Traditionally, Medicare has allowed use of FDA approved drugs with unapproved uses from the American Hospital Formulary System (AHFS). It is a drug information service that essentially provides Compendia listing for drugs based on a minimum of 2 peer reviewed journal articles with published clinical trial data.
What are the benefits of Compendia listing?
Well, for cancer patients with advanced stage disease there are often few therapies available after several lines of treatment have been explored and in many cases, radiotherapy or chemotherapy may not be effective, so use of off-label drugs offers a chance of hope that something new that is being tested in clinical trials may help them.
For pharmaceutical companies, thre are also benefits since Medicare will pay for the therapy and thus the patients are not subjected to the vagaries of compassionate use and all the bureaucracy that goes with it. This is frustrating for all concerned – insurers, patients, doctors and pharma companies.
In other words, Compendia listing, offers a fair and equitable solution all around that is evidence based. Usually, the pharma company is completing the larger phase III trials and Compendia listing is based on limited phase II data. Pharma companies should not be forced into a situation where they have to play 'God' either through granting compassionate use or a lottery system. Far better to allow the patients and doctors to decide what is the best option, providing insurance coverage is viable.
My own experience with this process was based on the listing for Gleevec, a targeted therapy that was approved for CML, a form of leukemia, and was undergoing trials for gastro-intestinal stromal tumours (GIST). By obtaining Compendia listing via publications from a reputable medical journal such as the New England Journal of Medicine, patients not included in the clinical trials were still able to gain access to the therapy for a condition that was unresponsive to chemotherapy. The drug was subsequently approved by the FDA for GIST, but a short term solution via Compendia listing was a win-win for all concerned.
Overall, the system has worked very well so far, but as the WSJ pointed out, expanding the rules to include other compendiums, including that of some physician networks, starts to raise issues of conflict of interest. That's not a good place to go and one wonders why change a workable system?