​US Tropical Disease Priority Review Vouchers: Lessons In Promoting Drug Development And Access

2021 ◽  
Vol 40 (8) ◽  
pp. 1243-1251
Author(s):  
David B. Ridley ◽  
Pranav Ganapathy ◽  
Hannah E. Kettler
Keyword(s):  
Drug Development ◽  
Tropical Disease ◽  
Priority Review

scholarly journals Integrating Engineering, Manufacturing, and Regulatory Considerations in the Development of Novel Antivenoms

Toxins ◽  
2018 ◽  
Vol 10 (8) ◽  
pp. 309 ◽  
Author(s):  
Andreas Laustsen ◽  
Netty Dorrestijn
Keyword(s):  
Monoclonal Antibodies ◽  
Drug Development ◽  
Treatment Modality ◽  
Development Process ◽  
Tropical Disease ◽  
Neglected Tropical Disease ◽  
Clinical Testing ◽  
Novel Treatment ◽  
Modern Drug

Snakebite envenoming is a neglected tropical disease that requires immediate attention. Conventional plasma-derived snakebite antivenoms have existed for more than 120 years and have been instrumental in saving thousands of lives. However, both a need and an opportunity exist for harnessing biotechnology and modern drug development approaches to develop novel snakebite antivenoms with better efficacy, safety, and affordability. For this to be realized, though, development approaches, clinical testing, and manufacturing must be feasible for any novel treatment modality to be brought to the clinic. Here, we present engineering, manufacturing, and regulatory considerations that need to be taken into account for any development process for a novel antivenom product, with a particular emphasis on novel antivenoms based on mixtures of monoclonal antibodies. We highlight key drug development challenges that must be addressed, and we attempt to outline some of the important shifts that may have to occur in the ways snakebite antivenoms are designed and evaluated.

scholarly journals Association of the Priority Review Voucher With Neglected Tropical Disease Drug and Vaccine Development

JAMA ◽  
2017 ◽  
Vol 318 (4) ◽  
pp. 388 ◽  
Author(s):  
Nina Jain ◽  
Thomas Hwang ◽  
Jessica M. Franklin ◽  
Aaron S. Kesselheim
Keyword(s):  
Vaccine Development ◽  
Tropical Disease ◽  
Priority Review ◽  
Neglected Tropical Disease

scholarly journals 1235. On the Edge of Tomorrow: Expedited Regulatory Pathways for Anti-Infective Therapies

2020 ◽  
Vol 7 (Supplement_1) ◽  
pp. S637-S637
Author(s):  
Cem Atillasoy ◽  
Panagiotis Gourlias
Keyword(s):  
Infectious Disease ◽  
Infectious Diseases ◽  
Drug Development ◽  
Orphan Drug ◽  
Fast Track ◽  
Orphan Drug Status ◽  
Priority Review ◽  
Drug Status ◽  
Development Programs ◽  
Accelerated Approval

Abstract Background The FDA has developed expedited review programs and pathways to increase drug development for products that have a major clinical benefit. These programs include: Fast Track, Orphan Drug Status, Accelerated Approval, Priority Review, Breakthrough Therapy (BTD) and Qualified Infectious Disease Products (QIPD). Given the heightened awareness of infectious diseases--and emerging global threats, such as resistant bacteria and Ebola—academia and industry have developed and received approval for 88 new infectious disease agents. The objective of this study was to assess the use of expedited review pathways for the 88 anti-infective agents that were approved between 2001-2020. FDA Expedited Drug Development Programs Methods We analyzed the FDA Drug Approval Database entitled, “Compilation of CDER New Molecular Entity (NME) Drug and New Biologic Approvals” for anti-infective therapies that were approved after 2000. Anti-infective therapies were defined as agents that were used to treat or prevent infectious diseases and include antibiotics, antivirals and antifungals. Our analysis focused on a comparison of the percentage of approved anti-infective agents that used each of the aforementioned designations across 2 decades (2001-2010 & 2011-2020). A drug may have one, none, or multiple of these designations. Results There were significant differences in the percentage of anti-infective agents approved with priority review, fast track and accelerated approval in 2001-2010 compared to 2011-2020 (See Results Figure 1) BTD and QIDP did not exist until 2012, thus preventing comparisons between decades. QIDP • Between 2012-2020, 16 anti-infectives have been approved with QIDP. From 2017-2020, 40% (n=10) of approved anti-infectives had QIDP. Orphan Drug Status: • Between 2017-2020, 32% of anti-infectives approved have the orphan drug designation. Comparison of FDA Expedited Drug Development Programs use between 2001-2010 and 2011-2020 Conclusion Our findings indicate Priority Review and Fast Track use has increased since 2010 among anti-infective products. Additionally, our analyses indicate that since 2017 there has been increased use of Orphan Drug Status and QIDP. However, there has been limited use of Breakthrough Therapy and Accelerated Approvals. These two pathways should be increasingly considered by academia, industry and the FDA to further expedite innovative anti-infective development. Disclosures All Authors: No reported disclosures

Challenges in Evaluating Safety and Efficacy in Drug Development for Rare Diseases: A Review for Pharmacists

2020 ◽  
pp. 089719002093097
Author(s):  
Kanya K. Shah ◽  
Stephen Kogut ◽  
Angela Slitt
Keyword(s):  
Clinical Trials ◽  
Drug Development ◽  
Rare Disease ◽  
Rare Diseases ◽  
The United States ◽  
Priority Review ◽  
Pharmaceutical Companies ◽  
Federal Programs ◽  
Financial Barriers ◽  
Safety And Efficacy

A rare disease, or orphan disease, in the United States is a condition with a national prevalence of fewer than 200,000 diagnoses. As therapies for rare diseases are developed and brought to market, pharmacists should understand the challenges of drug development for rare diseases and aid in educating patients about the approval process for rare disease therapies. Developing drugs for treating rare diseases presents unique challenges in proving the drug’s safety and efficacy with adequate study design, power, and validity. Results of the clinical trials for rare diseases may be weakened by small patient populations, limited disease information, and difficulty defining end points and biomarkers. In addition to investigational barriers, pharmaceutical companies face financial barriers in justifying the investment of bringing a rare disease therapy to market. Federal programs, such as the Orphan Drug Act of 1983, expedited review, the Rare Pediatric Disease Priority Review Vouchers (RPD PRV) program, and the 21st Century Cures Act, give pharmaceutical companies motivation to develop therapies for rare diseases. The objective of this article is to provide pharmacists with an understanding of the challenges in designing clinical trials for drugs for rare diseases and discuss federal programs that address efforts to develop safe and efficacious drugs for rare diseases.

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