Food And Drug Administration Authority Research Articles

Improving Food and Drug Administration-Centers for Medicare and Medicaid Services Coordination for Drugs Granted Accelerated Approval.

The Food and Drug Administration (FDA)'s accelerated-approval pathway expedites patient access to promising treatments. However, increasing use of this pathway has challenged the Medicare program, which often pays for expensive therapies despite substantial uncertainty about benefits and risks to Medicare beneficiaries. We examined approaches to improve coordination between the FDA and Centers for Medicare and Medicaid Services (CMS) for drugs granted accelerated approval. We argue that policymakers have focused on expedited pathways at the FDA without sufficient attention to complementary policies at the CMS. Although differences between the FDA and CMS decisions are to be expected given the agencies' different missions and statutory obligations, procedural improvements can ensure that Medicare beneficiaries have timely access to novel therapies that are likely to improve health outcomes. To inform policy options and recommendations, we conducted semistructured interviews with stakeholders to capture diverse perspectives on the topic. We recommend ten areas for consideration: clarifying the FDA's evidentiary standards; strengthening FDA authorities; promoting earlier discussions among the FDA, the CMS, and drug companies; improving Medicare's coverage with evidence development program; tying Medicare payment for accelerated-approval drugs to evidence generation milestones; issuing CMS guidance on real-world evidence; clarifying Medicare's "reasonable and necessary" criteria; adopting lessons from international regulatory-reimbursement harmonization efforts; ensuring that the CMS has adequate staffing and expertise; and emphasizing equity. Better coordination between the FDA and CMS could improve the transparency and predictability of drug approval and coverage around accelerated-approval drugs, with important implications for patient outcomes, health spending, and evidence generation processes. Improved coordination will require reforms at both the FDA and CMS, with special attention to honoring the agencies' distinct authorities. It will require administrative and legislative actions, new resources, and strong leadership at both agencies.

Improving Drug Therapy for Pediatric Patients: Unfinished History of Pediatric Drug Development.

Improving Drug Therapy for Pediatric Patients: Unfinished History of Pediatric Drug Development.

Challenges and opportunities for IBD drug development: from early stage to regulatory approval

Increased understanding of the immunopathology of inflammatory bowel disease (IBD) has resulted in the development of novel therapies such as vedolizumab or ustekinumab, and the investigation of new agents including...

Too Much Carrot and Not Enough Stick in New Stem Cell Oversight Trends.

Regulators are now more often distinguishing between perceived good citizens and "bad actors" in stem cell and regenerative medicine clinical research, resulting in relatively more polar, carrot-and-stick oversight approaches. Here, I discuss why there may be too much carrot and not enough stick by regulators for effective enforcement.

Postmarket studies required by the US Food and Drug Administration for new drugs and biologics approved between 2009 and 2012: cross sectional analysis

ObjectivesTo characterize postmarketing requirements for new drugs and biologics approved by the US Food and Drug Administration (FDA), and to examine rates and timeliness of registration, results reporting, and publication...

Laboratory-Developed Tests: A Legislative and Regulatory Review.

Twenty-five years ago, the Food and Drug Administration (FDA) asserted in a draft document that "home brew" tests-now commonly referred to as laboratory-developed tests (LDTs)-are subject to the same regulatory oversight as other in vitro diagnostics (IVDs)4. In 2010, the FDA began work on developing a proposed framework for future LDT oversight. Released in 2014, the draft guidance sparked an intense debate over potential LDT regulation. While the proposed guidance has not been implemented, many questions regarding LDT oversight remain unresolved. This review provides an overview of federal statutes and regulations related to IVDs and clinical laboratory operations, with a focus on those potentially applicable to LDTs and proposed regulatory efforts. Sources reviewed include the Code of Federal Regulations, the Federal Register, congressional hearings, guidance and policy documents, position statements, published literature, and websites. Federal statutes regarding IVDs were passed without substantive evidence of congressional consideration toward the concept of LDTs. The FDA has clear oversight authority over IVD reagents introduced into interstate commerce. A 16-year delay in publicly asserting FDA authority over LDTs, the pursuit of a draft guidance approach toward oversight, and establishment of regulations under the Clinical Laboratory Improvement Amendments of 1988 (CLIA'88) applicable to LDTs contributed to community uncertainty toward LDT oversight. Future regulatory and/or legislative efforts may be required to resolve this uncertainty.

Perceptions of the Food and Drug Administration as a Tobacco Regulator.

