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Questions and Answers: The debate over the ‘right to try’ experimental drugs

WASHINGTON (AP) — The idea is a political crowd-pleaser with a catchy slogan: giving desperately ill patients the “right to try” experimental medicines.

Lawmakers in the U.S. House of Representatives on Wednesday became the latest group of politicians to back the effort, sending a bill to the Senate which President Donald Trump has pledged to sign into law.

A federal right-to-try law — first championed by anti-regulatory libertarians — would overturn decades of precedent in which the government served as the gatekeeper to unproven medicines. And critics fear it could rollback long-standing safety regulations that protect patients.

A look at the issue:

WHAT IS RIGHT-TO-TRY LEGISLATION?

The idea is to give terminally ill patients quick, unfettered access to experimental drugs that might help them, without having to seek approval from the Food and Drug Administration. Lawmakers in 38 states have already written it into law. Vice President Mike Pence signed Indiana’s law while serving as governor. But the state laws have little effect without a change to the federal requirement for FDA oversight.

Opponents say right-to-try is political theater that won’t actually help more patients access unapproved drugs. They worry that the measure could expose patients to potentially harmful drugs and allow unscrupulous actors to take advantage of those desperately seeking treatments.

This week, four former FDA commissioners who served under Presidents Barack Obama and George W. Bush issued a joint statement opposing the federal legislation, saying it would “create a dangerous precedent that would erode protections for vulnerable patients.”

DOES FDA EVER GRANT PERMISSION NOW?

In almost all cases, the answer is yes. The FDA receives about 1,000 requests for so-called “compassionate use” each year and approves more than 99 percent of them, according to agency figures. The process usually takes about four days, although in emergency cases regulators can grant permission immediately over the phone.

While the paperwork used to take about 100 hours, the FDA revamped the process in 2016 to a single form that takes about 45 minutes to complete, according to an agency spokeswoman. The requests are submitted by a patient’s physician, who must decide whether the possible benefits of taking an unproven medicine are likely to outweigh the risks. New drugs undergo years of testing before drugmakers seek FDA approval to put them on the market.

The FDA also enforces rules that prevent companies and doctors from profiting from selling unproven drugs. Companies can only charge patients enough to cover the costs of providing the drug. Without that oversight, opponents of the bill fear it could do more harm than good.

WOULD DRUGMAKERS HAVE TO COMPLY?

No, there’s nothing in the state laws or the federal bill that would require pharmaceutical companies to provide experimental drugs, which they spend millions of dollars developing and testing. Drugmakers are usually the main obstacle to gaining early access, though how often such requests are denied isn’t clear.

There are a variety of reasons, including legal risks, for why a company might decline requests. Some do not have enough of the experimental drug to provide it outside of their own testing. For smaller companies, they may not have the staff and resources to field and process dozens or sometimes hundreds of requests.

The federal bill includes language designed to ease the legal risks of making drugs available. It shields drugmakers from most lawsuits that might emerge from an injury or side effect of an experimental drug.

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