A Pennsylvania "Right-to-Try" bill that would allow eligible terminally-ill patients to use investigational drugs, biological products, and medical devices not yet approved by the U.S. Food and Drug Administration, was signed into law on by Gov. Tom Wolf on Oct. 11, 2017.
The legislation was introduced by Rep. Robert Godshall (R-53rd District). Pennsylvania has become the 38th state to enact a Right-to-Try law.
1. What is the purpose of this law?
This law allows terminally ill patients to seek access to potentially life-saving investigational drugs, biological products and medical services.
2. When does this law take effect?
The law takes effect 60 days after the governor signed the bill into law.
3. Who is eligible to use the products?
To be eligible to use investigational drugs, biological products or medical services under this law, an individual must be deemed an "eligible patient." An "eligible patient" is defined as an individual who has:
(1) A terminal illness, attested to by the patient's treating physician;
(2) Carefully considered all other treatment options approved by the Federal Food and Drug Administration;
(3) Been unable to participate in a clinical trial for the terminal illness that is located within 100 miles of the patient's home address or has not been accepted to the clinical trial within one week of completion of the clinical trial application process;
(4) Received a recommendation from the patient's treating physician for an investigational drug, biological product or medical device;
(5) Given written, informed consent for the use of the investigational drug, biological product or medical device, or if the patient is either a minor or lacks the mental capacity to provide informed consent, a parent or legally authorized representative has given written, informed consent on the patient's behalf; and
(6) Documentation from the patient's treating physician that the patient meets the above requirements.
4. Are there any patients that are ineligible under this law?
Yes. An "eligible patient" does not include an individual being treated as an inpatient in any hospital.
5. What is an "investigational drug, biological product or medical device"?
These terms are defined as "a drug, biological product or medical device that has successfully completed phase one of a clinical trial but has not yet been approved for general use by the Federal Food and Drug Administration and remains under investigation in a clinical trial approved by the Federal Food and Drug Administration."
A phase one clinical trial is performed with a small group of people, who may be healthy volunteers, to test drug safety and safe dosage range. It is not necessarily performed with patients who have the disease the patient seeks to treat.
6. Who is considered a "terminally ill" patient?
An individual who has a "disease or condition that, without life-sustaining procedures, will soon result in death or a state of permanent unconsciousness from which recovery is unlikely."
7. What is considered "written, informed consent" for a patient?
A patient is required to give written, informed consent prior to using an investigational drug, biological product or medical device. There are several parameters that must be met by a physician in providing information to a patient for a patient to give this type of consent. Under the law, "written, informed consent" is considered "a written document placed in the patient's medical record signed by the patient and attested to by the patient's treating physician and a witness that, at a minimum:
(1) Explains the currently approved products and treatments for the disease or condition from which the patient suffers.
(2) Attests to the fact that the patient concurs with the patient's treating physician in believing that all currently approved and conventionally recognized treatments are unlikely to prolong the patient's life.
(3) Identifies clearly the specific proposed investigational drug, biological product or medical device that the patient is seeking to use.
(4) Describes the potentially best and worst outcomes of using the investigational drug, biological product or medical device with a realistic description of the most likely outcome, including the possibility that new, unanticipated, different or worse symptoms might result, and that death could be hastened by the proposed treatment, based on the treating physician's knowledge of the proposed treatment and the patient's condition.
(5) Makes clear that the patient's health insurer and health care provider are not obligated to pay for the use of the investigational drug, biological product or medical device or any care or treatments consequent to the use of the investigational drug, biological product or medical device.
(6) Makes clear that the patient's eligibility for hospice care may be withdrawn if the patient begins curative treatment and care may be reinstated if the curative treatment ends and the patient meets hospice eligibility requirements.
(7) Makes clear that in-home health care may be denied if treatment begins.
(8) States that the patient understands that the patient is liable for all expenses consequent to the use of the investigational drug, biological product or medical device, and that this liability extends to the patient's estate, unless a contract between the patient and the manufacturer of the investigational drug, biological product or medical device states otherwise."
8. Is there a standardized form that can be used to obtain a patient's written, informed consent?
At this time, no. If a Commonwealth agency develops a standardized form that may be used by physicians, PAMED will notify its members.
9. Are insurance companies required to cover any costs under this law?
Other than any costs that are a covered benefit under an eligible patient's health insurance policy, the law does not require a health insurer to provide coverage for any health care services, including investigational drugs, biological products or medical devices, including any adverse effects that may result from the use of these products. In addition, the law places no legal obligations on insurers to pay for the routine costs of care associated with delivery of investigational treatment and if complications arise from an investigational treatment. Any individual seeking access to investigational drugs, biological products or medical devices should speak to their health insurance company to determine if these products, including any adverse effects that may result from using these products, will be covered and to what extent.
10. What are the potential costs for obtaining these products from the manufacturer?
A manufacturer of an investigational drug, biological product or medical device is authorized under the law to provide these products without receiving compensation. However, manufacturers are also authorized to require an eligible patient to pay the costs of, or the costs associated with, the manufacture of the investigational drug, biological product or medical device.
Be aware that a manufacturer of these products is not required to provide access to these products. What this law authorizes is patients being able to seek access to these products, and if access is successful, to use them if the requirements of the law are met.
11. Are physicians required to participate under this law?
12. If a physician does participate, is there any type of immunity since these products are not yet federally approved?
Yes. If a physician exercises reasonable care in recommending or participating in the use of any of these products, and follows the requirements under the law, a physician may not be subject to criminal or civil liability, nor be found to have committed an act of unprofessional conduct under any law of the Commonwealth relating to licensure.
A licensure board may not take action against a licensee based solely on the licensee's recommendations to an eligible patient regarding access to, or treatment with, an investigational drug, biological product or medical device, if the recommendations are consistent with medical standards of care. In addition, a licensure board may not take action against a licensee solely for participating in the use of an investigational drug, biological product or medical device in good faith and in accordance with this law.
13. May a patient sue a physician for any injuries suffered from use of these products?
The law specifies that nothing contained in the law may be construed as creating a private cause of action against a person or entity (including a manufacturer) involved in the care of an eligible patient using an investigational drug, biological product or medical device for any injury suffered by the eligible patient resulting from the investigational drug, biological product or medical device, as long as that person or entity acted in accordance with the law, except when the injury results from a failure to exercise reasonable care.
14. Can physicians still use the FDA's expanded access program – also known as "compassionate use" – to submit a request for a patient to use an investigational product outside of a clinical trial?
Yes, physicians can still request expanded access for an individual patient through the FDA's application process. The FDA says it authorizes 99 percent of expanded access requests it receives. Get details on the FDA's expanded access webpage.