EXCELLENT FIRST STEP FOR 'EXPANDED ACCESS'
NOTE: Expanded access, sometimes called 'compassionate use,' is a provision within the FDA statute, which allows patients to seek access to an investigational medical product (not yet approved by FDA) outside of a clinical trial, if certain conditions are met--usually, when the "physician determines that there is no comparable or satisfactory alternative therapy available to diagnose, monitor, or treat the person’s disease or condition."
Kudos to Associate FDA Commissioner, Dr. Peter Lurie, for proposing a simplified 'expanded access' application for investigational drugs. I personally experienced this process from three different perspectives:
- as a patient who ran out of treatment options,
- as a former healthcare executive,
- and as a strategist involved in designing new drugs and clinical trials.
Of the three, being the patient was by far the hardest. Here is why:
I spent years working side by side with doctors and scientists of all disciplines to design medicines for incurable diseases. My kids went through the entire K-12 school system in the time it took for one of these medicines to succeed. It was meaningful work, and I was proud of it.
I was well aware that most pharmaceutical companies offer investigational compounds (drugs still in development), as well as existing drugs (those already approved, but for other indications) to patients who don’t have other treatment options.
Then I became a patient whose cancer kept coming back. Seven times. Still, it wasn’t until my oncologist said, “I’m very sorry, we don’t have any other options,” that the critical importance of 'expanded access' became blindingly obvious. I immediately realized the process was too complex and way too long for people whose time was running out on them.
My oncologist was willing to write an investigational treatment protocol for an n of one. Me. I was an educated patient who knew the process inside out and was prepared to help with the submission. But we got stuck on the 100 hours of paperwork, in the hospital bureaucracy (protocols seem to get submitted in groups), and we never even got to the FDA submission, let alone requesting the drug from the manufacturer.
Thanks to many (already approved) drugs, extremely risky medical interventions, great doctors, tenacious self-advocacy, and a huge dose of sheer luck, I am still here.
I applaud the FDA for recognizing the need to simplify the 'compassionate use' process. I also believe that this is only one component that requires fixing. Other elements must be simplified as well: internal hospital/clinic policies, insurance coverage for 'compassionate use' drugs, and easier access to investigational compounds from the manufacturers.
All patients should have access to investigational drugs instead of getting lost in the procedural maze.
To find out more about FDA's expanded access ('compassionate use') program, visit here.