June 8, 2018

Right to Try Law Enacted by Congress: Implementation Unclear

Holland & Knight Healthcare Blog
Michael J. Werner

On May 30, 2018, President Donald Trump signed the Trickett Wendler, Frank Mongiello, Jordan McLinn and Matthew Bellina Right to Try Act of 2018. The bill allows the provision of certain unapproved, investigational drugs to patients with life-threatening diseases who have exhausted approved treatment options and are unable to participate in a clinical trial involving the drug. Similar legislation has been enacted in 40 states.

Under the act, terminally ill patients can request a drug manufacturer to provide an experimental product so long as: it has completed Phase I clinical testing; the patient has provided written informed consent to receive the treatment; and a physician certifies the patient has exhausted approved treatments and is ineligible for the clinical trial. In so doing, the act creates a new pathway whereby patients may be able to receive unapproved products outside the clinical trial process without Food and Drug Administration (FDA) or Institutional Review Board (IRB) oversight.

The law's supporters have argued that this will improve the speed with which seriously ill patients can access investigational products, while opponents have criticized right to try laws as offering patients false hope and increasing risks. Note that the act explicitly does not require manufacturers to provide experimental treatments.

Many questions remain, however, about implementation of the Right to Try Act. For example, Dr. Janet Woodcock, Director of FDA's Center for Drug Evaluation and Research, suggested that the agency would issue guidance and urged FDA staff not to provide advice to patients and drug manufacturers until the agency "develops further information." The chief Senate sponsor of the legislation has taken the opposite perspective and argued that FDA guidance and regulations are unnecessary saying: "This law intends to diminish the FDA's power over people's lives, not increase it."

On June 1, FDA announced that it is putting together an internal group that will determine how the agency should move forward with implementing the act.

In the interim, manufacturers, research institutions and other stakeholders will need to determine answers to questions such as:

  • How this program will relate – if at all – to existing early access (sometimes called "compassionate use") programs?
  • What should be the contents of informed consent under right to try?
  • What roles should IRBs play even if not required by the Act?
  • Is the company subject to a state right to try law?

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