The US Food and Drug Administration (FDA) has issued draft guidance to clarify which pharmacogenomic study findings and data should be included in regulatory submissions for investigational new drug applications (INDs), new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 ...

The US Food and Drug Administration (FDA) has issued draft guidance on the development of local anesthetic products with a prolonged duration of effect that can last for days.   The guidance, published on 16 March 2023, is part of a larger effort to reduce the use of opioid analgesic drugs, according to FDA. “Although different local anesthetic drug products have different pharmacokinetic (PK) profiles, in general their effects last a few hours. However, the increasing...

A review of the last five years of the Parallel Scientific Advice (PSA) program between the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA) shows that the agencies received a total of 37 applications from sponsors of new medicines and accepted 70% of requests.   The PSA program, which launched in 2005, provides a mechanism for sponsors of new medicines to seek concurrent advice from regulators in the US and Europe on scientific issues invo...

The number of US generic drug approvals rebounded in 2022 after a drop in 2021. In calendar year 2022, the US Food and Drug Administration (FDA) approved or tentatively approved 917 abbreviated new drug applications (ANDAs).   In 2021 , FDA approved or tentatively approved 776 ANDAs for generic drugs, down from 948 in 2020 and 1,014 in 2019 . The latest figures are part of FDA’s Office of Generic Drugs (OGD) 2022 Annual Report . (RELATED: Generic drug approvals ...

The US Food and Drug Administration (FDA) has issued draft guidance for industry on the development of drugs to treat neovascular or “wet” age-related macular degeneration, an eye disorder that can lead to blindness.   The draft guidance , issued on 24 February 2023, offers the agency’s recommendations for clinical trials of drugs and biologics, including eligibility criteria, trial design and efficacy endpoints. The document was developed jointly by the FDA’s Center ...

Surveillance of the generic drug supply chain has typically focused on the number of manufacturers of finished drug products, but that approach may overlook vulnerabilities from having a limited number of suppliers of active pharmaceutical ingredients (APIs), according to an analysis in Health Affairs .   “When there is only one API supplier for a certain generic drug, that drug will have a high risk for shortage regardless of the number of manufacturers commercializi...

Some form of data extrapolation – either full or partial – was used in making more than 63% of pediatric labeling changes in the United States between 2015 and 2021, according to a new analysis.   The findings, which were published in the Journal of Biopharmaceutical Statistics , revealed that the use of extrapolation increased the rates of new and expanded pediatric indications during the study period.   “Extrapolation is a key tool in pediatric drug development ...

The Clinical Research Data Sharing Alliance (CRDSA) is leading a demonstration project that seeks to establish best practices around the use of clinical trial and real-world data (RWD) in building supplemental control arms, as well as set standards for data provenance and patient selection methodologies when mixing data sources.   “While external control arms have been previously submitted to the [US Food and Drug Administration] in support of regulatory assessment, th...

The US Food and Drug Administration’s recent preliminary assessment that some naloxone products are safe and effective for over-the-counter (OTC) use received overwhelming support in public comments, but stakeholders also raised concerns about affordability, end-user education and the need to include products delivered via intramuscular injection. In November 2022, FDA published a preliminary assessment that nasal sprays that deliver up to 4 mg of naloxone, as well as i...

The widespread use of real-world evidence (RWE) in regulatory decisions continues to face challenges, including problems with data quality and methodologies, and a disconnect between the endpoints used by regulators and the information collected in clinical practice, according to panelists at the 2023 Innovations in Regulatory Science Summit sponsored by the UCSF-Stanford Center of Excellence in Regulatory Science and Innovation (CERSI).   “I wish we could get rid of t...

Encouraging researchers and sponsors to simplify clinical trials and employ a fit-for-purpose protocol is one way that the US Food and Drug Administration (FDA) can promote recruitment of trial populations that are racially and ethnically diverse, according to Patrizia Cavazzoni, director of the Center for Drug Evaluation and Research (CDER).   FDA has been focused on increasing clinical trial diversity, issuing draft guidance in April 2022 that calls on sponsors to de...

Past and present commissioners of the US Food and Drug Administration (FDA) say the agency needs partners in combatting public health misinformation, and industry, clinicians, patient advocates and academic leaders all have a role to play.   “Realistically, FDA needs help,” Mark McClellan, MD, PhD, who served as FDA Commissioner from 2002-2004, said at the 2023 Innovations in Regulatory Science Summit sponsored by the UCSF-Stanford Center of Excellence in Regulatory ...