BIO submitted comments on the Food and Drug Administration’s (FDA) Draft Guidance, Evaluation of Bulk Drug Substances Nominated for Use in Compounding Under Section 503B of the Federal Food, Drug, and Cosmetic Act. Compounded drugs can serve an important role for patients who have clinical needs that cannot be met by an FDA-approved product. Access to medically necessary compounded medicines is important – but access cannot and should not come at the expense of product quality and patient safety. BIO strongly encourages and supports the FDA’s allocation of resources to enable the safety of compounded drugs. BIO agrees these drugs should only be used by patients whose medical needs cannot be met by an FDA-approved drug. In addition, BIO is pleased to see the FDA discuss the importance of the drug approval process and the need to ensure compounded drugs do not weaken incentives for companies to conduct clinical testing and bring new drugs to market. In the comments, BIO also discusses issues including the process for nomination and review of the 503B list for bulk compounding, clarification of what constitutes “clinical need,” and non-biologic complex drugs and narrow therapeutic index drugs.

Good morning Chairman Huizenga, Ranking Member Maloney, and Members of the Capital Markets, Securities, and Investment Subcommittee. My name is Brian Hahn, and I am the Chief Financial Officer of GlycoMimetics, Inc., a 48-employee public biotech company based in Rockville, Maryland. I am also the Co-Chair of the Finance and Tax Committee at the Biotechnology Innovation Organization (BIO), which represents GlycoMimetics and over 1,100 other growth-stage biotechs that are driving the search for the next generation of cures and breakthrough medicines.The ability of growing businesses to access the public markets, as supported by the JOBS Act, is of paramount importance to biotechnology innovation because investment capital is the lifeblood of scientific advancement. It costs over $1 billion to develop a single life-saving treatment, and most companies spend more than a decade in the lab before their first therapy is approved. During this long development process, virtually every dollar spent by an emerging biotech comes directly from investors. Expenses ranging from buy-in-bulk beakers to $150 million clinical trials are all funded by investment capital because biotechs remain pre-revenue through their entire time in the lab and the clinic.