The U. S. Food and Drug Administration (FDA) now has regulatory authority over all tobacco products. Little is known about public awareness and perceptions of FDA in their new role as a tobacco regulator. This research utilizes focus groups to examine perceptions of FDA as a tobacco regulator so that FDA can better communicate with the public about this role. We conducted 6 focus groups in 2014 among a diverse sample of smokers and non-smokers. Participants were asked if they had heard of FDA, what they knew about FDA, if they associated FDA with tobacco, and their thoughts about this FDA role. A total of 41 individuals participated. Although nearly all participants had heard of FDA, most were not aware of FDA's regulatory authority over tobacco products, did not associate the role of FDA with tobacco, and some drew comparisons between FDA's work in tobacco and their work regulating food and drugs. Data suggest that although public awareness of FDA regulatory authority over tobacco is low, with proper public education, the public may find FDA to be a trustworthy source of tobacco regulation.

Unicorns cartoons: marketing sweet and creamy e-juice to youth

In 2009, the Food and Drug Administration (FDA) banned characterising flavours for cigarettes with the exception of menthol and tobacco.1 New regulations, released in May 2016, deemed electronic cigarettes to...

Nutrition labels to change in the United States

Nutrition labels to change in the United States

Consumers’ Understanding of FDA Approval Requirements and Composite Scores in Direct-to-Consumer Prescription Drug Print Ads

In 2 studies, we investigated how laypersons perceive the Food and Drug Administration (FDA) approval process, FDA authority, and the presentation of composite scores in direct-to-consumer (DTC) prescription drug print ads. The 1st study consisted of 4 focus groups (N = 38) in 2 cities. Using a semi-structured guide, a moderator led participants through the viewing of 3 existing DTC print ads that differed in the presence or absence of composite score information, and participants discussed their views of the ads and their understanding of composite scores. The 2nd study surveyed a nationally representative sample of 1,629 individuals from the general population who saw a fictitious DTC print ad and answered closed-ended questions about the same topics. Results showed that knowledge of FDA approval and authority was mixed, with several misconceptions apparent. Many consumers were not familiar with the use of composite scores in a medical context or in advertising and, in the 1st study, expressed distrust of the product and the ad after learning about how composite scores are used. In the 2nd study, receiving composite score information changed the perceived clarity of the ad but not the perceived risk or benefits. Implications for the presentation of complex medical information are discussed.

FDA regulates e‐cigarettes, but advocates say more action is needed to protect youth

Last month, the Food and Drug Administration (FDA) issued rules that regulate e‐cigarettes, as well as other tobacco‐related products such as cigars, hookahs, and pipes. The rule extends the FDA's tobacco authority and implements the Family Smoking Prevention and Tobacco Control Act of 2009. The main focus is on banning e‐cigarette use by youth under age 18; e‐cigarette use (“vaping”) has been increasing in young people even as there have been tremendous decreases in smoking in that age group.

Graphic warning labels and the demand for cigarettes

BackgroundIn 2010, the US Food and Drug Administration (FDA) proposed requiring tobacco companies to add graphic warning labels (GWLs) to cigarette packs. GWLs are large prominently placed warnings that use...

Trends in market share of leading cigarette brands in the USA: national survey on drug use and health 2002–2013

ObjectivesThe main objective of this study is to examine trends in market share for leading cigarette brands, both before (2002–2008) and after (2009–2013) Food and Drug Administration (FDA) regulation of...

Testing times ahead

The research and testing of drugs and devices within the rigid three-tier clinical trial framework is designed to ensure both the safety and efficacy of a drug or device as a prerequisite for marketing authorisation from the regulatory body. Under this criterion, experimental drugs can be given only to people participating in a clinical trial; under the jurisdiction of the US Food and Drug Administration (FDA), however, there are several very different mechanisms to grant access to experimental treatments when the standard of care may be inadequate or even non-existent.1http://www.fda.gov/MedicalDevices/DeviceRegulationandGuidance/HowtoMarketYourDevice/InvestigationalDeviceExemptionIDE/ucm051345.htm (accessed July 22).Google Scholar Such early or extended access options include emergency use, compassionate use, off-label use, and humanitarian exemptions.2DiPALS Study Group CollaboratorsSafety and efficacy of diaphragm pacing in patients with respiratory insufficiency due to amyotrophic lateral sclerosis (DiPALS): a multicentre, open-label, randomised controlled trial.Lancet Neurol. 2015; 14: 883-892Google Scholar Although such applications can provide accelerated access for patients, in the USA this process is administered by the FDA, and although efforts have been made to streamline applications, many think that the process is still overly bureaucratic. This may, in some part, have contributed to the proliferation of right-to-try legislation across many US states.3FDA to simplify ‘Compassionate Use’ drug applications.Medscape. Feb 04, 2015; (accessed July 22).Google Scholar Under this legislation, terminally ill patients who have exhausted other available treatments will have a means to apply, through their physician, to the manufacturer of drugs that have passed phase 1 of a clinical trial, bypassing the need for federal approval and moving the decision to grant access onto the pharmaceutical company. Following on from individual state laws, on July 9, 2015, the Right to Try Act of 2015, which aims to prohibit federal government agencies from taking action to prevent terminally ill patients from accessing experimental drugs, was introduced to US Congress.4States move to give terminally ill ‘right-to-try’ drugs.USA Today. Feb 20, 2015; Google Scholar, 5http://www.fdalawblog.net/fda_law_blog_hyman_phelps/2015/07/the-right-to-try-act-of-2015-a-serious-challenge-to-fda-control-of-expanded-access-.html (accessed July 22).Google Scholar Understandably, these legislatures have divided commentators on issues of patient rights and the undermining of the FDA's authority. These arguments aside, the eventual outcomes of these deliberations will doubtless have a huge effect on patients, physicians, federal agencies, and pharmaceutical companies alike.

