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

USA

FDA Seeks Comments on Proposed Consumer Rx Advertising Survey

The FDA Office of Prescription Drug Promotion (OPDP) is seeking input on its proposal to conduct research into how consumers interpret certain quantitative information about prescription drugs often included in direct-to-consumer (DTC) advertising.

FDA draft guidance aims to help sponsors develop drugs for radiation exposure

The US Food and Drug Administration (FDA) on 19 April issued draft guidance that encourages sponsors to have “early and ongoing” communication with the agency in developing drugs to prevent or treat acute radiation syndrome (ARS), in an effort to overcome some of the challenges in developing these drugs. ARS applies to a variety of clinical indications resulting from high doses of exposure to radiation.  The Centers for Disease Control and Prevention (CDC) defines ARS as “an acute illness caused by irradiation of the entire body (or most of the body) by a high dose of penetrating radiation in a very short period of time (usually a matter of 45 minutes).”

FDA officials offer CMC advice for gene therapies at OTP town hall

Officials from the US Food and Drug Administration’s (FDA) Office of Therapeutic Products (OTP) within the Center for Biologics Evaluation and Research (CBER) urged developers of gene therapies to avoid implementing manufacturing process changes during later pivotal studies to avoid problems later, such as products being out-of-specification (OOS). So asserted Graeme Price, a team lead for the Gene Therapy Branch 1 within OTP, who spoke at a virtual town hall on 25 April to answer stakeholder questions related to chemistry, manufacturing and controls (CMC) for gene therapy products as part of investigational new drug applications (INDs) and licensure applications.

FDA wants feedback on DTC quantitative claims survey

The US Food and Drug Administration’s (FDA) Office of Prescription Drug Promotion (OPDP) announced plans to conduct a study gauging respondents’ perception and understanding of quantitative claims drawn from direct-to-consumer advertisements. FDA said it conducted a literature review and found that while some types of quantitative information are well-studied, “many questions remain on how best to communicate certain quantitative information about prescription drugs.”

FDA ramps up for transition to QMSR

The US Food and Drug Administration (FDA) is working to finalize its proposal to align its device Quality System Regulation (QSR) with the international standard ISO 13485:2016, creating the Quality Management System Regulation (QMSR). In preparation for the final rule, the agency is readying for the change internally by updating its technology systems, training staff and replacing the Quality System Inspection Technique (QSIT).

FDA Offers Formatting and Content Guidelines for OMORs in Draft Guidance

In a new draft guidance, the FDA offered advice on the format and content of an over-the-counter (OTC) monograph order request (OMOR)  which allows an OTC drug covered by a monograph to be marketed without an approved drug application. Sponsors submit an OMOR to request that the agency issue a final order on whether a drug already is generally recognized as safe and effective (GRASE) or whether a change to the use of a drug is GRASE. OMOR requirements include mandatory electronic submission for five modules  administrative information, summaries, quality, nonclinical study reports and clinical study reports.

Q&A Guidance Gives Risk-Based Monitoring Advice for Sponsors

The FDA offers expanded advice for sponsors on risk-based monitoring of clinical trials in a new final guidance that updates the agency’s previous guidance on the topic issued in 2013.

FDA Issues Draft Guidance on Manufacturing Interruptions to Reduce Shortages

In a move to help prevent or mitigate shortages, the FDA is asking drugmakers to provide more information regarding manufacturing disruptions that could affect finished drugs and biological products as well as some active pharmaceutical ingredients (API).

FDA Requires Opioids Producers to Supply Mail-Back Envelopes for Disposal

In its latest move to address the opioid overdose crisis, the FDA is requiring opioid manufacturers to provide prepaid mail-back envelopes to outpatient pharmacies and other dispensers to make it easier for patients to dispose of unused opioids.

EUROPE

EMA Adopts Final ICH S12 Covering Nonclinical BD Studies for Gene Therapies

The European Medicines Agency (EMA) has become the first regulator to accept the International Council for Harmonization (ICH) S12 guideline, laying out the council’s recommendations for how to conduct nonclinical bio distribution (BD) studies when developing gene therapy products.

EU Proposes Sweeping New Drug Development Law, Pharma Industry Balks

On Wednesday, the EU released the long-anticipated, wide-ranging revision of its drug development legislation, proposing changes that would affect nearly every part of drug development and authorization and raising opposition in the pharmaceutical industry.

EMA to Resume Trial Data Publication Efforts in September

Starting in September, the European Medicines Agency (EMA) will begin gradually resuming its efforts to openly share clinical trial data for medical products, the agency reported.

EMA Adds New Elements to Its PRIME Review Pathway

The European Medicines Agency (EMA) is updating its Priority Medicines (PRIME) program to include new ways to track a product’s progress toward approval and to get more timely advice from the agency.

EU patent reform proposal addresses compulsory licensing and SPCs

The European Commission on Thursday published a package of regulations that would establish an EU-wide compulsory licensing scheme allowing other companies to make drugs without the patent holder’s consent in emergency situations and establishes community-wide supplementary protection certificates (SPCs) that would extend the patent term for pharmaceutical products for up to five additional years. Currently, SPCs are only granted at the national level. The Commission said that these proposals “will create a more transparent, effective and futureproof intellectual property rights framework.”

 EU releases draft legislation that will reshape pharma regulation

The European Commission on Wednesday published the thrice-delayed draft of the contentious legislation that will reshape the regulation of the pharma sector and set the course of the industry for years to come. After a series of delays, the draft document is now available for public scrutiny. The draft is the product of a long, heated debate into the future of the European pharma industry that at various points has seen politicians disparagingly call the delays “a huge victory for the pharmaceutical lobby” and the trade group EFPIA warn that a leaked, earlier draft would “irretrievably sabotage” the industry and “send Europe to the back of the queue for healthcare treatments, clinical research, jobs and global investment.”

EMA addresses considerations for single-arm trials

A new reflection paper from the European Medicines Agency (EMA) offers some suggestions to sponsors on how to design single-arm trials (SATs) and strategies for minimizing bias in these trials to support new marketing authorization applications (MAAs). The paper aims to “stimulate the scientific discussion around key concepts and challenges associated with single-arm trials and to improve their design and conduct,” announced EMA. Yet defining general conditions under which SATs may be considered acceptable as pivotal evidence for MAAs is outside its scope.