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...

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...

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...

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 ...

The fire stoked by rising drug prices is starting to spread through government, particularly as Sens. Susan Collins (R-ME) and Claire McCaskill (D-MO) on Wednesday announced a bipartisan Senate investigation into pharmaceutical price gouging, with letters to four companies. The letters sent Wednesday follow on the heels of HHS announcing Tuesday that it would dig into drug pricing at an invitation-only forum on 20 November. The forum will feature five panel sessions on t...

In a major move, the European Medicines Agency (EMA) announced today it is revoking or revising most product class waivers that allow companies to bypass the requirement to test new drugs in pediatric populations before they can be authorized. Background Companies are often reluctant to conduct clinical studies in children over ethical concerns and fear of adverse events occurring during trials. These reservations have created a gap in the number of products authorized ...

After weeks of heightened speculation, the European Council has agreed on its approach to new medical devices and in vitro diagnostics (IVD) legislation. Background Regulators in Europe have been discussing the need to reform the legislative framework for medical devices and IVD for many years. In the EU, the legislative process is structured so that only the EC can initiate new legislation. The European Parliament then votes on the commission's proposal and legisl...

The European Medicines Agency (EMA) is piloting a new initiative to offer early meetings with companies to foster discussions about their pediatric development strategies for new medicines. Background Companies are often reluctant to conduct clinical studies in children due to ethical concerns and fear of adverse events during trials. However, conducting clinical research on children is critical to determining whether drugs are safe and effective in pediatric population...

The Nuffield Council on Bioethics has released a report on the ethical issues surrounding clinical research involving children in the UK and EU. The report, which looks at the current status of clinical research and regulations, argues better regulation and greater involvement from children is critical to increasing the number of clinical trials involving children. Background Companies are often reluctant to conduct clinical studies on children due to ethical concerns ...

Pharmaceutical manufacturer GlaxoSmithKline is warning consumers that some bottles of its over-the-counter weight loss drug Alli (orlistat capsules) have "tampered with" in at least seven US states. News of the tampering was announced by GSK in a 26 March 2014 press release, in which the company said it was working with the US Food and Drug Administration (FDA) to determine what had happened and the extent of the problem. GSK said the tampered products had packaging tha...

In January 2013, the US Food and Drug Administration (FDA) published something of an unusual notice in the Federal Register . The notice indicated that FDA would withdraw a final rule that had been published just a few months prior, thereby permitting access to records regarding scientific research misconduct proceedings involving its staff. Now the agency has announced the reinstatement of the final rule, saying the records are now exempt from certain requirements under...

For years, regulators around the globe have been touting the challenges and benefits of globalization, explaining that a globally diverse product supply chain means needing to rely on regulatory partners to ensure that products are safe for consumption. Now UK regulators are learning a related lesson: A scandal that affects products intended for one country may well affect more than just one regulatory authority. Background On 13 May 2013, Ranbaxy Laboratories pled gui...