Conservatism, Consumer Choice, and the Food and Drug Administration during the Reagan Era: A Prescription for Scandal, by Lucas Richert

Conservatism, Consumer Choice, and the Food and Drug Administration during the Reagan Era: A Prescription for Scandal, by Lucas Richert. Lanham, Lexington Books, 2014. viii, 221 pp. $90.00 US (cloth). Lucas Richert frames the Food and Drug Administration (FDA) within the rise of conservatism, a force within the Republican Party and one that attracted Ronald Reagan as perhaps its exemplar. In this sense Conservatism, Consumer Choice, and the Food and Drug Administration during the Reagan Era is as much about the FDA as it is about the ideology that has attempted to constrict the agency. In this context the book may be best understood as an example of the triumph of conservatism in modem American life. Scholars have produced many studies of the pharmaceutical industry and the FDA. What Richert delivers is a political narrative of conservatism within the context of capitalism, scientific research, and the rise and fall of ideologies. The author begins his treatment with a brief history of the FDA, tracing its roots to what one might call its proto inception in the US State Department in 1839. Much credit of course is due Upton Sinclair and Harvey Wiley in the first decade of the twentieth century, but the story is too well-known to merit treatment. Within this historical framework it is useful to trace changes in the scope of the FDA's authority. Over the short term Richert focuses sharply on Reagan as the catalyst of modem conservatism. Well-known was the president's penchant for portraying government as antithetical to freedom. Accordingly the discourse shifted toward the prospect of shrinking the size and scope of government. As president, Reagan had the federal government in mind, but plenty of his sycophants applied his principles to state and local governments. Reagan took aim at a number of federal agencies including the FDA. In this context arose questions about the FDA's actions. To what degree did the FDA have the authority to regulate drugs and other substances that Americans ingested? Did this authority conflict with freedom of choice? Did the FDA operate outside the confines of capitalism? Richert explores the tensions within such inquiries, but authoritative judgments are difficult to make. The author stretches his chronology between 1974 and 2008 but spends much of his energy examining the 1970s and 1980s. During this era, pharmaceutical companies were eager that Reagan, certainly by the 1980s, diminish the regulatory powers of the FDA. In this context the innovation argument came to the fore as drug companies argued that federal regulations penalized them for pursuing new lines of research and developing truly novel therapies. …

The Codex Alimentarius Commission, Corporate Influence, and International Trade: A Perspective on FDA's Global Role

The FDA Food Safety Modernization Act (FSMA) is by all accounts the most sweeping and comprehensive update to U.S. food laws in seventy years, aiming to confront the reality that the nation's food supply has undergone fundamental shifts in its sources, distribution channels, and intermediate handlers. The law's intent is to prevent problems that can cause foodborne illness and enable the Food and Drug Administration (FDA) to keep a record of facilities processing food for sale in the United States, a mandate that expands FDA's already global regulatory activities. FSMA gives FDA broad new powers to prevent food safety problems, detect and respond to food safety issues, and improve the safety of imported foods. Because the law specifically aims to update FDA authority in light of the reality of global food and food additive markets, Section 305 FSMA calls for FDA to develop a comprehensive plan to expand the “technical, scientific, and regulatory capacity of foreign governments and their respective food industries in countries that export foods to the United States.”

