Pharma/Biotech – USA, Europe Regulatory News, Jan 2023 Vol. 1

EU

EMA recommendation paper: Safety, data integrity key to decentralized trial conduct

The European Medicines Agency (EMA), European Commission (EC), and Heads of Medicines Agencies (HMA) recently acknowledged the growing role of decentralized elements in the conduct of clinical trials and offered recommendations for their use, with a focus on ensuring trial participant safety and data integrity. Clinical trials of medicine products already have adopted several decentralized elements, including electronic diaries, wearables, phone calls, and online appointments. However, the appropriate use of these elements is dependent on the type of clinical trial, the trial population, the disease being treated, the trial participant’s condition, the type of medical product, as well as the product’s characteristics and stage of development, the agencies wrote

European Commission moves to simplify EMA fee legislation

The European Commission is planning to update and simplify the legislation on fees charged to drug and medical device companies, aiming to ensure that fees better reflect the underlying costs of the work done and coordinated by the European Medicines Agency (EMA). In 2019, the Commission ran a consultation on plans to change the fee legislation that drew responses from organizations including EMA. The consultation and broader review of the current legislation found an array of problems with the existing system, including misalignment of fees with the underlying costs of the work, rigidity that makes it hard to keep pace with innovation and complexity that creates unnecessary burdens. The Commission found the issues are a challenge to EMA’s financial sustainability. The Commission identified four potential ways to reform the system, ranging from only making changes to resolve problems created by the Veterinary Medicinal Products Regulation though to the introduction of a cost-based fee system for all activities and the significant simplification of the fee structure.

Clinical Trials Information System (CTIS) – Sponsor Handbook

EMA developed CTIS sponsor handbook in collaboration with representatives of industry stakeholders. The Handbook addresses key questions on CTIS and provides a compilation and references to key guidance, technical information, recommendations, training materials, and supportive documentation to facilitate the submission and assessment of CTAs and additional information during the lifecycle of a trial.

Order on fees for pharmaceuticals and pharmaceutical companies by Danish medicines agency

Danish medicines agency published a document regarding fees for pharmaceuticals and pharmaceutical companies from January 1, 2023. This document includes fees for approval of medicinal products, fees for permits for handling medicines, fees for new marketing authorization and extension of marketing authorization, fees for variations, fees for applying for a permit for or registering businesses as well as annual fees for approved companies.

US

FDA Revises Guidance on Controlled Correspondence for Generics

The FDA has updated its draft guidance for generic drug makers on submitting controlled correspondence to the agency to include new review goals under the reauthorization of the Generic Drug User Fee Amendments (GDUFA III).

New FDA Guidance on Controlled Correspondence for Generics

The FDA has updated its draft guidance for generic drug makers on submitting controlled correspondence to the agency to include new review goals under the reauthorization of the Generic Drug User Fee Amendments (GDUFA III). The 26-page draft updates a December 2020 draft guidance by revising the number of days the FDA has to respond to level 1 – general requests for information – and level 2 – inquiries about the content and approach to a specific abbreviated new drug application (ANDA). The FDA agreed to respond to 90 percent of level 1 controlled correspondence within 60 calendar days of the date of submission. The agency also pledged to review and respond to 90 percent of level 2 controlled correspondence within 120 calendar days of the date of submission.

Omnibus Spending Bill Includes Accelerated Approval Reforms, Increased FDA Budget

Many of the riders and amendments left on the cutting room floor in September when Congress passed user fee legislation have now been taken up again in a year-end omnibus spending package, a draft of which was released this week.

FDA Updates Guidance on Responding to Complete Response Letters for ANDAs

The FDA has revised a guidance issued in July on failure to respond to a Complete Response Letter (CRL) from the agency for an abbreviated new drug application (ANDA), updating it to include commitments it made for reauthorization of the Generic Drug User Fee Amendments (GDUFA III).

Biden Administration Proposes Change to Generic Drug Coverage Under ACA

The Biden Administration will seek a change in the coverage of generic medicines in plans offered through the Affordable Care Act (ACA) marketplace, according to a notice issued by HHS.

Take FDA Untitled Letters Seriously, Advises Former Agency Compliance Director

An “untitled” letter from the FDA requires the same level of response as a warning letter, says one former FDA compliance director.

FDA Targets Specific Homeopathic Drug Categories

The FDA plans to prioritize its enforcement and regulatory actions against specific homeopathic drug categories that pose a significant risk to public health, the agency said in a final guidance. The guidance, which includes only minor revisions to a previous draft issued in October 2019, says the agency will take a risk-based approach towards the manufacturing, distribution and marketing of these products, and will focus on products marketed without the necessary FDA approval. “For example, injectable drug products and ophthalmic drug products in general pose a greater risk of harm to users because the routes of administration for these products bypass some of the body’s natural defenses,” the agency said.

FDA’s new real-world evidence program offers early agency feedback

The US Food and Drug Administration is aiming to improve the quality of real-world evidence (RWE) used in regulatory submissions through a new program offering early agency meetings – before full protocol development or study initiation – for sponsors using RWE in medical product development. The Advancing RWE Program, first announced in October 2022, will provide a small number of sponsors with early, iterative feedback on RWE-based proposals that are intended to support new labeling claims or to satisfy postapproval study requirements, according to the FDA (RELATED: FDA starts PDUFDA VII programs for real-world evidence, innovative trial designs, Regulatory Focus 19 October 2022).

US FDA sees boost in FY23 budget proposal but no diagnostics reform

US lawmakers have negotiated an FY 2023 budget that would increase funding to the US Food and Drug Administration (FDA) by more than $226 million over last year’s budget. While the proposal includes several ancillary measures, it notably does not include a diagnostic reform bill that the agency and other stakeholders sought. Congressional leaders proposed a fiscal year 2023 omnibus spending bill of $1.7 trillion on 20 December that includes $6.6 billion for the FDA; of that, $3.5 billion is appropriated funding while the rest will be collected from user fees from regulated industries. In all, the agency would get $226 million more in discretionary funding as compared to last year’s budget, an increase of more than 6.5%. Almost $43 million is allocated to medical product safety, $41 million for food safety, $121 million for cross-cutting initiatives, and $21 million for infrastructure investments.

USP, Phlow say new labs will bolster continuous manufacturing

The United States Pharmacopeia (USP) and Phlow Corporation on Monday opened new laboratories at the Advanced Pharmaceutical Development Center that the pair say could spur the uptake of continuous manufacturing in the pharmaceutical industry. Officials at the labs’ unveiling promoted the ability of continuous manufacturing to increase drug quality and improve the resilience of the drug supply chain alongside Virginia Secretary of Commerce and Trade Caren Merrick. The alliance will expedite Phlow’s application of continuous manufacturing to produce essential drugs that are in shortage, while USP’s role is to test the final products produced from this mode of manufacturing, said a joint announcement.