Empowering Early Access to Investigational Medical Products

Our Story

For More Than 30 Years, Our Team Members Have Supported Patient’s Access To Investigational Medical Products For Treatment Outside Of Clinical Trials

Our story started close to home, first highlighted by George, a family friend and patient advocate. George was a lay individual with no knowledge of clinical trials or regulations for obtaining experimental treatments. George was a family friend living in Canada, whose wife was diagnosed with advanced stage 4 breast cancer. After exhausting all available therapies in Canada, George’s wife’s oncologist told him about an experimental treatment made by a pharmaceutical company in Japan. After discussing all treatment options, the oncologist made a request to the pharmaceutical company on behalf of George’s wife. The request was approved by the pharmaceutical company, and by Health Canada. It was up to George to work through the red tape and arrange for the importation of the medicine into Canada. George told me the story of how he arranged for the importation using a red-pouch special courier. This was my first experience with a patient advocate, actively learning about Special Access Programs (the current Canadian term for compassionate use pathway of approval).

Several years later, now a Fellow at the National Institutes of Health in Bethesda, Maryland. We had a group of patients experiencing a life-threatening rhythm disorder requiring an investigational drug currently in Phase 1 studies by a pharmaceutical company based in the United Kingdom. At that time the U.S. Food and Drug Administration was just laying down the regulations for what would become an intermediate Expanded Access Protocol. I submitted a protocol to the pharmaceutical company in the U.K., and the pharmaceutical company agreed to provide the medication if I could secure the required authorization and other documents from the FDA. This was my first foray into expanded access. It was in the late 1980s when there was a growing need for the FDA to develop a formalized pathway for the review and approval of such requests. There were no guidelines, but I didn’t let it deter me. George, and his unabashed advocacy for his loved one, served as my inspiration. The request was approved by the FDA and the intermediate expanded access protocol was able to provide investigational treatment to eighteen patients with a refractory rhythm disorder.

The FDA has supported patients’ access to investigational medical products for treatment, outside of participation in a clinical trial, when appropriate, for over 30 years. I have been on this journey for the same period of time, and this is how Early Access Care came about. We needed experts, familiar with both the pharmaceutical development world and global regulations, to be able to enable patients with serious or chronic conditions without treatment options, the opportunity to receive treatment. Fostering these opportunities are what each and every one of us at Early Access Care is about.

Helping to facilitate access to promising medicines for patients with serious or immediately life-threatening diseases or conditions when no comparable or satisfactory alternative therapy options are available is the commitment of each Early Access Care colleague.