Colorado becomes first state to give terminally ill patients options and access to investigational treatments
by Susan Koontz, JD, CMS General Counsel Jean Martin, MD, JD, COPIC Legal Counsel
Featured in the September/October 2014 Colorado Medicine.
Colorado recently became the first state to enact legislation intended to allow terminally ill patients access to investigational drugs, biological products and devices (“investigational treatments”) that have not yet completed clinical trial and been approved for general use by the U.S. Food and Drug Administration. Colorado House Bill 14-1281, titled “Access to Treatments for Terminally Ill Patients” and known as the “Right To Try Act,” was passed unanimously by the Colorado legislature and signed into law by Governor John Hickenlooper on May 17, 2014.
One of the co-sponsors of the bill, Representative Joann Ginal, (D-Fort Collins), described the bill in this way: “The Right to Try bill is a choice that terminally ill patients may be able to qualify for when all other methods to help are exhausted. It is an option other than death. It is a bill of hope, when all else has failed.”
Despite the passage of this new state law, its interaction with matters subject to federal jurisdiction and the potential consequences for physicians who seek to act under its provisions are currently unclear.
The Right To Try Act applies to treatments that have successfully completed Phase 1 of a clinical trial but have not been approved by the FDA for general use and remain under investigation in a FDA-approved clinical trial. Although current federal law permits the FDA to allow manufacturers to provide investigational treatments to terminally ill patients outside of clinical trials on a case-by-case basis, supporters of Colorado’s Right To Try Act maintain the FDA’s approval process is too lengthy and complex, with only a small number of patients (under 600 in 2013) successfully gaining access to investigational treatments through the FDA.
At the outset, it is important to understand that the Right To Try Act does not require a physician to recommend an investigational treatment not yet approved by the FDA for general use. A physician should always exercise his or her independent medical judgment in recommending any treatment for a patient, whether investigational or not. Rather, the Act provides authorization under Colorado law for a physician to recommend an investigational treatment to 1) a terminally ill patient 2) who has considered all other treatment options currently approved by the FDA and 3) is unable to participate in a clinical trial located within 100 miles of their home. Inpatients at Colorado licensed hospitals are excluded from eligibility for investigational treatments under the Right To Try Act.
The Right To Try Act “authorizes” physicians to recommend investigational treatments pursuant to Colorado law in three ways. The Act 1) precludes a state licensing board from taking action against the physician solely for recommending an investigational treatment, 2) precludes action against a physician’s Medicare certification solely for recommending an investigational treatment, and 3) states that the Act does not create a private right of action against a physician for harm caused to a patient as a result of the use of an investigational treatment. However, the Act is clear that physicians are still bound to act reasonably and in accordance with accepted medical standards of care. This begs the question: What are the accepted medical standards of care regarding the use of an investigational treatment not approved for general use by the FDA? One can imagine a patient’s family contending that a physician’s knowing recommendation of an investigational treatment in contravention of current federal laws and regulations is per se unreasonable and not in accord with accepted medical standards of care. Such a question may ultimately be left to a civil jury to resolve.
“Physicians prescribing experimental treatment under the new law will want to strictly comply with the law’s requirements and be aware that a reasonableness standard applies,” said CMS outside counsel John L. Conklin, JD.
Potential professional liability
Under the Supremacy Clause of the U.S. Constitution, state laws are void if they conflict with valid federal statutes and stand in obstacle to the accomplishment and execution of Congress’s full purposes and objectives. Colorado’s Right To Try Act does not set aside federal law and regulations regarding access to investigational treatments or limit the FDA’s exclusive jurisdiction to regulate drugs and biological products and devices. Thus the Right To Try Act does not insulate physicians from potential repercussions at the federal level. Furthermore, the Right To Try Act may be subject to a future constitutional challenge on the grounds that it conflicts with a matter of exclusive federal regulation in contravention of the Supremacy Clause. Although the validity of Colorado’s Right To Try Act has not yet been challenged in the legal system, the FDA has issued a statement expressing concern about state efforts that may undermine its “Congressionally mandated authority and agency mission to protect the public from therapies that are not safe and effective.”
Given the uncertain legal landscape and concurrent but differing state and federal laws and regulations, prudence dictates physicians adhere to both state and federal requirements concerning access to investigational treatments. Physicians considering treating a patient with an investigational drug outside of an approved protocol should discuss this with their malpractice insurance carrier. The professional liability policy may have an exclusion for using a drug that is not approved by the FDA for any purpose. Physicians should also be aware that this issue is subject to further legal developments. Possible examples would include Colorado’s Medical Board or Department of Health issuing rules or adopting policies concerning the implementation of the Right To Try Act; Colorado enacting additional state laws clarifying the Right To Try Act; or the FDA or other federal agencies issuing regulations concerning investigational treatments.
To make a recommendation of an investigational treatment, the Right To Try Act requires a physician certify in writing that the patient meets the requirements for eligibility of the Act. The Act also requires that a physician obtain written informed consent from the patient that includes: 1) an explanation of the currently approved treatments; 2) an attestation that the patient agrees with the physician that currently approved treatments are unlikely to prolong the patient’s life; 3) a clear identification of the specific proposed investigational treatment; 4) a description of the best, worst and most likely outcomes of the proposed investigational treatment, including the possibility that new, unanticipated, different or worse symptoms could arise and death could be hastened; 5) an explanation that a health insurer is not obligated to pay for care and treatment subsequent to an investigational treatment; 6) a statement that the patient’s eligibility for hospice care can be withdrawn if the patient begins an investigational treatment; 7) a statement that in-home health care may be denied if the patient begins an investigational treatment; and 8) a statement that the patient and his or her estate are liable for health care expenses consequent to the use of an investigational treatment.
The Right To Try Act expressly allows health insurers to discontinue coverage of benefits related to the investigational treatment from the time the patient starts an investigational treatment until six months after the end of the investigational treatment. Although the Act does not require a health insurer to deny coverage and requires the maintenance of coverage for pre-existing conditions, the potential financial ramifications of instituting an investigational treatment suggest that a physician exercise caution and spend considerable time counseling the patient to ensure the patient is aware and informed about the financial risks.
Although the Right To Try Act is silent with regard to the specialization of a recommending physician, the requirement that written informed consent contains a description of the worst, best and most likely outcomes suggests that appropriate specialist consultation and participation is a necessary part of obtaining the required informed consent. Finally, the Act does not require manufacturers to provide patients access to investigational treatments upon a physician’s recommendation. A physician should be aware that a manufacturer may not be incentivized to endanger their investment in the FDA approval process by distributing an investigational treatment that is still undergoing clinical trial and has not yet received final approved from the FDA.
Colorado’s Right To Try Act stands at the forefront of a novel state law movement seeking to expand patient access to investigational treatments. Accordingly, physicians must act cautiously and give due consideration to the potential risks of recommending an investigational treatment. At a minimum, CMS urges its members to ensure they comply with all applicable state and federal laws and regulations in considering recommending an investigational treatment.