Asia-Pacific Roundup: Saudi FDA streamlines rare disease drug development
The Saudi Food and Drug Authority (SFDA) has launched the New Accelerator Program for Drugs in Rare Diseases (NADR) to streamline the development, evaluation, and authorization of drugs for rare diseases.
NADR is open to medicines targeting diseases for which there are no satisfactory treatment options in Saudi Arabia, as well as to products that offer significant clinical benefit over currently available therapies. To qualify, products must be under active development at any stage or already authorized by any regulatory authority but not yet registered in Saudi Arabia.
Eligible drugs benefit from regulatory incentives and support measures, including dedicated scientific and regulatory guidance. SFDA will offer “flexible and innovative approaches to the evaluation of clinical evidence that take into account the challenges associated with small patient populations.”
In guidance on the NADR, the agency named adaptive trial designs among the types of evidence it will accept for rare disease filings, including Bayesian adaptive, N-of-1, basket, umbrella, and platform studies, “that maximize information yield from small patient samples.” SFDA will also accept natural history data, modeling and simulation outputs, surrogate endpoints, and knowledge of platform technologies.
New approach methodologies (NAM) are also acceptable. SFDA will accept data derived from artificial intelligence, machine learning, and in silico computational models, as well as from organ-on-a-chip in vitro models. The agency plans to publish dedicated guidance on NAMs in 2026 or 2027.
The SFDA will apply a proportionate evidence approach to periodic safety update reports and registries, “tailoring requirements to the specific patient population size to ensure technical feasibility and encourage industry compliance.” The flexibility extends to exemptions from chemistry, manufacturing, and controls data requirements, including provisions to accelerate production site registration.
Companies interested in participating in the program should organize a presubmission meeting with SFDA. Applicants without an orphan drug designation (ODD) can request that status in parallel to applying for the NADR. The agency will review NADR and ODD requests within 20 working days of receiving the complete submission. Companies will then have six months to apply for marketing authorization.
TGA seeks feedback on publishing information about medical devices in Australia
Australia’s Therapeutic Goods Administration (TGA) is consulting on plans to improve the sharing of information about the safety, quality, and performance of medical devices.
TGA has requested feedback on proposals to provide the public with more information after postmarket reviews or investigations of medical devices, particularly when findings relate to the safety, quality, performance, or use of a product. The plans align with the 2023 “An Action Plan for Medical Devices,” in which TGA outlined ways to improve transparency and boost public confidence in medtech regulations.
Under the new proposal, TGA will change Australian legislation to allow the publication of more explicit information about post-market reviews and their outcomes. Australian law currently restricts the TGA from accessing high-level information when it takes actions such as conducting a recall or issuing a hazard alert. If the TGA finds a device compliant or a sponsor takes voluntary action, the agency is limited in what it can say.
The proposals will allow TGA to identify the specific devices that were the subject of a review, explain how they met the requirements for quality, safety, and performance, and discuss potential limitations when the product is used in particular circumstances. TGA will also be able to comment on the device's safe use or performance.
Officials want to know if people generally agree to providing the public with more information about the devices included in post-market reviews and investigations. The agency is also seeking feedback on what types of information are appropriate to share, such as the reason for the review and the resulting regulatory actions.
TGA started the consultation the day after responding to media reports about the safety of defibrillator devices used in some ambulance services and healthcare facilities. Because the media reports may cause concern, TGA released a statement to reassure the public that the devices are not being recalled. Rather, the agency suspended the supply of new devices while the manufacturer addresses concerns.
The agency added that it “has already been undertaking work to further increase transparency around its regulatory decisions” and would start a consultation in the coming days. The consultation closes on 2 October.
Singapore’s HSA finalizes medtech cybersecurity best practices after positive feedback
Singapore’s Health Sciences Authority (HSA) has finalized its best practices guide for medical device cybersecurity.
HSA held a public consultation on a draft version of the guide last year. The 16 stakeholders that responded provided positive feedback, HSA said, and rated their understanding of the document at 4.5 out of 5. Almost 40% of the 123 responses submitted by the 16 stakeholders were editorial in nature. HSA classified most of the other responses as technical.
The agency has incorporated the feedback into its final guide to improve the document's clarity. HSA has rewritten large sections of the document, including completely changing the text on general principles.
“Cybersecurity should not be treated as a separate set of discrete tasks, but as an interconnected series of actions that mutually reinforce one another,” HSA said in the final guide. “This approach moves away from reactive problem-solving and instead focuses on embedding security into every stage of the device's life cycle.”
The section on risk management has undergone extensive changes. In the final guide, HSA emphasizes that risks can occur at any stage in a medical device’s lifecycle. Risks identified at one stage can be fixed at a different stage, for example, by implementing stronger secure-by-design controls in the next product iteration to mitigate a vulnerability found in postmarket use of an older device.
HSA has revised its draft advice on analyzing, evaluating, and controlling risks. The agency recommends evaluating the residual risk after implementing cybersecurity controls and actively monitoring threats to support continuous improvement.
India’s CDSCO clarifies rules after requests to import and market unapproved medicines
The Central Drugs Standard Control Organization (CDSCO) has clarified its rules after receiving applications for permission to import and market unapproved new drugs in India.
Companies requested the permissions for new drugs that are not approved anywhere in the world based on global Phase 3 trials that “are either ongoing or have been completed with the participation of Indian subjects,” CDSCO said. The requests prompted discussions within CDSCO’s technical committee.
“After detailed deliberations, the committee opined that such investigational new drug (IND) applications, which are under review by foreign regulatory authorities, [are] required to be processed by the IND Division, as these applications require a comprehensive evaluation of non-clinical and clinical data,” CDSCO said.
The conclusion led CDSCO to ask applicants to file their import and marketing requests with its IND unit. The division will examine and process the applications in accordance with the provisions of India’s New Drugs and Clinical Trials Rules, 2019.
