Benefit-risk (BR) assessment, a central part of regulatory decision making, has become increasingly structured in response to the need for consistency and transparency, and in alignment with the US Food and Drug Administration’s (FDA’s) 2021 draft BR guidance. Early planning and established processes are critical to successful execution. To help regulatory affairs professionals guide clinical development teams in planning and conducting patient-focused structured BR assess...

Patient-provided information (PPI) and patient-generated health data (PGHD) are critical sources of real-world data and evidence. Collecting this data empowers patients to become partners in informed decision making by all healthcare stakeholders. However, the increased demand for this type of data has given rise to logistic, technological, and ethical challenges. Technologies and operating systems such as the high-performance integrated virtual environment (HIVE) could si...

After the US Food and Drug Administration (FDA) solicited comments on an educational program for a new Empower Rare Disease Drug Developers (LEADER 3D) initiative in the Accelerating Rare disease Cures (ARC) program, nearly two dozen stakeholders offered feedback on what topics they believe are important for inclusion in educational materials on rare disease drug development.   FDA’s Center for Drug Evaluation and Research (CDER) asked for comments from rare disease st...

There’s no one correct path into regulatory affairs.  If anything, one of the most common ways to get into regulatory affairs is “by accident.” That’s how Eleanor Panico, director of regulatory affairs at Eisai, describes the beginning of her regulatory affairs journey.  Eleanor joined us at RAPS Convergence 2022 in Phoenix for a chat about the regulatory profession, her path into it and much more. This interview has been lightly edited for clarity.  Why do you thin...

The head of the National Cancer Institute’s (NCI) investigational drug research branch said that recent draft guidance from the US Food and Administration (FDA) on finding the optimal dosage for cancer drugs could slow drug development. On the other hand, several stakeholders praised the guidance while asking for additional details such as how to factor in dosing for multi-drug treatments.   Earlier this year FDA published a draft guidance to help sponsors identify the...

The US Food and Drug Administration (FDA) has issued draft guidance to clarify which pharmacogenomic study findings and data should be included in regulatory submissions for investigational new drug applications (INDs), new drug applications (NDAs) and biologics license applications (BLAs).    The guidance, issued on 16 March 2023, also provides recommendations to sponsors on the format and level of detail for reporting pharmacogenomic data submissions, which will vary ...

Welcome to Regulatory Reconnaissance, your regulatory news and intelligence briefing.   In Focus: US FDA May Authorize Additional Covid-19 Booster Shots ( WSJ ) ( Reuters ) US FDA to take steps to help gene therapies get accelerated approval ( Reuters ) ( STAT ) ALS drug rekindles accelerated approval debate ( Politico ) Thermo Fisher, Celltrion vie for Baxter's biopharma unit ( Reuters ) Moderna expects to price its COVID vaccine at about $130 in the US ( Reuters...

Japan’s Pharmaceuticals and Medical Devices Agency (PMDA) has set out the safety control measures it plans to implement in response to the launch of generic versions of Bristol Myers Squibb’s blood cancer drug Revlimid (lenalidomide).   The dispensing of lenalidomide Japan is covered by the RevMate control procedures because the drug molecule has a similar chemical structure to thalidomide and was shown to cause birth defects and abnormalities in animal studies. Japan’...

In a nondescript building in North Bethesda, MD, the US Food and Drug Administration (FDA) houses a cybersecurity control center responsible for protecting its digital assets. Since the beginning of the COVID-19 pandemic, the agency said it has seen a sharp uptick in cyber threats, which it said have  been successfully thwarted in part due to its recent modernization efforts.   FDA’s Cybersecurity and Infrastructure Operations Coordination Center (CIOCC) monitors, anal...

The International Council for Harmonisation (ICH) last week announced that its S12 guideline on nonclinical biodistribution (BD) considerations for gene therapy products has reached Step 4 of the ICH process, meaning it’s ready for regulators to adopt in their jurisdictions.   The guideline provides considerations for the design, timing and conduct of preclinical BD studies, and offers various recommendations for sponsors on the animal species or model to be used, the ...

Welcome to Regulatory Reconnaissance, your regulatory news and intelligence briefing.   In Focus: US California Picks Generic Drug Company Civica to Produce Low-Cost Insulin ( KHN ) ‘A safe place to fail’: ARPA-H chief on building a culture of risk-taking in science ( STAT ) Moderna CEO made $398 million in 2022, but still pledges to give most to charity ( STAT ) FDA staff says safety issues with Biogen's ALS drug to not prevent approval ( Reuters ) Wyoming govern...

Welcome to another installment of This Week at FDA, your weekly source for updates – big and small – on FDA, drug and medical device regulation, and what we’re reading from around the web. This week, FDA released more details on its FY2024 budget request, Pfizer announced it is recalling millions of packages of a migraine drug over concerns about its packaging and FDA advisors back the approval of Pfizer’s COVID-19 drug Paxlovid.   FDA’s nearly 300-page FY 2024 budget ...