FDA Facilitated Regulatory Pathways: Visualizing Their Characteristics, Development, and Authorization Timelines

Liberti, Lawrence; Bujar, Magda; Breckenridge, Alasdair; Hoekman, Jarno; McAuslane, Neil; Stolk, Pieter; Leufkens, Hubert G.M.

doi:10.3389/fphar.2017.00161

Cited by 23 publications

(25 citation statements)

References 2 publications

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“…This trend matches the acceleration of new drug approvals seen across the field of oncology as a whole [4,5], which results from the impressive progress in translational cancer research and from the evolving regulatory environment [6,7]. Together, these factors have profoundly changed the way in which new cancer drugs are discovered, studied, reviewed, and approved [8][9][10].…”

Section: Introductionmentioning

confidence: 77%

FDA and EMA Approvals of New Breast Cancer Drugs—A Comparative Regulatory Analysis

Leo¹,

Hentschel²,

Szucs

et al. 2020

Cancers

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Breast cancer is the most common cancer in women worldwide and the solid tumor type for which the highest number of drugs have been approved to date. This study examines new drug approvals for breast cancer by the United States Food and Drug Administration (FDA) and the European Medicines Agency (EMA), based on an analysis of regulatory documents from both agencies for the period from 1995 to 2018. Of the 29 breast cancer drugs approved over this time span, 17 received positive decisions from both the FDA and EMA, including all drugs licensed after 2008. Nineteen of the 25 FDA-approved drugs, but none of the EMA approvals, benefited from special regulatory pathways (such as fast track, breakthrough therapy, or priority review). In the U.S.A., four accelerated approvals were granted (of which one, for bevacizumab, was later revoked), while only two drugs received provisional approvals following EMA review. New breast cancer drugs were approved approximately twelve months earlier in the United States than in Europe. These results suggest that a broader use of special regulatory pathways by EMA could help to accelerate access to novel drugs for European breast cancer patients.

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Section: Introductionmentioning

confidence: 77%

FDA and EMA Approvals of New Breast Cancer Drugs—A Comparative Regulatory Analysis

Leo¹,

Hentschel²,

Szucs

et al. 2020

Cancers

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“…Linked with the increase in innovative clinical trial designs, multiple stakeholders, including regulators, payers, Health Technology Assessment (HTA) bodies, patient groups, and drug developers across the globe have recognized the need to develop pathways for accelerated access to innovative drugs . Regulators have developed programs to expedite drug development to bring innovative medicines to patients more quickly.…”

Section: Novel Pathways For Faster Drug Accessmentioning

confidence: 99%

Innovation in Regulatory Science Is Meeting Evolution of Clinical Evidence Generation

Lee

Möller³

et al. 2019

Clin Pharma and Therapeutics

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At the turn of the century, the pharmaceutical industry began a transition toward a focus on oncology, rare diseases, and other areas of high unmet need that required a new, more complex approach to drug development. For many of these disease states and novel approaches to therapy, traditional approaches to clinical trial design fall short, and a number of innovative trial designs have emerged. In light of these changes, regulators across the globe are implementing new programs to provide regular development program support, facilitate accelerated access, use real‐world data, and use digital tools to improve patients’ lives. Emerging market regulators are also focusing on simplifying their regulatory pathways via regional harmonization schemes with varying levels of ambition. These changes in the external environment imply that biopharma regulatory teams need to adapt and evolve, leveraging digital tools, data, and analytics, and positioning themselves as strategic advisors during development.

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“…Liberti et al assessed the impact of facilitating regulatory pathways in general and their combined use by assessment of 125 products approved by FDA between 2013 and 2015. 60 It was demonstrated that the review time decreased for therapies using one or more regulatory tools (243 days) compared to products without a special approval pathway or designation (365 days). Nevertheless, the overall median development time for designated products was not shorter when compared to undesignated products especially regarding Fast Track designated products and therapies qualified for PR.…”

Section: Value Of Expedited Programs and Their Combinationmentioning

confidence: 99%

Expediting Drug Development: FDA’s New Regenerative Medicine Advanced Therapy Designation

Vaggelas

Seimetz

2019

Ther Innov Regul Sci

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In March 2017, the US FDA introduced the new Regenerative Medicine Advanced Therapy (RMAT) designation thus recognizing the enormous potential of these medicines and the need for efficient regulatory tools to accelerate their development and their commercial availability. The development of regenerative medicines is very challenging because of their complex and unique nature, especially to the rather unexperienced small- and medium-sized developing enterprises. With the new RMAT designation, FDA aims at providing intensive support to companies developing cell- and tissue-based therapies, tissue-engineering products, and combination treatments. This may also include cell-based products where the genome has been edited by emerging technologies such as CRISPR-Cas9. This article presents the newly launched "Regenerative Medicine Advanced Therapy" (RMAT) designation, outlines existing FDA regulatory tools aiming at expediting approval, and discusses the overall value of these programs. Additionally, recommendations are provided for companies developing these very specific and complex therapies on how and when to consider these tools for an integrated development and regulatory strategy.

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FDA Facilitated Regulatory Pathways: Visualizing Their Characteristics, Development, and Authorization Timelines

Cited by 23 publications

References 2 publications

FDA and EMA Approvals of New Breast Cancer Drugs—A Comparative Regulatory Analysis

FDA and EMA Approvals of New Breast Cancer Drugs—A Comparative Regulatory Analysis

Innovation in Regulatory Science Is Meeting Evolution of Clinical Evidence Generation

Expediting Drug Development: FDA’s New Regenerative Medicine Advanced Therapy Designation

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