OLR Bill Analysis

sHB 6709



Under specified conditions, this bill allows terminally ill patients to access medications not approved for general use by the federal Food and Drug Administration (FDA). The bill applies to investigational drugs, biological products, or devices (hereinafter “investigational drugs”) that have completed Phase 1 of an FDA-approved clinical trial and are still part of the clinical trial. Among other eligibility criteria, patients must complete a detailed informed consent document.

The bill allows manufacturers to provide investigational drugs to eligible patients. If they provide these drugs, they can charge patients for them. The bill does not require health insurers to cover these drugs, and it specifies when insurers can deny coverage to patients being treated with them.

The bill specifies that it does not create a private cause of action against an investigational drug manufacturer, the treating physician, or other people or entities involved in the patient's care for any harm done by an investigational drug.

The bill prohibits the Department of Public Health or Medical Examining Board from taking any disciplinary action against a physician solely for recommending that a patient take or use an investigational drug, as long as the recommendation is consistent with medical standards of care. It also prohibits state officials, employees, or agents from preventing or attempting to prevent an eligible patient from accessing such a drug.

It is unclear how the bill interacts with federal law, which vests in the FDA the authority to approve drugs before they can be sold. The FDA also has a process to make investigational drugs available outside of a clinical trial before all phases of the trial are complete. Under this process (called “expanded access”), the patient's physician must submit the request to the FDA for approval (see BACKGROUND).

EFFECTIVE DATE: October 1, 2015


Patient Eligibility and Documentation

The bill allows terminally ill patients to receive treatment with investigational drugs under certain conditions. It defines a “terminal illness” as a medical condition that the treating physician anticipates, with reasonable medical judgment, will result in a patient's death or a state of unconsciousness from which recovery is unlikely within a year.

Under the bill, to be eligible to receive treatment with an investigational drug, a patient must:

1. have a terminal illness verified by his or her treating physician (a state-licensed physician with primary responsibility for the patient's medical care and treatment of the terminal illness);

2. not be an inpatient at a hospital;

3. consider all other FDA-approved treatment options;

4. be unable to participate in a clinical trial within 100 miles of his or her home, or not be accepted into a clinical trial no more than a week after the end of the trial application process;

5. receive a recommendation for the drug from his or her treating physician;

6. give written informed consent for the drug's use (see below); and

7. obtain from the treating physician written documentation that the patient meets requirements (3) through (6).

The required informed consent document must be verified by the treating physician and a witness. The document must be signed by the patient, except that where the patient is a minor or lacks the capacity to provide informed consent, a parent or legal guardian must consent on the patient's behalf.

The document must:

1. explain the currently approved products and treatments for the terminal illness;

2. verify that the patient agrees with the treating physician in believing that all currently approved and conventional treatments are unlikely to prolong the patient's life;

3. clearly identify the specific proposed investigational drug with which the patient is seeking treatment;

4. describe the potentially best and worst outcomes of using the drug with a realistic description of the most likely outcome, including the possibility that new, unanticipated, or worse symptoms may result and that the treatment could hasten death, based on the physician's knowledge of the treatment and awareness of the patient's condition; and

5. state that the patient understands that he or she is liable for all expenses resulting from taking the drug and that this liability extends to the patient's estate, unless a contract between the patient and the drug manufacturer provides otherwise.

The document must also make clear that:

1. the patient's health insurer, treating physician, or other providers are not obligated to pay for any care or treatment resulting from taking the investigational drug;

2. the patient's hospice eligibility may be withdrawn if the patient begins treatment with such a drug, but hospice care may be reinstated if the treatment ends and the patient is hospice eligible; and

3. in-home health care may be denied if the treatment begins.

Insurance Provisions

Under the bill, health insurers may cover investigational drugs for eligible patients but are not required to do so. While the patient is taking the drug and for the following six months, insurers may deny coverage to the patient except for (1) preexisting conditions or (2) benefits that began before treatment with the drug.

The bill specifies that (1) treatment with investigational drugs as set forth in the bill is not considered a clinical trial for purposes of the law's requirements for insurance coverage of certain clinical trial costs and (2) it does not affect those requirements.


FDA Drug Approval Process, Clinical Trials, and Expanded Access

Drug companies seeking to have a new drug approved for sale in the United States must receive FDA approval. This process involves several steps, including clinical trials. According to the FDA, Phase 1 trials focus on the drug's safety and typically involve 20 to 80 subjects. Phase 2 trials focus on effectiveness and typically involve hundreds of subjects. Phase 3 trials focus on safety, effectiveness, and related issues (such as interactions with other drugs), and involve thousands of subjects.

For individual patients seeking access to investigational drugs that have not yet received FDA approval for sale, a physician can apply to the FDA under the “expanded access” process. Federal law and regulations specify the conditions under which the FDA can grant such access. Among other things, the FDA must determine that:

1. the patient has a serious or immediately life-threatening condition and there is no comparable or satisfactory alternative therapy;

2. the potential benefit justifies the potential risks and those risks are not unreasonable in the context of the condition; and

3. providing the drug will not interfere with clinical investigations that could support marketing approval of the expanded access use or otherwise compromise the potential development of that use (21 CFR 312.305).

Even where the FDA grants approval, manufacturers are not required to provide the drug.


Public Health Committee

Joint Favorable Substitute