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FDA and USP take sides in debate on biologic drug standards.
The May 2019 approval of Zolgensma, a gene therapy to treat spinal muscular atrophy, by FDA’s Center for Biologics Evaluation and Research was marked by sticker shock: a $2.1-million price tag for the drug, making it the most expensive drug approved to date (1). Patient-advocates noted the curative nature of the therapy was worth the price, and the drug company offered a phased-payment approach. Proponents cited it as another example of the high costs of drugs (2).
Meanwhile, a bipartisan effort by the Senate Health, Education, Labor and Pensions (HELP) committee has led to a contentious debate between FDA and standards-setting organization, the US Pharmacopeial Convention (USP). The committee voted 20–3 on June 26, 2019 to advance legislation designed to lower healthcare and drug costs; proposals include changes to patent policy, ending “surprises” in medical bills, increasing transparency in healthcare, and reducing roadblocks that delay generic drugs and biosimilars getting to market (3).
For example, Section 208 of the proposed legislation changes existing regulations so drug manufacturers cannot receive new chemical entity exclusivity for making small changes to existing drugs.
It is provisions in Section 207, how- ever-designated as promoting biological drug innovation-that are proving controversial, pitting FDA and USP in an exchange of policy statements. Ac- cording to HELP committee documents, Section207isintendedtopreventde- lays in the licensure of biosimilar and interchangeable products by excluding biological products subject to regulation under the Public Health Service
Act from requirements to follow United States Pharmacopeia compendial standards. A similar provision to make bio- logics exempt from meeting USP standards was removed from the final 21st Century Cures Act of 2016 (4).
“A biological product is so inherently complex and variable that the established structure of the USP monograph standards process does not serve it well, and in fact, can impede technological progress or innovation,” noted Steven Kiozlowski, director of the Office of Bio- technology Products in FDA’s Office of Pharmaceutical Quality in an interview published by FDA (5).
Due to the inherent differences in bio- logical products, the “sameness” standard used in USP monographs for chemical- based drugs cannot apply to biologic drugs, FDA argues.
USP, backed by patient groups and pharmacists, argues that the elimination of required standards would harm patients. In a letter to the committee, USP noted the role standards play in providing quality benchmarks; supply chain security; reliability and predict- ability for drug product development, manufacturing, and distribution; and promote transparency and accountability that leads to patient trust (6).
“Essential to the framework that safeguards the quality and safety of medicines in the United States is the principle that public quality standards, required under the law, establish and articulate quality expectations for medicines, including biologics,” USP noted in the letter.
FDA has no legal authority to control the price of drugs. However, the agency notes it is “committed to facilitating increased competition in the market for prescription drugs through the approval of lower-cost, generic medicines” and “makes sure that safe and effective drugs are available to improve the health of consumers” (7).
That’s a lot of priorities to balance in today’s partisan environment.
1. R. Stein, “At $2.1 Million, New Gene Therapy Is the Most Expensive Drug Ever,” npr.org, May 24, 2019.
2. Novartis, “AveXis Announces Innovative Zolgensma Gene Therapy Access Programs for US Payers and Families,” Press Release, May 24, 2019.
3. S. 1895, US Senate, 116th Congress, 1st Session (Washington, DC).
4. J. Wechsler, “FDA Challenges USP Standards for Biologics,” www.PharmTech. com, June 25, 2019.
5. FDA, CDER Conversation: Ensuring That Standardization Does Not Impede Biological Product Innovation, June 12, 2019.
(HELP), June 11, 2019.
7. FDA, Frequently Asked Questions about CDER, www.fda.gov, accessed June 27, 2019.
Vol. 43, No. 7
When referring to this article, please cite it as R. Peters, “A Biologics Partisan Divide," Pharmaceutical Technology 43 (7) 2019.