“Right To Try” and Ortho

The new “Right To Try” legislation signed into law by the President on May 30, 2018, will generally impact orthopedic providers if they have patients who have been diagnosed with a life-threatening disease or condition, have exhausted all approved treatment options, must be unable to participate in a clinical trial involving the investigational drug, and must provide informed consent.

Specifically, how orthopedic surgeons will be impacted is still a little unclear. An American Academy of Orthopaedic Surgeons (AAOS) spokesperson told us that the organization “has no position on ‘Right To Try’ and continues to be supportive of the FDA and advocate only for approved devices and medications.”

The law gives terminally ill patients the right to seek drug treatments that remain in clinical trials and have passed Phase 1 of the FDA’s approval process but have not been fully approved by the FDA.

“Right-To-Try” laws already exist in 38 states and the FDA has an existing expanded access program, through which clinical trial sponsors and health care providers can provide access to investigational drugs for serious diseases or conditions upon FDA approval.

The FDA says it tries to respond to requests for the current expanded use within hours and approved 1,831 out of 1,862 requests in 2017. But the GAO reported that some stakeholders found the FDA’s forms difficult to understand.

Dr. Arthur L. Caplan, a bioethicist at New York University reportedly said the bill, “does somewhere between nothing and absolutely nothing to help you.” That’s his opinion because he says companies are reluctant to allow their unapproved drugs to be used even if the FDA has granted a patient permission.

Prerequisites for Use

According to Phoebe Mounts, Kathleen Sanzo and Jacqueline Berman of the law firm of Morgan Lewis & Bockius LLP, (Morgan Lewis) the particular investigational drug must also meet certain prerequisites.

A phase one clinical trial must be complete; the drug must not be approved for any use by the FDA; either an FDA marketing application must be submitted, or the drug must be under investigation in a clinical trial that is intended to form the primary basis of an efficacy claim and subject to an active IND application; and an active drug development or production program must be ongoing and not discontinued or placed on clinical hold.

Eligible investigational drugs that are provided to eligible patients are exempt from requirements for FDA approval, INDs and most of the corresponding IND regulations, certain labeling requirements including adequate directions for use, and the FDA’s regulations on informed consent and institutional review board, or IRB, approval.

Action Items for Providers

While the impact of the new law is unclear for now. The lawyers from Morgan Lewis say there are actions clinical trial sponsors and health care entities can take. Here are some of the things they say sponsors should consider:

• Sponsors should develop a clear internal policy on their approach to requests for access. This policy should include whether and how access will be provided, and how requests for access will be handled (e.g., who is authorized to handle such requests, how requests should be routed and what responses may be provided).
• Sponsors should have a procedure for how access decisions will be made and what information is required. The applicable information may include information on the patient, patient’s condition and patient’s treatment.
• All persons and entities involved in clinical trial and development programs should be trained on these policies, procedures and requirements.
• Sponsors should also ensure that there is a clear understanding of differences between federal law, the FDA’s expanded access regulations and the individual state requirements concerning treatment use of investigational products. States in which clinical trial sites are located may be likely targets for access requests from patients.

To read the entire Morgan Lewis recommendations, click here.