Pediatric drug access receives policy boost

China plans to improve access to pediatric drugs, with a focus on boosting research and development and strengthening supply chain stability, according to a guideline recently released by the National Health Commission and seven government departments.

The commission said that while demand for medicines targeting common childhood diseases can largely be met at present, there remains a shortage in the overall number of pediatric drugs, including child-appropriate dosage forms and strengths. Many drug labels also provide insufficient or substandard information on pediatric use.

"Pharmaceutical companies remain inadequately incentivized to develop and produce pediatric drugs, making the supply of certain low-volume but clinically essential children's medicines persistently unreliable," the commission said, adding that many hospitals stock only a limited range of child-specific drugs.

To mitigate these problems, the guideline calls for refining supporting policies for the priority pediatric drug catalog and granting listed products accelerated review and approval pathways. Such drugs will be given preferential inclusion in the national essential medicines list, major state-level R&D programs and coverage under the national drug reimbursement list.

Drug regulators will also enhance early and continuous communication with developers and help improve R&D efficiency for innovative pediatric drugs in the pipeline through advanced interactions and rolling data submissions.

Efforts will also be made to support the establishment of pediatric research wards at major medical centers and hospitals, and to explore the construction of a national pediatric clinical trial network and a cross-institutional ethics review mechanism to pool resources, coordinate participant recruitment and standardize trial site management.

"Healthcare institutions will be guided to conduct joint research on drugs suitable for children but lacking pediatric labeling, allowing safety data from Chinese adults to be extrapolated to pediatric populations," the guideline said.

To strengthen supply, the guideline said more pediatric medicines will be added to centralized production bases dedicated to manufacturing drugs with low production volumes or in short supply, while procurement policies will also be improved.

"Monitoring of the production of pediatric drugs and supplies of specialized materials will also be strengthened," it said. "During seasonal infectious disease outbreaks, coordinated monitoring, information sharing and early warning mechanisms will be improved, with reinforced supplies of antivirals and pediatric medicines for relieving fever and pain."

As part of efforts to intensify quality supervision, the guideline calls for supporting pediatric drug manufacturers in upgrading technologies and equipment while strengthening full-chain traceability supervision.

In addition, the guideline highlights the continuation of the temporary import system to ensure access to drugs for special populations with rare diseases and other critical illnesses.

Commercial healthcare insurers will also be encouraged to develop pediatric insurance products that include innovative drugs and rare disease medications in their coverage.

Deng Yong, a health law professor at Beijing University of Chinese Medicine, said the guideline targets chronic challenges in pediatric drug supplies, such as limited drug varieties and dosage forms, weak R&D, unstable supply, inadequate use as well as high costs.

By introducing systemic reforms across the full chain from R&D to insurance coverage, the document is expected to significantly improve the accessibility, safety and affordability of children's medicines, he added.

wangxiaoyu@chinadaily.com.cn