Asia-Pacific Roundup: PMDA outlines initiatives to promote pediatric drug development in Japan

The Pharmaceuticals and Medical Devices Agency (PMDA) has published details of the steps it is taking to promote the development of drugs for use in children in Japan.

PDMA's report, published in Japanese in March and translated into English in April, outlines the need for action. The agency notes “growing concerns about the expansion of ‘drug loss’ in Japan, where pharmaceuticals approved in the EU and the US are not being developed for Japanese patients.” The problem is particularly challenging for pediatric and orphan drugs, PMDA wrote.

“PMDA has decided to provide essential advice and guidance based on a comprehensive understanding of the overall drug development plan, including the pediatric drug development plan, during its clinical trial consultations and other services,” the agency wrote.

The decision follows talks led by a cross-sectoral project team to address inquiries about pediatric development status. Informed by the team’s focus on regulatory science, PMDA has summarized the basic principles and important considerations for pediatric drug development.

PMDA said it is crucial in Japan to formulate comprehensive plans that include pediatric drug development and to consider children from the early stages of adult R&D programs. Sponsors should assume Japan’s participation in multi-regional trials starting when pediatric programs are formulated to meet the US and European Union requirements. The exception is when “clear differences in intrinsic or extrinsic ethnic factors are confirmed or anticipated,” PMDA said.

“Based on these principles, PMDA will, during clinical trial consultations for adult drug development, actively verify the status of pediatric drug development and provide advice or guidance as necessary to ensure that pediatric drug development in Japan is initiated without lagging behind adult drug development,” the agency said.

Starting in Phase II, companies applying to discuss adult clinical trials should factor pediatric studies into their plans. PMDA wants applicants to say whether pediatric programs exist in Japan and overseas in their consultation briefing documents. Applicants should describe the overall pediatric development plan if their program includes children who can be evaluated alongside adults.

PMDA may need to change the basic principles and important considerations. The agency identified talks about revising Japan’s pharmaceutical and medical device regulations as an initiative that could force it to change the document. The talks, which generated a policy document in January, have covered the need to promote pediatric drug development and find solutions for drug loss. Companies seeking authorization for new drugs or indications may be obligated to plan pediatric development.

PMDA Statement

NPRA shares findings from Malaysia’s voluntary pharmacovigilance inspections

The National Pharmaceutical Regulatory Agency (NPRA) has published findings from the voluntary good pharmacovigilance practice (GVP) inspection program it ran in 2023 and 2024.

Malaysian officials began establishing GVP capabilities in 2017, leading to a call for product registration holders who were willing to undergo voluntary inspections. Thirteen companies contacted NPRA. Four were not selected because they were assessed as lower risk. The nine companies chosen for inspection included five multinational businesses.

NPRA made 20 findings related to quality management systems (QMS). The management of adverse drug reactions (ADRs) and risk management were the second and third most common findings, 15 and 12, respectively. The collection and collation of ADRs was the fourth most common finding. NPRA said this was a particular issue for local manufacturers.

The agency provided common inspection findings within each category. Common QMS issues included the lack of pharmacovigilance agreements with third parties such as distributors and the failure to audit internal and external units involved in the activities. ADR collection and collation findings included a lack of systems for surveilling populations such as breastfeeding women and for local literature screening.

NPRA also highlighted positive observations, noting that all participants showed a strong commitment to the program and most shared the requested documents within the specified timelines. Some companies have successfully developed their own pharmacovigilance systems without external guidance. NPRA said the projects show the first edition of its GVP guideline is a practical and effective framework.

NPRA Report

TGA seeks feedback on expanding use of psychedelic psilocybin in Australia

The Therapeutic Goods Administration (TGA) is gathering views on whether to reclassify psilocybin to allow the use of the psychedelic in people experiencing existential distress toward the end of their lives.

Physicians can already prescribe psilocybin, the active ingredient in magic mushrooms, for use in people with treatment-resistant depression in Australia. An applicant has asked TGA to reclassify the medicine to enable access for patients facing a terminal illness accompanied by severe existential or psychosocial suffering.

The drug would be given in medically controlled environments after at least one independent reviewing specialist doctor had confirmed the patient’s diagnosis and treatment plan. Palliative care specialists who have received specific training would prescribe the medicine.

“Given the strong clinical evidence for relief of spiritual and emotional suffering, it would be inconsistent and unethical to withhold this evidence-based therapy from mentally competent, terminally ill individuals who seek it,” the applicant said. “Treatment would be under the strict medical supervision to ensure safety and minimizing any risk of misuse or abuse.”

TGA is seeking feedback on the proposals until 21 May.

TGA Consultation

CDSCO flags ‘serious risk to public health’ from drug combinations in India

The Central Drugs Standard Control Organization (CDSCO) has said fixed-dose combinations (FDC) that have been licensed without being evaluated for safety and efficacy pose a serious risk to public health and safety in India.

Officials have been grappling with FDCs that came to market without undergoing the typical assessments for more than 10 years. Despite the long effort to stop the sale of irrational or unsafe FDCs, CDSCO is yet to eradicate the problem. Officials said it has come to the notice of the agency that drugs have been licensed without prior evaluation of safety and efficacy.

“The approval of such unapproved FDCs compromises patient safety and may lead to adverse drug reactions, drug interactions, and other health hazards due to the absence of scientific validation,” CDSCO said. “Upon issuance of show cause notices to the manufacturers, they have stated that these licenses were granted by the respective drug licensing authorities and have not violated any rules.”

CDSCO has asked state regulators to review their approval processes and ensure strict compliance.

CDSCO Notice, More

PMDA staffer notes need for global re-evaluation of biosimilar efficacy trial rules

A PMDA employee has said the re-evaluation of the need for biosimilar efficacy clinical trials should be done globally.

In some circumstances, the European Medicines Agency recently proposed dropping the need for clinical efficacy trials, and other bodies have looked at the question. A PMDA reviewer discussed the events in a recent presentation. The employee said the re-evaluation should be done globally and “a framework for streamlining biosimilar development should be harmonized.”

The presentation also covered actions PMDA has taken to encourage the development of biosimilars in Japan. PMDA has approved fewer biosimilars than its counterparts in the US and EU, leading the agency to update its guidance to encourage development. The agency has changed the rules regarding the need to include Japanese patients in pharmacokinetic and efficacy studies.

PMDA Presentation

Asia-Pacific Roundup: PMDA outlines initiatives to promote pediatric drug development in Japan

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