Achieving health equity – the opportunity for all patients to live a healthy life, irrespective of their circumstances – requires the collective effort of the entire medical product development ecosystem. An example of such a collaboration took place at the September 2022 RAPS Convergence in Phoenix, Arizona, with joint presentations by representatives from the US Food and Drug Administration (FDA), Health Canada, and industry (Pfizer) who discussed enhancing diversity in ...

For most of regulatory science’s established history, regulations governing drug safety and efficacy were established on a country-by-country basis. For instance, a US-centric framework was appropriate when most US-manufactured drugs were distributed and sold largely on the US market. Today, the pharmaceutical industry’s expanding global reach means drug distribution is multinational, yet the current regulatory frameworks still are largely national or regional in focus. Th...

Pediatric oncology patients continue to have significant unmet medical need. Although the Pediatric Research Equity Act (PREA) mandated evaluation of new drugs for pediatric patients, the rarity and uniquity of pediatric cancers allowed for waivers or exemptions from PREA requirements. The passing of FDA Reauthorization Act amendments in 2017 included the Research Acceleration for Cure and Equity Act, which expands the scope of oncology products subject to PREA. Sponsors d...

A three-pronged plan to modernize the US Food and Drug Administration’s (FDA) approach to data was unveiled Wednesday, building on the agency’s previous technology modernization effort.   In a blog post announcing the new Data Modernization Action Plan (DMAP), Acting FDA Commissioner Janet Woodcock and Principal Deputy Commissioner Amy Abernethy write that data modernization is the “next step” for the agency after its 2019 Technology Modernization Action Plan (TMAP)...

As hormonal drugs to prevent pregnancy have evolved over the years, the US Food and Drug Administration (FDA) on Thursday drafted new guidance for drug developers on the recommendations for clinical trials designed to establish clinical effectiveness and safety for such products. In terms of key considerations for clinical trial design features, the six-page draft begins by explaining the recommended enrollment criteria for nonpregnant, premenopausal woman in trials, wh...

The European Medicines Agency (EMA) on Monday released recommendations from its European network of pediatric research (Enpr)-EMA working group on opportunities during various stages of development to consult with pediatric research groups.   “Enpr-EMA suggests that all companies consider using these opportunities in a spirit of shared learning about the best way to collaborate during [pediatric investigation plan] planning,” EMA writes, noting that companies can benef...

The Senate’s tax plan, running counter to the House’s call to eliminate the orphan drug tax credit for research on drugs to treat rare diseases, seeks to modify the credit instead. The proposal would limit what level of expenses will qualify and limit qualified clinical expenses if they are related to the use of a drug which has previously been approved for diseases or conditions affecting more than 200,000 persons in the US. The tax credit is part of a law from...

Ten years ago, the European Commission created a new regulation as part of an effort to try to encourage more pediatric research. But in the years since, companies have been slow to increase their development of treatments for pediatric populations, particularly in oncology, where many of the medicines used were developed in the 1990s, “if they exist at all,” an EC report released Thursday said. The report highlighted that the number of completed pediatric investig...

The US Food and Drug Administration (FDA) on Friday released a plan submitted to Congress for how it will use $500 million in potential new funding from the 21st Century Cures Act over the next nine fiscal years. The plan, similar to a proposal released in May, would see about $185 million of the total go to updates and modernizing FDA’s regulation of combination products, developing FDA standards and consensus definitions to support the development and revie...

The UK’s Medicines and Healthcare products Regulatory Agency (MHRA) on Friday released a business plan for the upcoming year, noting its primary focus on developing a consensus around a proposed model for the future of the country’s regulation of medicines and medical devices following its departure from the EU. MHRA is pushing for a more international strategy that focuses on collaboration and engagement with key partners and stakeholders and continuing to partici...

A Chinese manufacturer of the active pharmaceutical ingredient (API) for the birth control drug levonorgestrel has been placed on the US Food and Drug Administration’s (FDA) import alert list, causing the World Health Organization (WHO) to scramble to find a replacement. "At the moment, there is no alternative levonorgestrel API which has been prequalified," WHO said Wednesday. When an API is prequalified by WHO, it can be used for the manufacture of finished pharmaceuti...

This article summarizes 10 years of experience with paediatric regulations in the EU. Introduction Almost 10 years ago, on 27 January 2007, new Paediatric Legislation (PL) came into force that reshaped the regulatory landscape for paediatric medicines in Europe. 1, 2 The objective of this Paediatric Regulation (PR) is to improve the health of children in the European Union (EU) by: facilitating the development and availability of medicines for children from birth ...