I'm often asked , what is Patient Advocacy and how does it work for sickle cell disease patients. Allow me to share some of the work done behind the scenes to provide an example of SCD patient advocacy on a national level. Following are excerpts from a letter I wrote to the then FDA Commissioner, Margaret Hamburg about how the bio/pharmaceuticals companies could be encouraged to develop SCD drugs with something known as a priority review voucher:
"Dear Commissioner Hamburg, As a parent of a young adult son with SCD and a former biotech industry marketer of orphan drugs, I have a unique perspective about the relevance of a Rare Pediatric Diseases (RPDs) designation for SCD (ref. docket number FDA-2014-D-1461). Primarily, the Priority Review Voucher (PRV) for RPDs provides a powerful stimulus for seeking more and better SCD treatments.
In 2012 Congress enacted the Creating Hope Act backed by a bipartisan coalition of lawmakers and championed by Congressman Butterfield (Dem - NC). This law encouraged the bio/pharmaceutical industry to develop therapies for rare pediatric diseases. Known as the Rare Pediatric Disease Priority Review Voucher or RPV, this provides industry with a unique tool, the right to receive an advance to the front of the line for review of a drug candidate application if the disease in question was considered to be rare and affecting the pediatric population. New SCD Therapies Needed
Clearly, there is an urgent need for new approaches and new therapies. After all, the medical literature reports an understanding of the underlying problem in SCD that goes back over 100 years. However, the bio/pharmaceutical industry lacks incentives for developing treatments. So that should there be any drug R&D, the industry will settle for the lowest standards in their clinical development programs. There may be drugs approved but they will be limited to trading off a day or so in the hospital on opioids versus interrupting or reversing the underlying pathology and other dimensions of SCD captured in patient reported outcomes.
The image of acute painful vaso-occlusive crises (VOCs) in SCD is tragic but just the tip of the iceberg. Below the surface the full burden of disease is more complex. Because the end organ damage from the ischemia of compromised blood flow and oxygenation is the real pathology of concern. The processes involved in preventing damage to tissues and organs and restoring function should be the focus of future treatments along with widely available cures. After all would it be appropriate to manage a heart attack or stroke with IVs and pain meds? Yet that, in effect, is how SCD VOC crises are managed. Crisis management has its place but the focus needs to be on innovation.