for right-to-try are not putting forth viable solutions to these problems. The only way forward is to work with companies and to find out what would make them willing to provide their experimental products to patients who wish to try them,” said Alison Bateman House, a bioethicist at New York University.

The Trump administration’s FDA chief raised these issues with House lawmakers in the fall.

“There is a perception that certain products that aren’t being offered under FDA expanded access … will be offered under right-to-try,” Commissioner Scott Gottlieb told the House Energy and Commerce Committee in October. “I don’t see that.”

Gottlieb, who homed in on the barriers drug companies have to providing experimental medicines, also emphasized that the vast majority of drugs provided to patients through its compassionate use program turn out to be ineffective. “We know that 70 percent of all drugs that are offered under an expanded access setting are never approved by FDA," Gottlieb said.

Right-to-try has been opposed by mainstream medical organization and patient groups like the American Cancer Society Cancer Action Network, the American Society of Clinical Oncologists and the National Organization for Rare Disorders.

The drug industry also has issues. Johnson & Johnson has already said it will continue to incorporate FDA review in all experimental access requests, noting the FDA may be “uniquely aware” of safety information that could affect whether a patient should receive a drug.

This combination of resistance — from the FDA chief, medical groups and the drug industry — threatened to doom right-to-try in the House, even though the Senate passed the bill by unanimous consent in August. For months, Energy and Commerce leadership appeared disinclined to move a bill and worked with Democrats on an alternative proposal.

But then came Trump's State of the Union appeal and congressional Republicans' annual retreat, where the White House aggressively pushed for passage of the measure.

“The State of the Union targeted this policy as an administration goal, thrusting the conversation into the national spotlight,” an Energy and Commerce Republican aide told POLITICO. This spurred talks with members spearheading the law, including Sen. Ron Johnson and Reps. Andy Biggs and Brian Fitzpatrick. The committee worked with those lawmakers and Vice President Pence to craft the consensus language.

Energy and Commerce Chairman Greg Walden sought assurance from FDA that tweaks to the law would ensure drug companies wouldn’t have perverse incentives to choose the FDA compassionate use program over right-to-try or vice versa. He also sought FDA feedback on getting the fastest update on patients using right-to-try, an aide familiar with the negotiations told POLITICO.

The House bill also underwent tweaks to satisfy some concerns from FDA and outside groups that the law put patient safety in jeopardy. It includes new requirements that FDA be made aware when drugs are provided under right-to-try and gets reports of any safety events with the drugs.

Those changes have been lauded — but critics still argue they're not enough.

“I think they’ve certainly tried to take steps to add some important provisions” like strengthening the informed consent of patients, said Jeff Allen, president and CEO of the Washington-based Friends of Cancer Research. But Allen said the bill still violates one of his organization's guiding principles: “The FDA is not the problem and taking them out would only introduce more problems,” he said.

Allen said patients may get more access to drugs under right-to-try, but they are unlikely to come from credible companies who he said will prefer the FDA’s process. “Unfortunately, I fear it will be drugs they shouldn’t be receiving or could be extremely unsafe or have flaws in the manufacturing practice or other risks that may not be known at the time.”

Such concerns could stoke Democratic resistance, though probably not enough to put the bill in danger. The legislation requires the support of two-thirds of members present and voting because it is being considered under the expedited process known as suspension of the rules.

Energy and Commerce ranking Democrat Frank Pallone said the bill “provides false hope to patients and their families, who will assume they will have access to investigational therapies even though the bill does nothing to require manufacturers to provide these therapies to the patient.” Only one doctor has publicly said he provided patients with drug under state right-to-try laws.

A Democratic aid expressed frustration with the way Republicans are “jamming” the bill through the House, noting suspension of the rules is supposed to be reserved for noncontroversial measures.

If the legislation passes the House, it will need to go back to the Senate to iron out differences such as precisely which patients would be eligible for right-to-try. Senate leadership would not comment on its plans to take up the legislation.

Regardless of the outcome, the House vote marks a big victory for the libertarian Goldwater Institute, which pushed the bills through the states and on the federal level. The issue is the first in a broader anti-regulatory agenda — and could lead to the roll back of other FDA laws designed to protect consumers from unsafe, and unproven drugs.

The group already has its next target: FDA marketing rules that keep drug companies from promoting their products for unapproved uses.


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