The U.S. Food and Drug Administration’s Complex Innovative Trial Design Pilot Meeting Program: Progress to date

2021 ◽  
pp. 174077452110505
Author(s):  
Dionne Price ◽  
John Scott
Keyword(s):  
Public Health ◽  
Drug Administration ◽  
Trial Design ◽  
Drug Evaluation ◽  
Food And Drug Administration ◽  
Medical Product ◽  
Global Public Health ◽  
Multiple Sources ◽  
Meeting Program ◽  
The U.S

Background The Center for Drug Evaluation and Research and the Center for Biologics Evaluation and Research of the U.S. Food and Drug Administration have been leaders in advancing science to protect and promote public health by ensuring that safe and effective drugs and biological products are available to those who need them. Recently, new therapeutic discoveries, increased understanding of disease mechanisms, the need for innovation to optimally use resources, and global public health crises have led to an evolving drug development landscape. As a result, the U.S. Food and Drug Administration and medical product developers are faced with unique challenges and opportunities. The U.S. Food and Drug Administration is proactively meeting the challenges of this evolving landscape through various efforts, including the Complex Innovative Trial Design Pilot Meeting Program. Our focus, here, will be on the pilot meeting program. Methods The U.S. Food and Drug Administration has defined a process to facilitate the implementation of the Complex Innovative Trial Design Pilot Meeting Program. The process is transparent and outlines the steps and timeline for submission, review, and meetings. Results Five submitted meeting requests have been selected for participation in the Complex Innovative Trial Design Pilot Meeting Program. Conclusion The pilot meeting program has been successful in further educating stakeholders on the potential uses of complex innovative designs in trials intended to provide substantial evidence of effectiveness. The selected submissions, thus far, have all utilized a Bayesian framework. The reasons for the use of Bayesian approaches may be due to the flexibility provided, the ability to incorporate multiple sources of evidence, and a desire to better understand the U.S. Food and Drug Administration perspective on such approaches. We are confident the pilot meeting program will have continued success and impact the collective goal of bringing safe and effective medical products to patients.

HIV

2018 ◽  
Author(s):  
Joshua M. Sharfstein
Keyword(s):  
Public Health ◽  
Drug Administration ◽  
Food And Drug Administration ◽  
Research Process ◽  
National Institutes Of Health ◽  
Health Agencies ◽  
Act Up ◽  
The U.S

The emergence of AIDS in the early 1980s caused a profound crisis for federal health agencies, particularly the National Institutes of Health (NIH) and the U.S. Food and Drug Administration (FDA). Activists in ACT UP, charging that these agencies were failing patients with AIDS, initiated a series of escalating protests. NIH officials, led by Dr. Anthony Fauci, began to talk with the advocates and make major changes in the research process. However, over at the FDA, a protest involving the arrest of hundreds of AIDS activists undermined the agency’s public health image. Eventually, under a new commissioner, the FDA earned back the trust of activists.

scholarly journals A Proposed Framework for Patient-Focused Policy at the U.S. Food and Drug Administration

Biomedicines ◽  
2019 ◽  
Vol 7 (3) ◽  
pp. 64 ◽  
Author(s):  
Carrie M. Kuehn
Keyword(s):  
Development Strategy ◽  
Drug Administration ◽  
Drug Evaluation ◽  
Food And Drug Administration ◽  
Medical Product ◽  
Specific Patient ◽  
Regulatory Decision ◽  
Regulatory Submissions ◽  
Guidance Documents ◽  
Product Strategies

Medical product sponsors are encouraged to include the patient perspective in their medical product development strategy to inform product design, augment regulatory submissions, argue for alternative clinical trial designs, or to support indications in specific patient populations. The goal is to create a patient-focused ecosystem that enables industry to integrate the patient voice throughout the medical product lifecycle. To this end, the U. S. Food and Drug Administration (FDA) has published several guidance documents to provide industry with the expectations and opportunities for conducting patient-focused activities. From an industry perspective, the Center for Devices and Radiologic Health (CDRH) and the Center for Drug Evaluation and Research (CDER)/Center for Biologics Evaluation and Research (CBER) patient-focused policies are complementary. The basic tenets promoted in all FDA patient-focused guidance could apply across therapeutic areas. However, there remain differences in these guidance documents across FDA centers, and there is no framework in place to provide industry with consistent recommendations. Without a coordinated patient-focused policy from the FDA, there is the potential for confusion and a lack of consistency among industry and regulatory decision-makers. The objective of this paper was to propose an alternative framework for patient-focused policy at the FDA, which recognizes the potential for different types of patient input to be used across therapeutic areas and medical product types. Further, these policies need to provide greater clarity on how patient input data is used, so that sponsors may navigate the opportunities to use patient input regardless of the FDA center under which their product is regulated. Creating consistent, coherent, and transparent FDA patient-focused policy will encourage sponsors to obtain patient input more often and with greater certainty of the value that these data may have to their medical product strategies.

