USA Law and Practice Contributed by: Daniel Kracov, David Marsh and Alice Ho, Arnold & Porter
1. Life Sciences Regulatory Framework 1.1 Legislation and Regulation for Pharmaceuticals and Medical Devices The primary legislation governing the authorisa- tion, marketing, sale and supply of pharmaceuti- cal products by the US Food and Drug Admin- istration (FDA) is the Federal Food, Drug, and Cosmetic Act (the “FD&C Act” ), which has been amended many times throughout the years to reflect increasing FDA mandates for the regu- lation of pharmaceutical products. The Public Health Service Act (the “PHS Act” ) is the specific authority used to approve or license biological (including biosimilar) products. The primary FDA regulations governing drugs and biologics are found in Chapter 21 of the Code of Federal Regulations. Controlled sub- stances, such as opioids, are also scheduled and subject to quotas and distribution controls under the Controlled Substances Act adminis- tered by the Drug Enforcement Administration (DEA). A drug is defined as: • an article recognised in the US Pharmaco- poeia, the Homeopathic Pharmacopoeia of the United States, or the National Formulary; • an article intended for use in the diagnosis, cure, mitigation, treatment or prevention of disease; • an article (other than food) intended to affect the structure or any function of the body; and • an article intended for use as a component of a drug but not as a device (or a component, part or accessory of a device). A biologic is defined under the PHS Act as “a virus, therapeutic serum, toxin, antitoxin, vac-
cine, blood, blood component (or derivative), allergenic product, protein (or analogous prod- uct), or arsphenamine or derivative of arsphena- mine (or any other trivalent organic arsenic com- pound) applicable to the prevention, treatment or cure of a disease or condition of human beings” . Notably, a protein is any alpha amino acid poly- mer with a specific, defined sequence that is greater than 40 amino acids in size. Biological products also fall within the drug definition and are generally covered by most of the same laws and regulations; however, differences exist in the regulatory approach. Medical devices are also regulated by the FDA under the FD&C Act and – although subject to similar intent standards – such products are pri- marily intended to act via mechanical rather than chemical or biological modes of action. Medical devices are classified by risk and may be: • exempt from FDA review; • subject to “510(k)” pre-market notification process if they show substantial equivalence to “predicate” device; • subject to down-classification via the de novo submission process; or • eligible for full approval via a pre-market approval (PMA) application. Although the FDA has traditionally been given significant independence as an agency, and the Commissioner is confirmed by the Senate, the FDA is part of the Department of Health and Human Services (HHS). The government agencies touching on pricing and reimbursement vary, depending upon the payor programme, and include the Centers for Medicare & Medicaid Services (CMS) (also part of the HHS), the Veterans Health Administration, and state Medicaid agencies. In addition, the
378 CHAMBERS.COM
Powered by FlippingBook