Quantum of Effectiveness Evidence in FDA’s Approval of Orphan Drugs: Update, July 2010 to June 2014

Sasinowski, Frank J.; Panico, Erika B.; Valentine, James

doi:10.1177/2168479015580383

Cited by 37 publications

(21 citation statements)

References 2 publications

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“…The FDA recognizes the unique challenges associated with rare disease drug development and has exercised flexibility in applying the statutory standards for approval of these drugs. 13 Regulatory flexibility is an important factor in rare disease drug development because it allows for a customized approach to the FDA's review, as well as the drug development and approval process. Enhanced transparency and communication between sponsors and the FDA enable the exercise of flexibility during drug development.…”

Section: Discussionmentioning

confidence: 99%

Pre—Investigational New Drug Meetings With the FDA: Evaluation of Meeting Content and Characteristics of Applications for New Drug and Biologic Products

Pariser

2015

Ther Innov Regul Sci

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Section: Discussionmentioning

confidence: 99%

Pre—Investigational New Drug Meetings With the FDA: Evaluation of Meeting Content and Characteristics of Applications for New Drug and Biologic Products

Pariser

2015

Ther Innov Regul Sci

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“…The National Organization for Rare Diseases catalogued the flexibility exhibited by the FDA in approving drugs against rare disease. 4 We applaud the FDA for weighing the clinical significance and not being subservient to statistical methodology. Would the draft rare disease Guidance suggest to sponsors that the FDA is open to such development paths?…”

Section: Acceptable Pathways For Registration Of Drugs Against Rare Dmentioning

confidence: 99%

Drugs Against Rare Diseases: Are The Regulatory Standards Higher?

Gobburu

Pastoor

2016

Clin Pharma and Therapeutics

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The US Food and Drug Administration (FDA) recently issued a draft Guidance for Industry for Rare Diseases: Common Issues in Drug Development (referred to as “Rare Diseases Guidance”). In our opinion, the FDA should consider: (a) explicitly acknowledging the standards are higher for rare diseases for the reasons presented in this article; and (b) illustrating innovative development pathways that may be acceptable for rare diseases, including case studies.

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“…Statutory standards for approval of orphan drugs developed for treatment of rare diseases are the same as those of common diseases: there must be substantial safety and efficacy evidence from well-controlled trials [3, 5]. However, in some cases it may not be possible to meet these standards when developing orphan drugs [2–4]; therefore, the FDA applies scientific judgment and regulatory flexibility when making decisions about drug development and approval in rare diseases [4–6]. Many orphan diseases are serious and/or life-threatening and primarily affect pediatric patients, underscoring both the challenges and urgency of effective drug development [1].…”

Section: Introductionmentioning

confidence: 99%

Regulatory strategies for rare diseases under current global regulatory statutes: a discussion with stakeholders

Mulberg

Bucci‐Rechtweg

Giuliano

et al. 2019

Orphanet J Rare Dis

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Rare or orphan diseases often are inherited and overwhelmingly affect children. Many of these diseases have no treatments, are incurable, and have a devastating impact on patients and their families. Regulatory standards for drug approval for rare diseases must ensure that patients receive safe and efficacious treatments. However, regulatory bodies have shown flexibility in applying these standards to drug development in rare diseases, given the unique challenges that hinder efficient and effective traditional clinical trials, including low patient numbers, limited understanding of disease pathology and progression, variability in disease presentation, and a lack of established endpoints.To take steps toward improving rare disease clinical development strategies under current global regulatory statutes, Amicus Therapeutics, Inc. and BioNJ convened a 1-day meeting that included representatives from the Food and Drug Administration (FDA), biopharmaceutical industry, and not-for-profit agencies. The meeting focused on orphan diseases in pediatric and adult patients and was intended to identify potential strategies to overcome regulatory hurdles through open collaboration.During this meeting, several strategies were identified to minimize the limitations associated with low patient numbers in rare diseases, including the use of natural history to generate historical control data in comparisons, simulations, and identifying inclusion/exclusion criteria and appropriate endpoints. Novel approaches to clinical trial design were discussed to minimize patient exposure to placebo and to reduce the numbers of patients and clinical trials needed for providing substantial evidence. Novel statistical analysis approaches were also discussed to address the inherent challenges of small patient numbers. Areas of urgent unmet need were identified, including the need to develop registries that protect patient identities, to establish close collaboration and communication between the sponsor and regulatory bodies to address methodological and statistical challenges, to collaborate in pre-competitive opportunities within multiple sponsors and in conjunction with academia and disease-specific patient advocacy groups for optimal data sharing, and to develop harmonized guidelines for data extrapolation from source to target pediatric populations. Ultimately, these innovations will help in solving many regulatory challenges in rare disease drug development and encourage the availability of new treatments for patients with rare diseases.

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Quantum of Effectiveness Evidence in FDA’s Approval of Orphan Drugs: Update, July 2010 to June 2014

Abstract: The findings further support that FDA has demonstrated extraordinarily reasonable flexibility in its review of certain applications for orphan drugs and reinforce the need for FDA and drug companies to better understand and discuss the various types of flexibility.

Cited by 37 publications

References 2 publications

Pre—Investigational New Drug Meetings With the FDA: Evaluation of Meeting Content and Characteristics of Applications for New Drug and Biologic Products

Pre—Investigational New Drug Meetings With the FDA: Evaluation of Meeting Content and Characteristics of Applications for New Drug and Biologic Products

Drugs Against Rare Diseases: Are The Regulatory Standards Higher?

Regulatory strategies for rare diseases under current global regulatory statutes: a discussion with stakeholders

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