Access to new drugs outside of the US is frequently delayed by several years following FDA approval in the US. For example, lenalidomide (Revlimid®) received US FDA approval in June 2006, yet is still not fully approved and available globally. This lack of access has a significant negative impact for myeloma patients. At the point of relapse, patients who may have been treated frontline with VTD (Velcade®, Thalomid®, dexamethasone) or CyBorD, only relatively recently and in some cases have been able to turn to Revlimid®.
In the case of pomalidomide (Pomalyst®), the most recently approved IMiD (approved in the US for relapse/refractory disease in February 2013 and in Europe in August 2013), global access is currently very limited. Pomalidomide is not available for relapsing patients in Asia.
The IMF’s Asian Myeloma Network has therefore negotiated with the Celgene Corporation to establish an individual or “named” patient access program for pomalidomide. Utilizing a treatment protocol very similar to the trials conducted in the US and Europe, eligible relapsing patients can now gain access to pomalidomide through the main Asian treatment centers. After overcoming numerous regulatory and logistic hurdles, the protocol was activated in December 2014 with Dr. Wee Joo Chng as the principal investigator. Dr. Chng, an IMWG and AMN member, is based at the University of Singapore where the data management and coordination center is housed and funded.
Accruals started rapidly in Korea with Dr. Jae Hoon Lee, who enrolled four myeloma patients in December. He is excited to report that at first follow-up, all patients showed evidence of response and excellent tolerance. Patients are now enrolling in Singapore, to be followed by Thailand, Hong Kong, and Taiwan. Unfortunately, it is not yet feasible to open the program in Japan and China, the other two AMN members.
The successful launch of this pomalidomide access program is very exciting for both patients and physicians in Asia. This is a model through which patients can gain early access to new drugs which can be literally lifesaving! It is hoped that it is just the beginning of programs to enhance global access to new FDA approved drugs, which are so desperately needed to improve myeloma patient survival.
Dr. Brian G.M. Durie serves as Chairman of the International Myeloma Foundation and serves on its Scientific Advisory Board. Additionally, he is Chairman of the IMF's International Myeloma Working Group, a consortium of nearly 200 myeloma experts from around the world. Dr. Durie also leads the IMF’s Black Swan Research Initiative®.