Will Controversial Bill Cure or Kill Patients?


In 2016, 10-year-old Joshua Hardy lost his long battle with cancer, in part because of a viral infection that resulted from a bone marrow transplant. Hardy’s family tried to get access to a new experimental antiviral drug from Chimerix, the company developing it. The company refused, not wanting to divert time and resources from its efforts to gain FDA approval for the drug. It agreed to make the drug available only after a social media campaign.

Hardy responded well to the drug, but it was too late.

Hardy’s story is one of those featured in the lobbying campaign for a federal Right to Try (RTT) bill, which passed the Senate in August and may soon get a vote in the House.

But the Senate-passed bill would not require drug companies to provide dying patients access to their experimental drugs. Instead, it largely goes after the Food and Drug Administration, and would remove FDA oversight from the process, while insulating the pharmaceutical industry and doctors from virtually any liability if patients are harmed.

RTT pits some desperate patients, along with libertarians and conservative and anti-regulation Republicans, including President Donald Trump and Vice President Mike Pence, against public health advocates, medical ethicists, and some national patient-advocacy groups.

The bill’s supporters frame the legislation as striking a blow for the rights of patients to try any drug without government interference. They predict that more pharmaceutical companies would give patients access to their unproven drugs if they weren’t worried about the FDA looking over their shoulders.

The bill’s opponents contend that any possible expansion of access comes at the cost of eliminating crucial protections for the most vulnerable patients, potentially hastening their deaths and making their end more painful.

Opponents are circulating a letter to the House this week charging that RTT should actually be called the “False Hope” bill. Indeed, given the philosophical bent of its strongest supporters, and their connection to free-market crusaders Charles and David Koch, RTT may be more about advancing a conservative, anti-government ideology than helping the terminally ill. […]

Diana Zuckerman, president of the National Center for Health Research, points out that drug companies rely on a three-phase clinical trial process to gain FDA approval for their products. If RTT siphoned off patients with life-threatening illnesses from clinical trials, particularly for trials of medications serving limited populations with rare diseases, drug companies might find it even harder to enroll enough patients to complete them. And health insurers will not pay for drugs that lack FDA approval. […]

RTT’s true beneficiaries may be new drug startups that are neither “patient-oriented nor compassionate, trying to create buzz for their product,” Zuckerman said. A biotech startup, she speculated, might be struggling with the high costs of making its product, and be happy to find wealthy, desperate patients willing to pay the freight. This might be particularly attractive since RTT gives patients only limited rights to sue, even if they are seriously harmed by defective products.

Unscrupulous doctors also might benefit, she added, since the law does not place any restrictions on the fees they may charge patients to administer the drug and monitor their progress. […]

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