Asia-Pacific Roundup: India updates cell and gene therapy rules to ensure uniform regulation
The Indian government has strengthened oversight of cell and gene therapies by bringing the novel modalities into the Centrally License Approving Authority (CLAA) framework.
Central and state regulators were already jointly responsible for critical drugs and biological products, including medicines based on recombinant DNA and vaccines. Last week, the central government passed changes to the Drugs Rules, 1945, to add cell- or stem cell-derived products, gene therapeutic products, and xenografts to the list of treatments under the joint supervision of central and state regulators.
Joint oversight will ensure “uniformity in regulatory standards across the country,” the government said. Other objectives include increasing regulatory rigor for emerging technologies and reinforcing India's regulatory framework in line with scientific advancements and global best practices. The government framed the changes as part of efforts to promote innovation and accelerate access to new therapies.
The products covered by the expanded CLAA framework include CAR-T cell therapies, gene editing and gene replacement treatments, and animal tissue-derived products such as heart valves. Such advanced therapeutic modalities need enhanced regulatory scrutiny because they are in highly complex, specialized, and rapidly evolving areas of medical science, the government said.
Lawmakers brought the modalities into the CLAA framework by adding the words “cell or stem cell derived products, gene therapeutic products, and xenografts” to multiple sections of the Drugs Rules, 1945, that previously referred to a narrower set of modalities including medicines based on recombinant DNA.
Medsafe shares guidance on New Zealand’s verification pathway for new drug filings
The New Zealand Medicines and Medical Devices Safety Authority (Medsafe) has published guidelines on the verification pathway for new medicine applications.
Last year, lawmakers created a fast-track pathway for medicines approved by two or more reference regulatory agencies. The government deferred implementation until Medsafe could consult with industry and finalize the technical details. Medsafe finalized the details in May, leading the government to set a 3 July implementation date for the pathway.
To support the implementation, Medsafe has published guidance that unpacks and clarifies the law. The agency discussed the eligibility criteria, explaining that an approval granted via a work-sharing assessment procedure such as Project Orbis will meet the definition of a full marketing authorization if all involved authorities are recognized regulatory authorities. A work-sharing review can provide both required authorizations if the applicant has two full sets of assessment reports for each dossier module.
Another section of the guideline addresses when Medsafe may accept differences between the primary marketing authorization and the dossier it receives. Formulation, manufacture, and quality attributes must be the same, but Medsafe may permit differences in secondary packaging sites and container closure systems. Medsafe may also allow minor differences in the drug substance or drug product specifications and the shelf life and storage conditions.
Dossiers submitted to Medsafe can include minor post-approval variations that have been notified to, or approved by, the primary recognized regulatory authority. Applicants must provide details of variations in a table. Medsafe dropped a requirement for applicants to provide “appropriate evidence” of approval of each variation in response to industry feedback.
Philippine FDA seeks feedback on classifying deficiencies seen in clinical trial inspections
The Philippine Food and Drug Administration (FDA) has published draft guidance on the classification of deficiencies identified during inspections of clinical trials.
FDA created the guidance to provide a uniform understanding and implementation of the classification of deficiencies. In doing so, the agency could establish a consistent understanding of the compliance requirements imposed by a series of regulatory and legislative documents and updates. The guidance applies to sponsors, clinical research organizations, and bioequivalence and bioavailability facilities.
The document features definitions of key terms, a high-level overview of the handling of deficiencies found in good clinical practice (GCP) inspections, and more specific guidelines. In the specific guidelines, FDA explains that inspectors will tell sites with critical deficiencies to stop administering the study drug and enrolling patients, suspend clinical activities, and nullify data from the final clinical trial report.
FDA listed types of GCP inspection observations in an annex. The annex covers aspects of clinical trial conduct such as the protocol and informed consent process. In each area, FDA listed types of deviations that its inspectors will categorize as critical and major compliance issues.
The draft is open for comment until 30 July.
India restricts drugmakers’ liability for overcharging, streamlines price fixation process
The Indian government has overhauled its drug pricing legislation, limiting the potential penalties for overcharging and providing a streamlined pathway for some products.
Through the update to the Drugs (Prices Control) Order, 2013, the government has set the overcharged amount based on “the stock handled by each retailer, distributor, or stockist found selling the formulation above the ceiling price (plus local taxes as applicable) pertaining to that batch.” Previously, the penalty was based on the entire batch, even if it was only partly sold at above the agreed price.
To benefit from the reduced liability for overcharging, manufacturers need to show evidence that they made “sufficient effort at disseminating the revision in price to all stakeholders,” including by circulating updated price lists to dealers within two weeks of the change.
Another revision streamlines the process for manufacturers launching new drugs within 12 months of authorities setting a retail price for the same medicine. Rather than applying for their own price approval, companies can share the details of the launch with authorities within one month of bringing the drug to market.
The changes took effect on 30 June.
Australia’s TGA implements mandatory unique identifiers for some medical devices
Australia’s Therapeutic Goods Administration (TGA) has begun enforcing unique device identification (UDI) requirements on higher-risk medical devices.
Starting 1 July, manufacturers of Class IIb and III devices must comply with Australia’s UDI labeling and record submission requirements. Affected manufacturers must supply their devices with UDI-compliant labeling and have UDI records submitted to the Australian UDI Database.
The imposition of the requirements on higher-risk devices is the start of a phased transition to the full implementation of the UDI rules. UDI labeling and record submission requirements will expand to Class IIa devices in July 2027 and to Class Is products in July 2028. TGA will impose direct marking requirements on Class III devices in January 2028 and on the other classes in January 2029.
TGA has identified UDIs as a way to improve transparency and traceability across the medical device lifecycle. The requirements will help strengthen safety and performance while enhancing information sharing and digital connectivity within the health system, the agency said.
Other News:
Japan’s Pharmaceuticals and Medical Devices Agency (PMDA) published advice on “driver mutations” in the development of oncology drugs. PMDA refers to mutations as drivers when “the degree of oncogene addiction is high and the drug efficacy can reasonably be predicted, by evaluating the results together with clinical studies at the early stage of development.” PMDA Guideline
The Philippine FDA released draft guidelines defining major and minor variations of medical and health-related devices throughout the lifecycle. The document specifies requirements for the submission of variation applications and establishes a risk-based approach to post-market variations. FDA is accepting feedback until 9 October. Draft Guidelines
