ASCO Supports FDA's Expanded Access Program, Wary of Right to Try

Thirty-three states in the United States have passed a right-to-try (RTT) law, which bypasses the FDA to provide access to phase 1 drugs for patients who are terminally ill. However, the American Society of Clinical Oncology (ASCO) has released a statement raising serious concerns about RTT while strongly supporting the FDA’s expanded access program.

In a position statement released today, ASCO said these measures lack adequate patient protections and do not remove any of the major barriers patients face in accessing investigational drugs outside of clinical trials.

“ASCO supports access to investigational drugs outside of clinical trials when adequate patient protections are in place,” said ASCO Chief Medical Officer Richard L. Schilsky, MD, FACP, FASCO. “We don't support right to try legislation, however, because these laws ignore key patient protections without actually improving patient access to investigational drugs outside of clinical trials.”

Also called “compassionate use,” the expanded access program allows use of investigational drugs, yet to be FDA approved, outside of a clinical trial, when a patient may not be eligible to participate in an ongoing trial. For a patient to qualify for expanded access, the FDA has laid out the following requirements:

• The patient and a licensed physician are both willing to participate
• The patient’s physician determines that there is no comparable or satisfactory therapy available to diagnose, monitor, or treat the patient’s disease or condition
• That the probable risk to the person from the investigational product is not greater than the probable risk from the disease or condition
• The FDA determines that there is sufficient evidence of the safety and effectiveness of the investigational product to support its use in the particular circumstance
• The FDA determines that providing the investigational product will not interfere with the initiation, conduct, or completion of clinical investigations to support marketing approval
• The sponsor, the clinical investigator, or the patient’s physician in the case of a single patient expanded access request, submits a clinical protocol that is consistent with the FDA’s statute and applicable regulations for investigational new drugs (INDs) or investigational device exemption applications describing the use of the investigational product
• The patient is unable to obtain the investigational drug under another IND or to participate in a clinical trial

In its statement, ASCO states that delays during the expanded access application process—such as a sponsor’s willingness to provide the investigational drug—are a reality that patients and providers are exposed to. However, the recently approved 21st Century Cures Act has provisions to this end, including forcing drug manufacturers to make their expanded access policies public knowledge and the length of time the manufacturer would need to acknowledge receipt of the patient’s request.

The Trickett Wendler Right to Try Act of 2017 requires federal government to allow unrestricted manufacturing, distribution, prescribing, and dispensing of experimental drugs, biological products, and medical devices that are intended to treat a patient who has been diagnosed with a terminal illness, and is authorized by state law. While RTT laws vary by state, the conditions usually state:

• That the patient have a terminal disease
• Patients have considered, but not necessarily received, FDA approved options
• Patients have a prescription from a physician
• Patients have signed an informed consent document

Under the bill, the FDA is required to report to Congress on efforts made to streamline patient access to the treatments, and that the product should have completed a phase 1 trial.

ASCO believes that this law does not improve access or afford sufficient protection to patients because of:

• Lack of an enforcement mechanism to provide patient access, since they do not require or compel drug manufacturers to provide investigational products
• Lack of legal obligations on insurers to pay for the routine care costs associated with delivery of treatment, particularly in the case of complications caused by these drugs—unlike coverage requirements that exist for patients who participate in clinical trials
• Lack of FDA involvement, unlike under the expanded access program, which is important for patient safety

ASCO believes that provisions within the 21st Century Cures Act will provide access to such INDs while ensuring patient safety. The statement also introduces the concept of developing a navigation service to assist patients with their application process, such as an online portal or tool.

“New policy initiatives should focus on increasing transparency among pharmaceutical manufacturers' expanded access policies while ensuring that existing expanded access programs are timely and efficient for patients and their physicians,” Schilsky said.