Access to Experimental Drugs and State “Right to Try” Laws

In November 2014, Colorado (Colo. Rev. Stat. §§ 25-45-101—108) became the first state to pass a “right to try” law giving terminally ill patients the right to try experimental drugs. Since Colorado’s law passed, several other states including Arizona (Ariz. Rev. Stat. §§ 36-1311—1314) , Michigan (Mich. Stat. § 16221), Missouri (Mo. Stat. § 191.480), and most recently Wyoming (to be codified at Wyo. Stat. §§ 35-7-1801—1806), have passed similar laws. Legislators in other states are considering similar bills to allow terminally ill patients access to experimental drugs.

“Right to Try” legislation is controversial. Supporters argue that the laws will give dying patients faster access to potentially lifesaving treatment than the Food and Drug Administration’s (FDA) compassionate use program. Detractors argue that the laws give false hope because there is no guaranteed access to the experimental treatment, no financial support, the unstudied treatments could cause harm and hasten death, and drug companies may not want to risk providing unapproved drugs and treatment for fear of angering federal regulators.

The Colorado law, which is similar to laws in other states, allows an eligible patient access to investigational drugs, biological products, or medical devices by allowing (but not requiring) doctors to prescribe and companies to provide these experimental treatments. An investigational drug, biological product, or device is defined as a drug, biological product, or device that has successfully completed phase one of a clinical trial but has not yet been approved for general use by the FDA and remains under investigation in an FDA-approved clinical trial.

Colorado defines an “eligible patient” as a person who has documentation from his or her physician that he or she meets the following requirements:

• Has a terminal illness, defined as a disease that, without life-sustaining procedures, will soon result in death or a state of permanent unconsciousness from which recovery is unlikely, as attested to by the patient’s treating physician;
• Considered all other treatment options currently approved by the FDA;
• Has been unable to participate in a clinical trial for the terminal illness within 100 miles of the patient’s home or has not been accepted to the clinical trial within one week of completing the clinical trial application process;
• Has received a recommendation from his or her physician for an investigational drug, biological product, or device; and
• Has given written, informed consent or if a minor or lacks capacity, a parent or legal guardian has given written, informed consent.

Colorado has specific requirements for written, informed consent to receive investigational drugs, biological products, or devices. Informed consent must be contained in a written document signed by the patient and attested to by the patient’s physician and a witness. At a minimum, the informed consent must contain the following:

• An explanation of the currently approved products and treatments for the disease or condition from which the patient suffers;
• An attestation that the patient concurs with his or her physician that all currently approved and conventionally recognized treatments are unlikely to prolong the patient’s life;
• A clear identification of the specific proposed investigational drug, biological product, or device that the patient want to use;
• A description of the potentially best and worst outcomes of using the investigational drug, biological product, or device, including a realistic description of the most likely outcome and the possibility that new, unanticipated, different or worse symptoms might result and that death could be hastened, based on the physician’s knowledge of the proposed treatment in conjunction with an awareness of the patient’s condition;
• A clear statement that the patient’s health insurer and provider are not obligated to pay for any care or treatments as a consequence of the investigational drug, biological product, or device;
• A clear statement that the patient’s eligibility for hospice care may be withdrawn if the patient begins curative treatment but that hospice may be reinstated if the curative treatment ends and the patient meets hospice criteria;
• A clear statement that in-home health care may be denied if treatment begins; and
• A statement that the patient understands that he or she is liable for all expenses as a consequence of using the investigational drug, biological product or device, and that this liability extends to the patient’s estate, unless a contract between the patient and the manufacturer state otherwise.

Finally, there are several other interesting provisions in Colorado’s law. The law prohibits a licensing board from revoking, failing to renew, suspending, or taking any action against a health care provider’s license based solely on the provider’s recommendation to an eligible patient concerning access to or treatment with an investigational drug, biologic product, or device as long as the advice is consistent with medical standards of care. In addition, the law clarifies that the Colorado statute does not create a private cause of action unless there was a failure to exercise “reasonable care.” Thus, the statute does not grant immunity to a manufacturer or any person or entity involved in the care of an eligible patient from a negligence lawsuit.

While the “right to try” laws codify that the state will not interfere with a terminally ill patient’s access to experimental treatments, the laws cannot eliminate the FDA’s authority to regulate these treatments. To date, the FDA has not taken an official position on state right to try laws. However, the FDA recently decided to streamline the process for patients to obtain access to unapproved investigational drugs. On February 4, 2015, the FDA released a draft guidance that when finalized is intended to streamline the individual patient expanded access application process. In addition, there is a provision that would allow emergency expanded access for an individual patient by allowing an FDA official to give authorization for emergency use over the telephone.

It is likely that there will be new developments in right to try legislation as the year progresses. Health care providers and drug manufacturers should pay attention to these developments, particularly the FDA’s actions.

Image courtesy of Flickr by epsos .de