The US Food and Drug Administration (FDA) on Monday finalized guidance to assist in the clinical development of treatments for amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease. The 11-page guidance, which finalizes a draft version from February 2018, includes information on general development, safety and effectiveness considerations, as well as specific effectiveness trial considerations. The guidance incorporates parts of a guidance put toget...

As part of work to implement the optional Safety and Performance Based Pathway for medical devices, the US Food and Drug Administration’s (FDA) Center for Devices and Radiological Health (CDRH) on Thursday issued four draft guidance documents identifying performance criteria and testing methodologies for certain devices within four class II device types. CDRH also released updated final guidance explaining the new pathway and to further clarify the information FDA wil...

The US Food and Drug Administration (FDA) on Wednesday finalized guidance discussing some of the considerations that the agency will take into account when determining whether citizen petitions and petitions for a stay of agency action are submitted with the primary purpose of delaying the approval of a pending generic drug, biosimilar or 505(b)(2) application. The final guidance comes as FDA in February explained its concerns to Congress that such petitions are often...

The UK’s Medicines and Healthcare products Regulatory Agency (MHRA) on Wednesday updated its no-deal Brexit guidance for medical devices, adding a new section on the role of the “UK Responsible Person.” According to the guidance, the UK Responsible Person, who must be established in the UK, acts on behalf of a non-UK manufacturer to carry out tasks for the manufacturer, which include registering with the  MHRA  before a device is placed on the UK market. “Only a manu...

About a dozen drugmakers and industry groups recently offered their opinions on a revised US Food and Drug Administration (FDA) draft guidance related to population pharmacokinetics (PK) analyses submitted as part of new drug applications and biologic license applications. The comments largely seek specific tweaks to the 23-page revised draft, which was released in July , although some of the comments also seek further clarity and new additions to the guidance. Indu...

The US Food and Drug Administration (FDA) on Monday released 53 product-specific guidance documents to aid generic drug development, including 34 new guidance documents, 26 guidances for treatments that lack generic competition and 16 for complex products. When finalized, the guidance documents will represent the current thinking of FDA on, among other things, the product-specific design of bioequivalence studies to support abbreviated new drug applications. Among th...

The US Food and Drug Administration’s (FDA) Center for Devices and Radiological Health (CDRH) on Thursday released four final guidance documents on the Special 510(k) program, the abbreviated 510(k) program, how to format traditional and abbreviated 510(k)s and CDRH’s refuse to accept policy for 510(k)s. As part of a wider, ongoing push to modernize FDA’s 510(k) program, the final guidance documents provide more clarity on several programs the agency has been working on...

As the US Food and Drug Administration (FDA) grapples with how to use real-world data for regulatory decisions, two FDA officials wrote in a perspective published last Friday in the Clinical Journal of the American Society of Nephrology that the agency is developing guidance on data quality issues unique to the real-world data setting and related study design considerations. Aliza Thompson and Mary Ross Southworth of the Division of Cardiovascular and Renal Products w...

Acting US Food and Drug Administration (FDA) Commissioner Ned Sharpless on Thursday announced that the agency would finalize guidance on developing treatments for amyotrophic lateral sclerosis (ALS) by the end of September. The 9-page draft guidance, first released in February 2018 , incorporated parts of a draft guidance put together by the ALS Association, known for its viral "ice bucket challenge," which FDA said provided the agency "with scientific advice and insig...

The question and answer (Q&A) document released by the European Medicines Agency (EMA) on Wednesday describes when an exemption from EU batch re-testing can be granted for imported advanced therapy medicinal products (ATMPs). The three-page Q&A begins by noting that a Qualified Person (QP) has to ensure each batch of imported ATMP is re-tested upon importation. But the QP can certify that the imported batch relies on controls conducted in a third country if the product ...

The US Food and Drug Administration (FDA) on Wednesday added three new questions and answers to its Laboratory Controls questions and answers (Q&A) on Current Good Manufacturing Practices (CGMP) guidance. The new Q&As discuss instrument calibration standards for chromatographic systems, system suitability and trial injections.   On the issue of trial injections, where a sample of a lot is injected into the chromatographic system with the intention of obtaining an unoff...

The US Food and Drug Administration (FDA) on Tuesday updated draft guidance from July 2015 to address public comments and reflect the agency’s current thinking on the development of clinical outcome assessment measures and statistical considerations to assess primary and secondary efficacy endpoints of clinical trials for gastroparesis. The nine-page draft explains: “Until a well-defined and reliable PRO [patient-reported outcome] instrument that measures all the clinic...