Elixir Sulfanilamide

2018 ◽  
Author(s):  
Joshua M. Sharfstein
Keyword(s):  
Public Health ◽  
Drug Administration ◽  
Food And Drug Administration ◽  
New Therapy ◽  
The Difference ◽  
The U.S

In the fall of 1937, patients began to die soon after consuming a new therapy called the Elixir Sulfanilamide. In response, the fledgling agency known as the U.S. Food and Drug Administration (FDA) jumped into action. The agency recovered the remaining 700 bottles and saved the lives of an estimated 4,000 Americans. The FDA also advocated for the landmark Food, Drug, and Cosmetic Act, which, among other major steps, authorized the FDA to establish the world’s first advance review for safety of medications. The story of the Elixir Sulfanilamide exemplifies how much in public health has been shaped by crisis. Strong preparation, a well-managed response, and astute legislative strategy made all the difference in turning crisis to opportunity. If, as Louis Pasteur has famously said, chance favors the prepared mind, then the story of the Elixir Sulfanilamide shows that crisis favors the prepared agency.

scholarly journals Estimating the number of symptomatic SARS-CoV-2 infections among vaccinated individuals in the United States-January-April, 2021

2021 ◽  
Author(s):  
Kiersten J Kugeler ◽  
John Williamson ◽  
Aaron Curns ◽  
Jessica M Healy ◽  
Leisha D Nolen ◽  
...  
Keyword(s):  
Public Health ◽  
United States ◽  
Drug Administration ◽  
Vaccine Efficacy ◽  
Food And Drug Administration ◽  
The United States ◽  
Population Immunity ◽  
The U.S ◽  
Health Messaging

As of March 2021, three COVID-19 vaccines have been authorized by the U.S. Food and Drug Administration (FDA) for use in the United States. Each has substantial efficacy in preventing COVID-19. However, as efficacy from trials was <100% for all three vaccines, disease in vaccinated people is expected to occur. We created a spreadsheet-based tool to estimate the number of symptomatic vaccine breakthrough infections based on published vaccine efficacy (VE) data, percent of the population that has been fully vaccinated, and average number of COVID-19 cases reported per day. We estimate that approximately 51,000 symptomatic vaccine breakthrough infections (95% CI: ~48,000-55,000 cases) occurred in the United States during January-April 2021 among >77 million fully vaccinated people, reflecting <0.5% of COVID-19 cases that occurred during that time. With ongoing SARS-CoV-2 transmission and increasing numbers of people vaccinated in the United States, vaccine breakthrough infections will continue to accumulate before population immunity is sufficient to interrupt transmission. Understanding expectations regarding number of vaccine breakthrough infections enables accurate public health messaging to help ensure that the occurrence of such cases does not negatively affect vaccine perceptions, confidence, and uptake.

Evaluation of Orphan Products by the U.S. Food and Drug Administration

1992 ◽  
Vol 8 (4) ◽  
pp. 647-657 ◽  
Author(s):  
Marlene E. Haffner ◽  
John V. Kelsey
Keyword(s):  
Drug Administration ◽  
Rare Diseases ◽  
Food And Drug Administration ◽  
Orphan Drugs ◽  
Marketing Approval ◽  
Drug Products ◽  
Governmental Agencies ◽  
Number Of Patients ◽  
Life Threatening ◽  
The U.S

AbstractOrphan drug products generally are used in treating or preventing rare diseases. The small number of patients available for study may create special problems in the evaluation of these products. This paper examines some of the special problems that are associated with the design and implementation of studies to evaluate the safety and efficacy of orphan drugs. The U.S. Food and Drug Administration (FDA) has not established special criteria for evaluating orphan drugs per se, but the FDA has been flexible in evaluating drug products that present special problems, especially when these products are for treatment of serious of life-threatening illnesses. The FDA and other U.S. governmental agencies also have taken steps to promote the development and availability of drugs for rare diseases, including making these products available to patients who are in need, even before the drugs have full FDA marketing approval.

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