Orphan Drugs: Understanding the FDA Approval Process

Srivastava, Gauri; Winslow, Ashley R.

doi:10.21428/b2e239dc.381f7d37

Cited by 4 publications

(2 citation statements)

References 0 publications

Supporting

Mentioning

Contrasting

Order By: Relevance

“…During the U.S. tax reform package, the tax credit was decreased to 27.5%, and as of now, orphan drugs receive a reduced tax credit of 25% for orphan drugs. The Internal Revenue Service (IRS) gives tax credits, not the FDA 6,7 .…”

Section: Tax Credits For Clinical Trialsmentioning

confidence: 99%

Untitled

2023

IJPSR

View full text Add to dashboard Cite

Orphan drugs are used to treat rare diseases that affect fewer than 200,000 people in the United States. Development and approval of orphan drugs are challenging due to the limited patient population and substantial development costs; the U.S. Food and Drug Administration (FDA) has established several programs, such as the Orphan Drug Designation program and Orphan Product Grants program to encourage the development of medications that treat rare diseases, the rare pediatric disease voucher program supports research and growth of rare diseases that affect pediatric populations, the humanitarian use device program regulates medical devices associated with rare diseases, the main aim of these incentives is to provide financial and regulatory support to sponsors. Overall, the regulatory framework in United States offers a supportive environment for drug manufacturers in developing and approving orphan products; these initiatives ensure that patients with rare diseases have access to safe and effective treatments. United States is one of the top leaders in developing orphan drugs and continues to play a critical role in the global effort to bring new therapies to patients with this rare disease. INTRODUCTION:Orphan drugs treat rare diseases or disorders that occur in less than 200,000 people in the U.S.; orphan drugs are drugs used in the prevention, treatment, and diagnosis of rare diseases (diseases with less available information) or diseases that do not commonly occur 1 . The drugs eligible for orphan drug designation are new molecular entities (drugs newly developed for treating certain rare diseases), secondary indications (expansion of already existing symptoms, and any newly developed formulations 2 .

show abstract

Section: Tax Credits For Clinical Trialsmentioning

confidence: 99%

Untitled

2023

IJPSR

View full text Add to dashboard Cite

show abstract

“…Additionally, with the enforcement of the Rare Disease Control Act in 2016, a legal framework was established to offer administrative and financial support for the production and sale of orphan drugs. While each jurisdiction has different designation criteria and benefits for rare diseases, they generally include economic incentives, expedited reviews, and market exclusivity ( Table 1 ) (Center, K. L. I., 2018 , 2020 ; CODE, U. S., 2023 ; EMA, 2023a , 2023c ; FDA, 2022a , 2023a ; GAO, U. S., 2020 ; Huang et al, 2022 ; MHLW, 2023 ; Nagai, 2019 ; Srivastava & Winslow, 2019 ). In other words, the designation of orphan drugs entails policy support throughout the entire drug development cycle, from R&D to new drug application (NDA) to the post-marketing stage.…”

Section: Introductionmentioning

confidence: 99%

International comparison of availability for orphan drugs: focused on approved orphan drugs in South Korea

Shin,

Hong,

Lim

et al. 2024

Journal of Pharmaceutical Policy and Practice

View full text Add to dashboard Cite

In vitro tools for orally inhaled drug products—state of the art for their application in pharmaceutical research and industry and regulatory challenges

Metz

Hittinger²,

Lehr

2021

In vitro models

View full text Add to dashboard Cite

The drug development process is a lengthy and expensive challenge for all involved players. Experience with the COVID-19 pandemic underlines the need for a rapid and effective approval for treatment options. As essential prerequisites for successful drug approval, a combination of high-quality studies and reliable research must be included. To this day, mainly in vivo data are requested and collected for assessing safety and efficacy and are therefore decisive for the pre-clinical evaluation of the respective drug. This review aims to summarize the current state of the art for safety and efficacy studies in pharmaceutical research and industry to address the relevant regulatory challenges and to provide an outlook on implementing more in vitro methods as alternative to animal testing. While the public demand for alternative methods is becoming louder, first examples have meanwhile found acceptance in relevant guidelines, e.g. the OECD guidelines for skin sensitizer. Besides ethically driven developments, also the rather low throughput and relatively high costs of animal experiments are forcing the industry towards the implementation of alternative methods. In this context, the development of orally inhaled drug products is particularly challenging due to the complexity of the lung as biological barrier and route of administration. The replacement of animal experiments with focus on the lungs requires special designed tools to achieve predictive data. New in vitro test systems of increasing complexity are presented in this review. Limits and advantages are discussed to provide some perspective for a future in vitro testing strategy for orally inhaled drug products. Graphical abstract

show abstract

Orphan Drugs: Understanding the FDA Approval Process

Cited by 4 publications

References 0 publications

Untitled

Untitled

International comparison of availability for orphan drugs: focused on approved orphan drugs in South Korea

In vitro tools for orally inhaled drug products—state of the art for their application in pharmaceutical research and industry and regulatory challenges

Contact Info

Product

Resources

About