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The “Right to Try” bill makes some procedural changes, intended to help the terminally ill. But without addressing core issues, it could be tough to meet the end goal of giving these individuals what they want and need—a chance.
The proposed federal legislation aims to expand access to experimental or investigational drugs that have cleared phase one testing. The current procedure is to contact the Food and Drug Administration, where a compassionate use request may be made. The new bill could allow that request to be made directly to the drug company.
The bill seems sensible. Patients gain quicker and greater access to experimental and potentially life-saving drugs, and manufacturers are protected from lawsuits if these drugs—not yet approved by the FDA—fail or accelerate the terminal diagnosis.
But although patients may request access to these experimental drugs, there’s no requirement that for-profit companies must grant those requests. In fact, the Senate’s version states, “No liability shall lie against a sponsor, manufacturer, prescriber, dispenser or other individual entity for its determination not to provide access to an eligible investigational drug…”
Dozens of states have similar legislation. Iowa’s law, “…does not require a manufacturer of an investigational drug, biological product, or device to provide or otherwise make available the investigational drug, biological product, or device to an eligible patient.”
Drug companies can’t be forced to provide a developing and preliminary product.
There are several reasons why a manufacturer may deny a compassionate use request for an unapproved drug. One thing that’s certain is that marketing and profitability come only when proven products receive official FDA approval, and that currently requires rigorous, controlled clinical testing.
The standard approval process includes a minimum of three testing phases on humans. Phase one tests 20-80 individuals and emphasizes safety and the discovery of side effects. Phase two tests hundreds of individuals with the disease or condition that the drug hopes to successfully treat. Typically, these are controlled tests with some receiving the experimental drug and others receiving a placebo. It tests for effectiveness and monitors safety. Phase three tests thousands of individuals under controlled conditions where safety and effectiveness continue to be monitored.
There is an accelerated approval program designed to fast-track drugs for life-threatening illnesses. But the fast-track approval process may still not be fast enough for the terminally ill.
And it remains that—despite this legislation that advocates for expanded access of unapproved drugs—the FDA continues to require the manufacturer to successfully finish phase two and three controlled clinical testing in order to potentially receive official drug approval.
It’s not clear, either, whether these separate compassionate use experiences could derail drug approval. The Senate’s version states that it will not use a clinical outcome associated with the use of an investigational drug to delay or adversely affect its review or approval. But the sentence continues with an “unless”—unless the clinical outcome is critical to determining the safety of the eligible investigational drug.
That provides zero clarity and assurances for manufacturers.
The House and Senate have passed their own versions on the “right to try.” Much more thought is needed as they work together to pass a bill that helps the terminally ill.
Consumer safety and company profitability are key in successfully bringing a drug to market for issues that are not life-threatening. The system in place can work well when time is on our side.
The terminally ill, though, need a second system—one where they can request access to a hopeful but unapproved drug and then have that request granted because it’s in the manufacturer’s best interest to do so.
The current bill doesn’t address this problem. For every person battling a terminal illness, I wish it would.
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