Right to Try - Unapproved Drugs or Biologics
In May 2018, the Federal Right to Try (RTT) Act was signed into law, creating a federal framework for patients to access investigational new drugs and biologics outside of clinical trials and outside of the U.S. Food and Drug Administration’s (FDA) expanded access program. The federal law enables manufacturers and physicians to provide investigational drugs to eligible patients without risk of liability. It follows California’s passage of the State’s Right to Try Act, signed into law in 2016. Similar to the federal law, the California law enables manufacturers and physicians to provide investigational products to eligible patients without risk of liability under state law. The state law also prohibits California medical boards from taking disciplinary action against physicians.
Given the stricter California law requirements for using an investigational drug without FDA approval and the additional protection the California law affords to physicians regarding licensure, UCI will comply with California’s RTT law requirements. Because the federal RTT law does not address the use of investigational devices UCI will not provide investigational devices to patients outside of the FDA’s expanded access program.
Both the federal and state RTT laws enable patients meeting certain criteria under each law to receive access to investigational test articles without FDA oversight. However, the laws differ in fundamental ways that should be considered before providing an investigational product to a patient without FDA authorization.
Federal Right to Try Act
The Federal Right to Act enables a patient with a “life-threatening” disease or condition to access investigational drugs and biologics that have completed Phase 1 testing under an FDA-approved clinical trial and which are either being actively developed or produced by the manufacturer or not placed on clinical hold. Importantly, the federal law does not address the use of investigational devices.
In order to receive an investigational drug or biologic under the federal RTT Act, the patient must:
- Have a life-threatening disease or condition;
- Have exhausted treatment options, as certified by the physician;
- Be unable to participate in a clinical trial involving the investigational drug, as certified by the physician; and
- Have given his or her written informed consent (or consent of a legally authorized representative) to the treating physician regarding use of the investigational drug.
Use of an investigational drug under the RTT is exempt from FDA requirements for review and authorization, so long as the sponsor or manufacturer of the drug is in compliance with FDA requirements applicable to investigational drugs. Note that the law does not limit compliance to the specific investigational drug that is the subject of the use.
The RTT act does require sponsors and manufacturers who have made their investigational drugs available under RTT to annually report to the FDA 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. In turn, the FDA must make this information publicly available on its website.
Patients may be charged the direct costs of making the investigational drug available for their use. The federal RTT does incentivizes sponsors and manufacturers to make their investigational drugs available by protecting them against liability with respect to acts and omissions regarding the investigational drug. The Act also protects prescribers, dispensers, and other individuals from liability, unless the act or omission constitutes reckless or willful misconduct, gross negligence, or an international tort under state law. Finally, the Act expressly protects against liability to any person for not providing access to an investigational drug under the Act.
California Right to Try Act
The California Right to Try (RTT) Act is similar to the federal Act, though there are notable differences.
In one respect, the California law is broader than the federal right to try law as it applies to use of investigational drugs, biologics and devices that have successfully completed an FDA-regulated Phase 1 trial and remain under active investigation by the FDA. The federal law does not include devices.
In most instances, the California law imposes more requirements to obtain access to an investigational drug outside of a clinical trial.
- The state RTT limits access to patients with an “immediately life-threatening disease or condition”, whereas the federal law only requires patients have a “life-threatening disease or condition.” Thus, under state law, the patient must be in a stage of disease in which there is a reasonable likelihood death will occur in a matter of months.
- The treating physician and a second consulting physician must both recommend that the patient receive the investigational product, attest to this recommendation, and attest that the patient meets the criteria of the state law.
- Specific informed consent requirements must be met. Like the federal law, a surrogate may consent on behalf of the patient consistent with California law requirements. However, the consent form must contain the information from the California Health and Safety Code - Section 111548.1(h)(1), and also must meet the requirements set forth in the California Protection of Human Subjects in Medical Experimentation Act.
- An IRB must review and approve the protocol and consent form.
Reporting requirements between the federal and California RTT Acts also differ. While federal law requires that the sponsor or manufacturer make information available to the FDA (which the FDA must publicly post), the California law imposes an obligation upon the IRB of record to biannually report information regarding the number of requests made to the IRB for an investigational product, the status of each request, the duration of treatment, the costs of treatment paid by patients, the success or failure of the investigational product in treatment, and adverse events.
Similar to federal law, California RTT does not impose an obligation upon manufacturers to make an investigational product available to a patient. California RTT also provides that a manufacturer may recover the costs of the manufacture of the product. However, the law removes any liability upon the patient’s heirs or the patient’s health benefit plan, for any outstanding debt related to the treatment using the investigational product.
In addition, the CA RTT specifically prohibits the Medical Board of California and the Osteopathic Medical Board of California from taking any disciplinary action against a physician’s license to practice medicine based solely upon the physician’s recommendation to treat or treatment of a patient with an investigational product, so long as the protocol was approved by an IRB. The Act also provides that any actions taken pursuant to the state law by a manufacturer or any other person or entity involved in caring for the patient cannot serve as the basis for any civil, criminal or disciplinary claim or cause of action under state law.
Procedures for IRB submission of Right to Try requests for unapproved drugs or biologics
Right to Try protocols must be submitted per the usual IRB process and require IRB review and approval. Provide the following information:
• the Treatment Plan, include the cost of treatment
• Draft a Consent Form
• Signed Treating Physician Attestation
You will also need to secure a signed Consulting Physician Attestation before treatment begins. Provide a copy of the signed attestation to the IRB within 5 days from the beginning of treatment. Please see the UCI Right to Try Attestation and Consent Template.
Other Treating Physician Responsibilities:
- Consult with Clinical Trials Team in Sponsored Projects Administration to determine whether an agreement is necessary with the sponsor/manufacturer.
- Consult with UCI Health Research Revenue Integrity (RRI) to determine billing implications.
- Consult with Investigational Drug Services pharmacy to determine drug requirements.
- Ensure patient understands financial and health care considerations outlined in consent form.
- Register the patient in OnCore, as determined by RRI.