Flexibility in the FDA approach to orphan drug development

Hunter, Nina L.; Rao, Gayatri R.; Sherman, Rachel E.

doi:10.1038/nrd.2017.151

Cited by 18 publications

(14 citation statements)

References 1 publication

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“…2,3 Since the introduction of the ODA, several Acts followed in the United States and worldwide to further spur orphan drug development. 4,5 Incentives included tax credits for qualified clinical trials, waivers of prescription drug user fees, priority review vouchers for drugs approved to treat pediatric rare diseases, and a potential 7 years of market exclusivity after approval for qualified orphan drug products. The US Food and Drug Administration (FDA) also provides more frequent assistance and timely guidance during drug development, and offers flexibility with the review process for drug products with orphan designations.…”

Section: Clinical Pharmacology In Drug Development For Rare Diseases ...mentioning

confidence: 99%

“…Recognizing these challenges, the US Orphan Drug Act (ODA) was enacted in 1983 to incentivize the development of drugs to treat rare diseases 2,3 . Since the introduction of the ODA, several Acts followed in the United States and worldwide to further spur orphan drug development 4,5 . Incentives included tax credits for qualified clinical trials, waivers of prescription drug user fees, priority review vouchers for drugs approved to treat pediatric rare diseases, and a potential 7 years of market exclusivity after approval for qualified orphan drug products.…”

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confidence: 99%

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Clinical Pharmacology in Drug Development for Rare Diseases in Neurology: Contributions and Opportunities

Abuasal

Ahmed

Patel

et al. 2021

Clin Pharma and Therapeutics

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Several challenges are associated with rare disease drug development in neurology. In this article, we summarize the US Food and Drug Administration’s experience with clinical drug development for rare neurological diseases and discuss clinical pharmacology’s critical contributions to drug development for rare diseases. We used publicly available information to identify and screen drug products approved for rare neurological indications between 1983 and 2019. We highlighted cases in which clinical pharmacology contributed to the evidence of drug efficacy, dose selection for pivotal clinical trials, dose optimization based on intrinsic and extrinsic factors, pharmacokinetic bridging for formulations, and efficacy bridging across different racial groups. Fifty‐one approved drug products were identified since the introduction of the Orphan Drug Act in 1983. Interestingly, the number of approvals in the last few years increased significantly, probably due to advances in genomic research and targeted drug modalities. Evaluation of dose selection in patient populations showed that in 52% of cases, the sponsors did not evaluate efficacy for more than one or two dose levels throughout the development program. Clinical pharmacology studies to evaluate the effect of intrinsic or extrinsic factors were adequately characterized in most of the applications. With the expansion of model informed drug development applications, (e.g., quantitative systems pharmacology and deep learning neural network models), the role and impact of clinical pharmacology is expected to grow exponentially in the next decade and enhance the development of novel treatment modalities for neurological rare diseases.

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Section: Clinical Pharmacology In Drug Development For Rare Diseases ...mentioning

confidence: 99%

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confidence: 99%

Clinical Pharmacology in Drug Development for Rare Diseases in Neurology: Contributions and Opportunities

Abuasal

Ahmed

Patel

et al. 2021

Clin Pharma and Therapeutics

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“…Since these medicines have converted former life‐threatening chronic diseases into manageable conditions, the focus of the pharmaceutical industry has shifted to unmet medical needs and complex diseases affecting only very small patient populations enabled by geno‐ and phenotyping leading increasingly to the personalization of medicines. For rare diseases affecting not more than 5 in 10 000 in Europe and less than 200 000 in the USA, an orphan drug development status can be designated to enable adequate return on investment budgets by specific legal measures, 19 not compromising on the required entire drug development stages and processes. In the USA, The Orphan Drug Act of 1983, has helped to get more than 600 drug substances approved by the FDA for orphan drug indications.…”

Section: Stages Of Industrial Drug Developmentmentioning

confidence: 99%

Challenges and opportunities to include patient‐centric product design in industrial medicines development to improve therapeutic goals

Timpe

Stegemann

Barrett

et al. 2020

Brit J Clinical Pharma

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In the past, drug developers in industry chose approaches mainly focusing on the drug product's efficacy, safety and quality according to the level required by regulatory expectations stipulated in guidelines, pharmacopoeia and other regulatory provisions. By putting more focus on the patient perspective, regulatory authorities are currently raising their requirements regarding successful product submissions. The increasing involvement of patients in the product development process (e.g. conduction of human factor use tests, integration of feedback from patient and patient advisory groups into clinical programmes) requires adaptations to the existing and established industrial drug development processes without compromising fast patient access to innovative therapies. This review provides an expert opinion on the emerging challenges and opportunities to implement a patient‐centric approach into new drug development programmes. The aim is to better understand the challenge of finding the right balance between bringing innovative drugs fast to the patients and to develop these in parallel in a patient‐centric product form as well as why this is an opportunity and how stakeholder parties (e.g. patients, clinicians, pharmacists, caregivers, regulators) can provide support to achieve desired outcomes.

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“…Orphan drugs are medicines used in the treatment of rare diseases, which are often associated with high treatment costs. 63 These drugs present a series of challenges regarding the development of biosimilars, including (a) the high costs of obtaining the RP for manufacturing purposes; (b) a reduced number of batches in order to determine batch-to-batch variability and to build extensive comparability data; (c) difficulties in obtaining a large enough population size for phase I and III trials; and (d) a heterogeneous population with the condition. 64 There are already some biosimilar orphans in development, ABP 959 and BOW080, which are two eculizumab-intended biosimilars.…”

Section: Ongoing Challengesmentioning

confidence: 99%

The biosimilars journey: current status and ongoing challenges

Kos

Azevedo

Neto

et al. 2018

DIC

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Biosimilar products are already approved and marketed in several countries. The Food and Drug Administration has approved ten different biosimilars, and the European Medicines Agency has approved 40. Even though this scenario has provided important experience with biosimilar products, there are still challenges and unanswered questions. Up to now, a good amount of knowledge has been gathered in order to support the importance of the totality of evidence and the construction of a biosimilarity exercise for regulatory approval. In addition, the extrapolation of indications has been proved viable when a careful analysis is performed. The models for clinical trials and the use of the most sensible populations have been extensively discussed, and there is apparent homogeneity in manufacturer choices for study designs. However, some challenges remain. The lack of regulatory harmony, especially concerning naming, the marketed intended copies, the interchangeability, and the biosimilars in orphan diseases are some of those and are the focus of discussion in this review.

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Flexibility in the FDA approach to orphan drug development

Cited by 18 publications

References 1 publication

Clinical Pharmacology in Drug Development for Rare Diseases in Neurology: Contributions and Opportunities

Clinical Pharmacology in Drug Development for Rare Diseases in Neurology: Contributions and Opportunities

Challenges and opportunities to include patient‐centric product design in industrial medicines development to improve therapeutic goals

The biosimilars journey: current status and ongoing challenges

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