E‐cigarettes and the future of tobacco control

E‐cigarettes and the future of tobacco control

A Question for Women's Health: Chemicals in Feminine Hygiene Products and Personal Lubricants

Vaginal research got a desperately needed boost at the National Institutes of Health (NIH) in 1992. That’s when Penny Hitchcock took over the Sexually Transmitted Diseases Branch at the National Institute of Allergy and Infectious Diseases and Nancy Alexander became chief of the Contraceptive Development Branch in the NIH Center for Population Research—posts previously held by men. “Those two got together and discovered NIH had no programs for vaginal research,” says Richard Cone, a Johns Hopkins biophysics professor. Cone had begun developing vaginal contraceptives that would protect against sexually transmitted infections (STIs) in 1980, and until then, struggled to get funding. Mucous membranes in the vagina and vulva rapidly absorb chemicals without metabolizing them. But until recently scant research existed on how chemicals in feminine hygiene products and personal lubricants may affect women’s health. Hitchcock and Alexander soon initiated research programs on vaginal physiology, immunology, and microbicides, eventually funding Cone’s work. These new programs led to groundbreaking discoveries in animals and humans that certain chemicals—including glycerin (glycerol), a common base for personal lubricants—can damage or irritate vaginal1 and rectal2 epithelial cells, potentially increasing the transmission of STIs such as herpes and human immunodeficiency virus. When it comes to reproductive health, research on contraceptives and STIs continues to garner interest worldwide. But a related area—chemical exposures from feminine hygiene products and personal lubricants—has received much less attention. In the United States alone, women spend well over $2 billion per year on feminine hygiene products,3 including tampons, pads, feminine washes, sprays, powders, and personal wipes. But until recently, scant research existed on how chemicals in these products may affect women’s health. As scattered findings emerge, several scientists and interest groups are calling for more research to fill in the gaps.4,5 A recent report by the nonprofit Women’s Voices for the Earth (WVE) points out that feminine hygiene products may use ingredients that are known or suspected endocrine-disrupting chemicals (EDCs), carcinogens, or allergens. And while nearly all women use tampons and sanitary pads, black and Latina women use douches, wipes, powders, and deodorizers more often than women of other races, putting them at greater risk of potential chemical exposures.4 “It’s a little bit edgy. People have been shocked, saying things like ‘I’ve never really thought about [the vagina] being an important internal link to your body,’” says Alexandra Scranton, director of science and research for WVE. “Although it is well known that the vaginal ecosystem is more sensitive and more absorbent than typical skin, there is surprisingly little research out there on feminine care products.” Surveys conducted in the 1990s–2000s gave a sense not only of how commonly some products are used but also how widely use can vary across racial/ethnic groups.

A Comprehensive Strategy to Overhaul FDA Authority for Misleading Food Labels

The modern food environment is considered a primary driver of obesity and other nutrition-related chronic diseases. A significant contribution to this environment is the proliferation of claims on food packaging that provides a misleading picture of a product's healthfulness. The Food and Drug Administration (FDA) is the agency responsible for food labels but it lacks the regulatory authority and adequate resources to address the majority of questionable labeling practices. The FDA's current system of enforcement is thus essentially based on voluntary compliance and consumer- and manufacturer-initiated litigation has not successfully filled the regulatory gap. This manuscript reviews the current state of food labeling claims and the FDA's inadequate authority over misbranded food products. It analyzes competing views on regulatory compliance strategies and argues that a regulatory overhaul consistent with the best science and the First Amendment is necessary. With increased resources and authority, the FDA can meet current public health challenges and adequately ensure that labels are clear and consumers are properly informed and protected.

Dye Sensitizers for Photodynamic Therapy.

Photofrin® was first approved in the 1990s as a sensitizer for use in treating cancer via photodynamic therapy (PDT). Since then a wide variety of dye sensitizers have been developed and a few have been approved for PDT treatment of skin and organ cancers and skin diseases such as acne vulgaris. Porphyrinoid derivatives and precursors have been the most successful in producing requisite singlet oxygen, with Photofrin® still remaining the most efficient sensitizer (quantum yield = 0.89) and having broad food and drug administration (FDA) approval for treatment of multiple cancer types. Other porphyrinoid compounds that have received approval from US FDA and regulatory authorities in other countries include benzoporphyrin derivative monoacid ring A (BPD-MA), meta-tetra(hydroxyphenyl)chlorin (m-THPC), N-aspartyl chlorin e6 (NPe6), and precursors to endogenous protoporphyrin IX (PpIX): 1,5-aminolevulinic acid (ALA), methyl aminolevulinate (MAL), hexaminolevulinate (HAL). Although no non-porphyrin sensitizer has been approved for PDT applications, a small number of anthraquinone, phenothiazine, xanthene, cyanine, and curcuminoid sensitizers are under consideration and some are being evaluated in clinical trials. This review focuses on the nature of PDT, dye sensitizers that have been approved for use in PDT, and compounds that have entered or completed clinical trials as PDT sensitizers.