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1 1 (January 18, 2018)

handle is hein.crs/govcgzw0001 and id is 1 raw text is: FF.ri E~$~ & January 18, 2018 Medical Product Innovation and Regulation: Benefits vs. Risks Prior to marketing in the United States, medical products are reviewed for safety and effectiveness by the Food and Drug Administration (FDA). Medical products regulated by FDA include prescription drugs, medical devices, and biologics. During the premarket review process, FDA balances the benefits that patients may receive from using the product against the harms or risks that some patients may experience. Congressional action to regulate medical products has often been in reaction to harm caused by an under regulated medical product. The Biologics Control Act of 1902 was the first attempt to regulate a pharmaceutical product at the national level. It was also the first premarket approval statute, in contrast to a retrospective postmarket product evaluation. The Biologics Control Act was passed in response to deaths, many in children, from tetanus contamination of smallpox vaccine and diphtheria antitoxin. The act focused on the manufacturing process and required that facilities be inspected before a federal license was issued to market a biological product. The regulation of drugs began with the 1906 Food and Drugs Act. The 1906 law did not involve any type of premarket control over new drugs to ensure safety and did not include inspections or any other regulation of manufacturing facilities. The law focused on the drug label, which could not be false or misleading, and required that the presence and amount of certain dangerous ingredients (such as alcohol, heroin, and cocaine) must be listed. Responding to another safety incident, in 1938 Congress replaced the Food and Drugs Act with the Federal Food, Drug, and Cosmetic Act (FFDCA). To make the taste of a new sulfa drug more appealing to pediatric patients, a drug company added a solvent to its product, Elixir Sulfanilamide. The solvent in the untested product was highly toxic and caused the death of over 100 people, including many children. The FFDCA required that drug manufacturers submit, prior to marketing, a new drug application (NDA) demonstrating, among other things, that the product was safe. The FFDCA also included some controls over manufacturing establishments. In 1962, Congress passed the Kefauver-Harris Drug Amendments to the FFDCA in reaction to the birth defects and deaths associated with the use of thalidomide by pregnant women in Europe. The 1962 law increased drug safety provisions and required that manufacturers provide evidence of drug effectiveness. The Medical Device Amendments of 1976 was the first major legislation enacted to address the review of medical devices. The law was passed following the deaths of 17 women and the injury of thousands associated with the use of the Dalkon Shield, a contraceptive intrauterine device. to Swpec%' Prock a3ct F'D~s~~-2A 'Rkekc.w Following the 1962 Kefauver-Harris Drug Amendments, early access (compassionate use) programs allowed some patients to use drugs that were still under investigation; FDA published a rule regarding such access to cancer drugs in 1979. In 1987, FDA formalized the steps to obtain an investigational drug outside a clinical trial (treatment IND, later codified by Congress). FDA established a policy in the early 1990s to allow access to HIV/AIDS drugs for patients unable to enroll in a clinical trial (parallel track). FDA also created mechanisms to speed drug development and review. Priority review was begun by FDA in 1974 and modified by the agency in 1987 and 1992; it directs attention and resources to drugs that offer a treatment advance. FDA launched fast track in 1988 to expedite the development, evaluation, and marketing of new therapies by allowing, for certain serious and life-threatening conditions, FDA review to begin before clinical trials are completed. FDA created the accelerated approval pathway in 1992 for serious or life-threatening diseases that lack effective treatment. It allows use of a surrogate endpoint, via a biomarker test, to predict likely patient improvement from a new treatment, rather than a clinical endpoint (symptom or death) that shows actual improvement in how a patient feels or length of life. A January 2017 FDA report states, however, that most biomarkers have not been shown to reliably predict clinical outcomes due to the complexity of diseases and therapies. Despite the use of these faster review mechanisms, industry and patient groups continued to press Congress for a more rapid drug review process. The Prescription Drug User Fee Act of 1992 (PDUFA, P.L. 102-571) gave FDA authority to collect fees from the pharmaceutical industry and to use the revenue to support the drug review process by hiring additional personnel to evaluate NDAs. Medical device user fees were added 10 years later by the Medical Device User Fee and Modernization Act of 2002 (P.L. 107-250). Congress has addressed FDA user fee reauthorization in five-year increments and often amends FDA regulatory authorities at the same time. For example, Congress codified priority review in PDUFA, fast track in the Food and Drug Administration Modernization Act of 1997 (P.L. 105-115), and accelerated approval in the Food and Drug Administration Safety and Innovation Act of 2012 (FDASIA, P.L. 112-144). Congress itself created the breakthrough drug category, another expedited review program added in FDASIA. However, studies have found that the term breakthrough drug is potentially misleading. The term is often misinterpreted by patients and physicians K~:> mppm qq\ a ' p\w -- ' gn'a', g-o I 'S 11LULANUALiN,

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