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From Health Law Daily, May 23, 2018

Congress gives patients the ‘Right to Try’ experimental drugs, bill heads to White House

By Harold Bishop, J.D.

Congress has passed "Right to Try" legislation that will require the federal government to allow unrestricted manufacturing, distribution, prescribing, and dispensing of experimental drugs and biologics that are intended to treat a patient who has been diagnosed with a terminal illness. The legislation does not require manufacturers to provide experimental products to patients who request them.

The House passed the legislation, known as the Trickett Wendler, Frank Mongiello, Jordan McLinn, and Matthew Bellina Right to Try Act of 2017 (S. 204), by a vote of 250 to 169. Twenty-two Democrats voted for the bill, joining 228 Republicans. The bill was unanimously passed by the Senate on August 3, 2018. On March 3, 2018, the House passed its own version of Right to Try (H.R. 5247), but it lost traction in the Senate. The White House requested action so the House decided to take up the previously passed Senate version.

Forces behind the legislation. Right to Try, or expanded access to investigational drugs by individual patients, has been spurred by a number of factors, including: (1) the enactment of the 21st Century Cures Act (P.L. 114-255), which requires public posting of expanded access policies (also known as "compassionate use") by drug sponsors; (2) reports in the media of patients in need of access to investigational drugs; and (3) corresponding social media campaigns.

The Goldwater Institute, a public interest nonprofit organization that advocates for open and transparent government, has effectively advocated for state Right to Try laws. In fact, according to the FDA, "the Goldwater Institute is behind an effort [emphasis added] to get states to adopt ‘Right to Try’ laws, which grant terminally ill patients access to drugs that have been approved by the FDA for clinical trials but may be years away from hitting the market."

In response to these factors, there have been 40 state Right to Try laws enacted to date. All these actions seem to have had a demonstrable effect on drug manufacturers. According to experts from Avalere Health, the percentage of pharmaceutical companies clearly publishing a compassionate use policy on their websites more than doubled, from 19 percent to 47 percent, from September 2016 to March 2017.

What the legislation provides. S. 204 provides that:

  • The federal government must allow unrestricted possession and use of expanded access treatments by patients certified by a physician as having exhausted all other treatment options.
  • A manufacturer, distributor, prescriber, dispenser, possessor, or user of such a treatment has no liability regarding the treatment, unless their conduct constitutes reckless or willful misconduct, gross negligence, or an intentional tort under state law.
  • The outcome of manufacture, distribution, prescribing, dispensing, possession, or use of expanded access treatment may not be used by the FDA to adversely impact review or approval of the treatment, unless the FDA makes a determination that use of such clinical outcome is critical to determining the safety of the eligible investigational drug or the sponsor requests use of such outcomes.
  • The treatment must: (1) have successfully completed a phase 1 (initial, small scale) clinical trial; (2) remain under investigation in a clinical trial approved by the FDA; and (3) not be approved, licensed, or cleared for sale.
  • The bill requires the manufacturer or sponsor of an investigational drug to submit to the Secretary of HHS an annual summary of any expanded use of the drug under Right to Try. The annual summary must include the number of doses supplied, the number of patients treated, the uses for which the drug was made available, and any known serious adverse events.

Finally, a sponsor, manufacturer, prescriber, dispenser or other entity may not be held liable for its determination not to provide expanded access to an eligible investigational drug.

Problems with the legislation. David Farber, a partner in the Washington, D.C. law firm King & Spalding, believes that "the legislation still makes a mistake in keeping the FDA out of the picture before the expanded access medication is dispensed, which keeps an important actor with valuable medical information about the unapproved drug from adding their voice to the process." Farber points to a January 10, 2018, New England Journal of Medicine (NEJM) article, entitled "Federal Right-to-Try Legislation – Threatening the FDA’s Public Health Mission," in support of his concerns about S. 204. According to the NEJM article, the bill’s critics raise two main concerns:

"First, despite the claim that dying patients have nothing to lose, granting very sick patients early access to unapproved products may be more likely to harm patients than to help them. Many drugs that look promising in early development are ultimately not proven safe or efficacious. There is a low probability of benefit, but a risk of worsened quality of life or accelerated death, from drugs that have progressed only through phase 1 trials—especially for frail patients or those with advanced disease. In addition, publicly available data are limited early in development; prescribers will therefore lack the evidence and expertise to mitigate the risks."

"The second concern is that, unless the requirement that patients be unable to participate in a clinical trial is interpreted strictly, expanding access outside trials may delay the generation of data needed to make evidence-based decisions about approval and use of new drugs. Even if access to investigational drugs is limited to patients who truly could not participate in trials testing them, the diversion of resources toward expanded access could have serious implications for the much larger number of patients who would benefit from expeditious approval of effective drugs."

The authors of the NEJM article concluded that in reality the bill would probably have minimal effect.

Farber also points out that FDA already approves more than 99 percent of the expanded access requests it receives and the Senate bill may cause patients to lose the benefit of meaningful safety-oriented changes that the FDA recommends in 10 percent of expanded access approvals on the basis of confidential information that is often unavailable to treating physicians (see Compassionate use: A look into Goldwater Institute v. HHS and ‘Right to Try’ legislation, February 1, 2018).

Amy Dow, a member in the Chicago law office of Epstein Becker & Green, P.C., has stated that while S. 204 would address some of the challenges that limit patients’ access to investigational products, it is not "a panacea."

According to Dow, "many of the more significant barriers to increasing access to investigational products cannot be adequately addressed by Right to Try legislation, as these bills do not obligate manufacturers to make investigational products available to patients or allocate funding to address the substantial costs associated with requests for access by individuals." As a result, Dow believes that "S. 204 is not likely to overcome challenges resulting from manufacturers’ often limited supplies of investigational products and the burdens imposed on manufacturers and their personnel, who may be operating with limited resources, to fulfill requests from patients for access to investigational products." She is also concerned that "S. 204 also does nothing to overcome ethical concerns regarding the diversion of resources, in the form of product, personnel and funds, from efforts to make a product available to all patients who could benefit from it to an individual patient."

Dow concludes "while the added protections from liability and regulatory burdens provided by S. 204 may be sufficient to convince some manufacturers to approve a greater number of requests for access to investigational products, I anticipate that many, particularly smaller manufacturers, will continue to limit approval of these requests."

For a full discussion of drug manufacturer concerns, see Individual compassionate use: concerns for drug manufacturers considering participation, April 17, 2015.

Companies: Avalere Health

MainStory: TopStory FederalLegislationNews HouseNews StateLegislationNews DrugBiologicNews FDCActNews ClinicalNews PLDrugNews RiskNews SafetyNews

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