Last week, the blood community and 10 organizations representing patients with rare diseases sent letters to the Food and Drug Administration in support of the blood community’s initiative for regulatory harmonization (available to ABC members at http://bit.ly/1hhAhZY). Specifically, these organizations are seeking the regulatory flexibility for blood centers to manage apheresis plasma efficiently, as they do recovered plasma from whole blood, so that it can be shipped for manufacture into plasma protein therapies at any time after collection. This regulatory change will advance population health by addressing the unique needs of patients with rare diseases that use life-saving plasma protein therapies.
FDA and the blood community share a collective responsibility to assure the safety and availability of the US blood supply and life-saving biologic therapies. Our community relies on FDA to make timely regulatory decisions based on scientific evidence. This is the genesis of our long-standing request for the creation of a single plasma product for further manufacture, whether from manual or apheresis collection.
In an environment that is sometimes marked by competing interests, it takes enormous discipline and focus to work collectively for the greater good. We have, on occasion, complained and expressed frustration about the pace and content of regulatory actions. However, complaints and frustration are not solutions. We, the blood community – including collectors, vendors and regulators- must come together calmly and methodically, to work with the agency to bring about change that maintains quality and safety, reduces unnecessary regulatory burden and expense, and provides access to blood products and needed therapies to patients.
The Beatles tune, “With a Little Help From My Friends,” is emblematic of the best approach to policy reform. ABC can accomplish many things with coordinated leadership, or “a little help from our friends.”
And who are our friends in this endeavor? Fellow blood community organizations like AABB and ARC, and other affinity organizations with shared interests like the Plasma Protein Therapeutics Association (PPTA), International Plasma Fractionation Association (IPFA), and AdvaMed. Patient organizations also support this venture, including Alpha-1 Foundation, Committee of Ten Thousand, GBS/CIDP Foundation International, Hemophilia Federation of America, Immune Deficiency Foundation, Jeffrey Modell Foundation, National Hemophilia Foundation, Patient Services Incorporated, Platelet Disorder Support Association, and the US Hereditary Angioedema Association. We also consider FDA an ally – yes, the FDA. They have been most helpful in discussions over the years, and within days of their posting we received a response to our letters from Karen Midthun, MD, director of FDA’s Center for Biologics Evaluation and Research, on behalf of FDA Commissioner Margaret Hamburg, MD. Our goal is to resolve this problem in 2014 with a little help from our friends.
Christine Zambricki, DNAP, CRNA, FAAN, Chief Executive Officer;CZambricki@americasblood.org