The FDA allows cancer patients to access investigational treatments outside an ongoing clinical trial through a program called Expanded Access. Colloquially, this is often referred to as Compassionate Use. Investigational treatments are unapproved medicines, which are currently in clinical trials. The Expanded Access program is defined and authorized under 21C FR part 312 subpart I.
To participate, patients must be*:
- in advanced stage, terminal disease;
- out of treatment options; and
- unable to access an existing clinical trial.
*Paraphrased for the sake of brevity. Full criteria are included in the website links provided below
The FDA’s Expanded Access program includes a significant number of gating criteria. Assuming a patient qualifies, a physician must make a request for access to an investigational treatment, and the company (sponsor) that owns the treatment must agree to the use of their investigational drug. If this happens, and all other basic qualifications are met, the FDA grants 99% of requests that it receives.
If you are a cancer patient who does not qualify for a clinical trial and meets the criteria above, you should speak to your physician about potentially accessing a promising new therapy through expanded access. Request to companies and the FDA must be made by a licensed physician. Each company has its own criteria for evaluating and granting Expanded Access requests. Notably, factors unrelated to the patient may limit access, such as insufficient clinical grade drug.
To assist cancer patients and physicians, CytImmune has compiled a list of pharmaceutical company websites and contact information through which physicians may begin the application process for Expanded Use.
|Website||Physician Contact/ Request Forms||Timeline|
|J&J||Program Information||U.S.: 1-800-Janssen or email@example.com|
|BMS||Program Information||Website/Form||48 hours|
|Lilly||Program Information||US: 1-800-LillyRx (1-800-545-5979)||5 Days|
|Takeda||Program Information||Not Listed|
|AstraZeneca||Program Information||US: 1-800-236-9933|
|Merck & Co.||Program Information||1-800-672-6372|
|Amgen||Program Information||1 800-772-6436|
|Pfizer||Program Information||<href=”http://www.cybergrants.com/pfizercares/docs/PfizerCAReS_physician_submission_job_aid.pdf”>Application Instructions|
|Astellas||Program Information||US: 800-727-7003|
|Sanofi||Program Information||Form on Program Info website|
|Eisai||Program Information||1-888-274-2378||2 days|
**CytImmune’s nanomedicine products cannot currently be accessed through the FDA’s Expanded Access program. Once our first and second generation clinical products are in an active clinical trial, we will post information regarding our Expanded Access policies and procedures.