Susan Haigney

FDA published two draft guidance documents on May 17, 2023 that provide recommendations to support pediatric drug approvals under the Pediatric Research Equity Act (PREA) and/or the Best Pharmaceuticals for Children Act (BPCA). The documents replace the draft guidance, 

How to Comply with the Pediatric Research Equity Act

Regulatory Considerations–Complying With the Pediatric Research Equity Act and Qualifying for Pediatric Exclusivity Under the Best Pharmaceuticals for Children Act 

was written to assist sponsors in complying with pediatric study requirements under the PREA. It also describes how to qualify for pediatric exclusivity and protections under BPCA. Provisions in the BPCA were extended to biologics in 2010.

Pediatric Drug Development Under the Pediatric Research Equity Act and the Best Pharmaceuticals for Children Act: Scientific Considerations 

helps sponsors develop data and information to support pediatric drug approvals. Selected clinical, scientific, and ethical issues are addressed.

Articles

The two draft guidance documents provide recommendations for the approval of pediatric drugs, biologics, and vaccines under the Pediatric Research Equity Act and the Best Pharmaceuticals for Children Act.

FDA Issues Guidance on Pediatric Drugs

The European Medicines Agency’s Pharmacovigilance Risk Assessment Committee (PRAC) met on May 10–12, 2023 to review medicines containing hydroxyprogesterone due to safety and effectiveness concerns. Hydroxyprogesterone caproate is available in the European Union and is given by injection to prevent pregnancy loss or premature birth. These medicines are also given in some countries to treat various gynecological disorders, according to the agency.

The French medicines agency requested PRAC review the medicines after a study suggested patients exposed to the drugs may have an increased risk of cancer that would increase when the drug was used in the first trimester of pregnancy and with the number of injections.

PRAC also issued a reminder about the risks associated with the use of fluoroquinolone antibiotics and said they should not be used to treat infections that might get better without treatment or to treat non-bacterial infections or mild bacterial infections. The restrictions imposed on these medicines were a result of a study that showed the risk of disabling, long-lasting side effects.

The agency’s safety committee is reviewing hydroxyprogesterone medicines and issuing a reminder on safety issues regarding fluoroquinolone antibiotics.

EMA Issues Drug Safety Reminders

On May 17, 2023, the European Medicines Agency announced it has published a guidance document that recommends good practices for securing the supply of medicines and preventing shortages. The document discusses the stakeholders involved in the supply chain and their responsibilities in preventing drug shortages. There are 10 recommendations for minimizing drug shortages. These are:

Informing national competent authorities of potential and actual shortages

Establishing prevention and shortage management plans

Communicating with supply chain stakeholders

Promoting fair and equitable distribution of medicines.

“Medicine shortages are a global health problem and are increasingly affecting European countries. Shortages can lead to medicine rationing and delay in critical treatments, with a significant impact on patient care. In addition, patients may need to use less effective alternatives and face an increased risk of medication errors. Ensuring the availability of authorized medicines in the European Union (EU) is a key priority for EMA and the European medicines regulatory network,” the agency stated in a press release.

The agency has published guidance on good practices for securing the supply of medicines.

EMA Guidance Looks to Prevent Drug Shortages

The European Medicines Agency published its Annual Report 2022 on May 15, 2023, outlining some of the agency’s milestones achieved in 2022 and highlights from its evaluation and monitoring of medicines. The new digital report also features advanced functionalities so readers can better explore documents and audio-visual materials.

COVID-19 and other health emergencies, such as the monkeypox outbreak, were a focus for the agency in 2022, with new vaccines and treatments approved. The report details activities EMA performed to reinforce its crisis preparedness and management as a result of these emergencies.

According to the report, EMA authorized 89 medicines in 2022 that included 41 new APIs. There were also 269 orphan drug applications received by the agency with 176 given the designation. There were also 51 requests for advanced therapy medicinal products, of which six were recommended for authorization.

