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Patients demand access to investigational therapies


Expanded access or “compassionate use” programs allow patients to use investigational treatments, medical devices, or tests, before they have received FDA approval.

Expanded access or “compassionate use” programs allow patients to use investigational treatments, medical devices, or tests, before they have received FDA approval. By definition, these agents and devices have not yet been deemed proven to be safe or effective by FDA.

Compassionate use of cancer treatments is most frequently sought by patients for whom tumors have become resistant to standard treatments, or patients who are not candidates for FDA-approved treatments. Demand for access to investigational cancer treatments among patients and clinicians has grown steadily over recent years.

McKeeDuring the American Society of Clinical Oncology (ASCO) 2016 Annual Meeting in Chicago, attendees heard more about patient access to investigational drugs during the June 6 session, “Expanded Access and the Right to Try: Navigating the Intersection of Drug Development and Patient Access to Investigational Agents.”

Amy E. McKee, MD, acting deputy office director in FDA’s Office of Hematology and Oncology Products (OHOP) and chair of the ASCO session, spoke to Managed Healthcare Executive (MHE) about some of the key challenges and opportunities presented by expanded-access programs.

MHE: What is driving expanded-access programs for investigational drugs?

McKee: There is clearly a strong patient voice asking for more access to investigational products, as evidenced by social media activity and the right-to-try laws in many states.

MHE:  Is there a legal obligation for drug companies to provide patients with access to unapproved products?

McKee: No. This has been litigated to the level of the Supreme Court, which found that companies are not legally obligated to provide access to investigational products.

MHE:  How have expanded-access programs changed over recent years?

McKee: In 2009, FDA revised its expanded access regulations to address concerns over the lack of clarity that resulted in disparate access for different types of patients and that access that was primarily limited to patients with certain diseases.

The revisions laid out three categories of expanded access:

  • For individual patients, including emergency use;

  • For intermediate-sized populations, usually more than 10 patients but less than hundreds; and

  • For large patient populations under a treatment investigational new drug application (IND) or treatment protocol.

FDA also listened to the physician community about the difficulty of paperwork for single-patient INDs (SPIs). In 2015, the FDA published a draft guidance on a new application, Form FDA 3926. The draft Form FDA 3926 is intended to provide a streamlined alternative for submitting an IND for individual patient access to an investigational product.

Next: What are the potential risks?



MHE: What are the potential risks of patient access to investigational therapies prior to FDA drug approval?

McKee: There are several potential concerns. First, for investigational products, FDA has not made a determination that the product is safe and effective for use. Therefore, each request for expanded access must be evaluated for its potential risk and benefit to the proposed patient or population. Second, as stated in FDA regulations, the expanded access use should not interfere with the clinical development of the product for marketing approval.

MHE: What is the FDA’s role in allowing pre-drug-approval access requests? What is the process for pre-approval access requests?

McKee: There are several requirements to meet for expanded access:

  • It must be for a serious or immediately life threatening illness/condition;

  • There cannot be a comparable or satisfactory alternative therapy available;

  • The product cannot be obtained under another IND or protocol;

  • The potential benefit justifies the potential risks, such that the risks are acceptable in the context of the disease; and

  • Providing expanded access will not interfere with its clinical development for marketing approval.

For single-patient INDs, the process is as follow:

  • A licensed physician determines probable risk from the drug does not exceed risk from disease for the individual patient;

  • The request can be submitted as a new IND by a licensed physician, usually cross-referencing an existing IND, or can be submitted by a commercial sponsor of an existing IND

  • Informed consent must be obtained from the patient; and

  • Institutional review board approval must be obtained before starting treatment, except in some cases for emergency use.

The submission should include a brief clinical history of the patient and the rationale for requesting the proposed treatment; treatment information, and a treatment plan, including planned dose, route, schedule of administration and monitoring procedures; a letter of authorization from the investigational product’s commercial sponsor; a physician’s qualification statement and contact information.

MHE: What criteria does FDA use in addressing pre-drug-approval access to investigational agents?

McKee: FDA examines SPI requests for all the criteria noted above. Furthermore, FDA makes a determination whether it agrees that the potential benefits outweigh the risks for the particular patient and whether there are any clinical trials to which the patient may be enrolled for the product. If all the criteria are met, the SPI is allowed to proceed.

MHE: How frequently does FDA approve such requests? 

McKee: OHOP in FDA approves the vast majority of SPIs it receives. In an internal review conducted of SPIs received by OHOP from 2012 to 2014 OHOP allowed more than 99% of them to proceed.



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