• RF Quarterly

    A history of expedited pathways: Breakthrough therapy designation, PRIME, Sakigake, and ILAP

    It can take at least a year to 18 months to get a new medicinal product approved in the US, European Union, or United Kingdom. Over the last decade, regulators have introduced initiatives to shorten the time to market for medicines that address seriously debilitating and life-threatening conditions. This article reviews the initiation and achievements of US Food and Drug Administration’s (FDA’s) breakthrough therapy designation (BTD), the European Medicines Agency’s (EMA) ...
  • Regulatory NewsRegulatory News

    FDA explains when it will rescind breakthrough designations

    The US Food and Drug Administration (FDA) on Thursday released draft guidance explaining its thought process for rescinding breakthrough therapy designation (BTD) during its evaluation of a drug.   While FDA has long asserted its authority to rescind or withdraw BTDs, the new guidance will add clarity for sponsors as to when the agency might revoke the designation. Since designation was created under the Food and Drug Administration Safety and Innovation Act in 2012,...
  • Regulatory NewsRegulatory News

    First in vitro diagnostic for early Alzheimer’s detection gets FDA blessing

    The US Food and Drug Administration (FDA) has authorized the first in vitro diagnostic to help diagnose early Alzheimer’s disease. The agency said the test minimizes radiation risks to patients who otherwise would need to be tested with positron emission tomography (PET) scans.   On 4 May, FDA announced it has given the green light to Fujirebio Diagnostics’ de novo application for its Lumipulse G β-Amyloid Ratio (1-42/1-40) test, which is intended for patients ages...
  • Feature ArticlesFeature Articles

    January’s Regulatory Focus: Leadership in RA, expanding diversity, and more

    Feature articles during January focused on regulatory leadership, a novel approach to hiring regulatory professionals, expedited programs for reducing time to approval for certain drugs, and the Cuban biotechnology industry and its response to COVID-19. Keywords – assessment aid, breakthrough therapy, Cuba, diversity, ILAP, leadership, PRIME   Leadership, hiring, and expanding diversity Effective leadership in regulatory affairs is rooted in an understanding a...
  • Regulatory NewsRegulatory News

    FDA: Device breakthrough program surpassed expectations, while novel device authorizations fell in 2021

    In its 2021 annual report, the US Food and Drug Administration’s (FDA) Center for Devices and Radiological Health (CDRH) said that it cleared, approved or authorized a record 13 devices with breakthrough status, though the number of novel devices it authorized fell from its peak last year.   The report also discusses the center’s actions to address device shortages, thanks to new funding from Congress, as well as its COVID-19 pandemic response, patient engagement and d...
  • Feature ArticlesFeature Articles

    Expedited pathways: Breakthrough therapy designation, PRIME, and innovative licensing & access

    Abstract Over the last decade, regulators have introduced initiatives to reduce time to approval for medicines that address an unmet medical need. This article compares the US Food and Drug Administration’s (FDA’s) breakthrough therapy designation (BTD), the European Medicine’s Agency’s (EMA’s) PRIority MEdicines (PRIME) scheme, and the innovative licensing and access pathway (ILAP) from the UK’s Medicines and Healthcare products Regulatory Agency (MHRA) and reviews th...
  • Regulatory NewsRegulatory News

    Not Quite a Breakthrough Device, FDA Introduces New Safer Technologies Program

    The US Food and Drug Administration (FDA) on Wednesday released draft guidance to introduce a new, voluntary program for certain medical devices and device-led combination products that are expected to improve the safety of currently available treatments or diagnostics but are used for morbidities and mortalities less serious than those eligible for the Breakthrough Devices Program. Devices that may be included in this Safer Technologies Program” or “STeP” may include t...
  • Regulatory NewsRegulatory News

    FDA-EMA Workshop Discusses Quality Challenges for PRIME, Breakthrough Therapies

    Last November, officials from the European Medicines Agency (EMA) and US Food and Drug Administration (FDA) met with industry representatives in London to discuss the various quality challenges that arise when the development of investigational products is accelerated. EMA’s launch of its priority medicines (PRIME) scheme and FDA’s breakthrough therapy designation have enabled companies to speed certain treatments to market, although there have been difficulties in ...
  • Regulatory NewsRegulatory News

    CMS Plots to Increase Breakthrough Device Payments

    The Centers for Medicare and Medicaid Services (CMS) late Tuesday proposed a new rule that would increase payments for medical devices designated by the US Food and Drug Administration (FDA) as breakthrough devices. CMS explains how at the time of approval for these devices with a breakthrough designation, real-world data on outcomes in different patient populations is often limited. So, it can be challenging for device firms to meet the requirement for demonstrating a ...
  • Regulatory NewsRegulatory News

    510(k) Modernization: FDA Floats Need to Pursue New Authority

    Plans at the US Food and Drug Administration (FDA) to “modernize” the most commonly used pathway for new medical device clearances “might require new authority,” FDA leadership said on Monday. FDA Commissioner Scott Gottlieb and Jeff Shuren, director of FDA’s Center for Devices and Radiological Health (CDRH), issued a joint statement to announce 2018 progress on novel device approvals, pointing out that the breakthrough 510(k) is one of two submission types that was new...
  • Regulatory NewsRegulatory News

    Industry Calls for Metrics on CDRH’s Breakthrough Device Program

    A consensus of industry participants at a US Food and Drug Administration (FDA) webinar on Thursday pressed for metrics on the medical device priority review program run by FDA’s Center for Devices and Radiological Health (CDRH). The webinar led by Maureen Dreher, policy analyst at CDRH’s Office of Device Evaluation, underscored the need for additional transparency around the Breakthrough Device Program. This request was supported across several participants during the ...
  • Regulatory NewsRegulatory News

    FDA Finalizes Breakthrough Device Guidance, Outlines Safer Technologies Program

    The US Food and Drug Administration (FDA) on Tuesday finalized guidance detailing its breakthrough devices program and announced plans to bring safer medical device alternatives to market via a new safer technologies program (STeP).   “We continue to encourage device manufacturers to consider the breakthrough devices program for innovative, lifesaving devices, and, in the near future, our goal is to have STeP as an option for those devices with new advances and adaptat...