“EMA’s annual report also draws attention to other major achievements of the [a]gency, high-impact activities, and challenges in 2022. These include the implementation of the Clinical Trials Regulation, which entered into application in January 2022, as well as the successful launches of the Clinical Trials Information System (CTIS) and the Accelerating Clinical Trials in the EU (ACT EU) initiative, which are remodeling the way clinical trials in Europe are initiated, designed, and run. Additionally, the report presents EMA’s initiatives aimed at driving transformation in regulatory decision-making by building capability and capacity in the analysis and use of data and real-world evidence,” the agency stated in a press release.

Check out the report for more on EMA’s accomplishments in 2022.

The report outlines highlights from the agency’s medicines evaluations and milestones for 2022.

EMA Publishes Annual Report for 2022

 that it, along with other European regulatory agencies, is ending COVID-19 business continuity measures after the “unprecedented operational challenges posed by the pandemic.” EMA introduced its business continuity plan in 2020 to harness resources for combatting the COVID-19 pandemic. The plan was originally put in place as a response to Brexit and the agency’s relocation from the United Kingdom to the Netherlands.

The continuity plan ensured that EMA, the European Union Member States, and the European Commission could continue to operate while prioritizing COVID-19 actions, such as vaccines and treatment approvals. In addition, activities that were suspended due to the pandemic are gradually being reinstated.

“Throughout the pandemic we proved time and again that public health was our priority and ensured that patients in Europe could continue to have access to the newest, highest-quality therapeutics and vaccines,” said Emer Cooke, EMA’s executive director, in a press release. “Now that the peak of the pandemic has passed, the time is right for EMA and the network to close our business continuity measures. Our approach during the pandemic ensured that we could accelerate the assessment of therapeutics and vaccines for COVID-19 in an unprecedented manner while keeping assessment times for non-COVID products on schedule.”

EMA and European regulators are lifting their COVID-19 business continuity measures as the pandemic’s peak ends.

Europe Lifts COVID-19 Business Continuity Status

FDA and the United States Pharmacopeia cosponsored the 

APEC Medical Product Supply Chain Dialogue event from April 25–26, 2023

. The event, which was conducted both in-person and virtually, featured a range of topics regarding the medical products supply chain in the Asia-Pacific region and worldwide. Experts from FDA, USP, the World Health Organization, various regulatory bodies, industry, and academia spoke about vulnerabilities in the supply chain including the pandemic, elemental impurities, and ingredient quality, as well as a variety of proposed solutions to strengthen the supply chain.

On Wednesday, pandemic preparedness was discussed by representatives of Chile’s Institute of Public Health, Duke-National University of Singapore Centre, the Brazilian Health Regulatory Agency, and Peru’s General Directorate of Medicines, Supplies and Drugs.

John CW Lim, executive director, Duke-National University of Singapore Centre of Regulatory Excellence; chairman of Consortium for Clinical Research & Innovation Singapore; and chair, USP Asia Pacific Regional Chapter, stressed how the pandemic highlighted the importance of real-world data and that new technologies should define how the industry moves ahead. He also discussed the importance of having the patient’s voice in decision making and involving stakeholders from all economic areas.

Lim’s talk echoed similar comments from other experts: communication and collaboration are key in securing the supply chain. Regulatory authorities must continue to work together and involve all sections of government in decision making. The importance of trust was a common theme in the talks.

In a Wednesday afternoon session, “Safeguarding Patients from Impurities in the Drug Supply Chain,” Jason Bunting from FDA’s Center for Drug Evaluation and Research (CDER) and Gary Condran, manager, Quality Risk Management and Operations Division, Bureau of Pharmaceutical Sciences, Pharmaceutical Drugs Directorate, Health Products and Food Branch, Health Canada, gave some background and an update to the nitrosamines contamination problem. One takeaway from this discussion was how complicated and ongoing the problem is, with regulators and industry continuing to work on better testing, monitoring, and risk assessment in pharmaceutical manufacturing. Manufacturers must look at their process and products and assess risks with help by regulators and pharmacopeias. They also need to look at potential compounds they might create, and if they are a safety risk, according to the panelists.

Once again, the overall theme of these discussions focused on harmonization, information sharing, and communication to address the various supply chain problems. The pandemic and the nitrosamines problem have highlighted the importance of working together.

For more information on the event, visit 

The event, cosponsored by FDA and USP, hosted regulators and industry leaders to discuss the challenges in strengthening the medical product supply chain.