Mirja Krause-Onwukwe, PhD, MSc
ISCT ANZ LRA Committee Member
Australia
Gabrielle O'Sullivan, PhD
ISCT ANZ LRA Committee Co-Chair
Australia
Australia and New Zealand Regulatory Updates
Medsafe
- Medsafe Guidance on Advanced Therapies – As an outcome of the consultation by the Ministry of Health to the Guideline on the Regulation of Therapeutic Products in New Zealand, ongoing updates to secondary guidance documents are being published in 2026: Considerations for first-in-human (FIH) and early phase clinical trials is active as of July 2026.
TGA
- TGA Consultation: Conformity Assessment Procedures for Medical Devices
The TGA is consulting on proposed amendments to conformity assessment procedures as part of its Action Plan for Medical Devices, aiming to strengthen oversight while remaining patient-focused and transparent. The changes seek to align Australia’s framework with international standards, including the European MDR and IVDR, IMDRF guidance, and the MDSAP.
- TGA Consultation: PIC/S GMP Annex 6 and Annex 15 Concept Papers – Public Consultation
The Pharmaceutical Inspection Co‑operation Scheme (PIC/S) has opened consultations on revisions to Annex 6 (Manufacture of Medicinal Gases) and Annex 15 (Qualification and Validation) of the PIC/S GMP Guide. The updates reflect current industry practices, including new technologies, computerised systems, and expanded scope aligned with ICH Q9 quality risk management principles. The TGA adopts these annexes as part of Australian Manufacturing Principles and encourages feedback from manufacturers.
- TGA Work-Sharing Initiative: Access Consortium New Active Substance (NAS) work-sharing initiative | Therapeutic Goods Administration (TGA)
On 3 March 2026, the TGA updated its information on the New Active Substance Work-Sharing Initiative (NASWSI) which is an innovative work sharing procedure for the coordinated assessment of new chemical entity or new biological entity applications, or new indication applications that are submitted to two or more Access agencies. By participating in this initiative, Access agencies are able to co-ordinate regulatory review procedures, and increase each agency’s capacity to ensure consumers have timely access to high quality, safe and effective therapeutic products.
- TGA-Industry Working Group on Good Manufacturing Practice (GMP) (TIWGG) Meeting
The TGA has indicated, through discussions at TIWGG (held on 25th Mar 2026), its intention to adopt the PIC/S standards, with current focus on the timing and implementation approach rather than the decision itself. Furthermore, the TGA has updated its website to improve usability, introducing a simplified, topic-based structure and enhanced navigation features to improve access to regulatory information.
Australian Law Reform Commission (ALRC)
The Australian Law Reform Commission (ALRC) has been conducting a review of Australian human tissue laws, which includes the Human Tissue Act 1983. The ALRC published a Discussion Paper on 21 November 2025 and consulted on it until 23 January 2026. It is finalising the review and is to provide its final report to the Government by 16 August 2026.
The Review of Human Tissue Laws Discussion Paper presents 49 proposals for reform, in areas including:
- Harmonising and modernising the national regulatory framework for human tissue laws to provide better oversight;
- Future proofing human tissue legislation, to account for innovations and advances in medicine and technology; and
- Improving access to human tissue for valid purposes, while protecting the rights and dignity of donors of human tissue.
International Regulatory Updates Relevant to Australia & New Zealand
International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use (ICH)
ICH E22 Draft Guideline: Patient Preference Studies
The ICH E22 draft guideline provides a globally harmonized framework for patient preference studies to inform drug development and regulatory submissions. It aims to harmonise patient preference studies using a globally agreed framework. A virtual stakeholder webinar was held in February 2026 — click here to view the recording.
FDA
- FDA Initiatives Supporting Cell and Gene Therapies (2026)
In early 2026, the FDA introduced several measures to accelerate development and regulatory review of cell and gene therapies (CGTs).
Key initiatives include:
- Increased Manufacturing Flexibility (11 January 2026): Loosened CMC requirements during development, exemptions from certain biologics specifications, and leniency for minor manufacturing changes in late-stage development.
- Targeted Pathway for Individualized Therapies (23 February 2026): New framework for gene editing and RNA-based therapies for ultra-rare diseases where large-scale randomized trials are not feasible.
- Ultra-Fast Review Timelines: Under the Commissioner’s National Priority Voucher (CNPV) pilot program, qualifying innovative therapies—including many CGTs—can have review times shortened from ~10 months to 1–2 months.
- Strategic National Priority: CGTs addressing large unmet medical needs or supporting domestic manufacturing capacity are prioritized for these regulatory pathways, facilitating more efficient clinical development and access.
- "Flexible Requirements for Cell and Gene Therapies to Advance Innovation" (US FDA).
- "FDA Increases Flexibility on Requirements for Cell and Gene Therapies to Advance Innovation" (US FDA).
- FDA Draft Guidance: Individualized Therapies for Ultra-Rare Diseases
The FDA has issued draft guidance in February introducing the Plausible Mechanism Framework, which provides a pathway for approval of genome editing, RNA-based, and other individualized therapies when randomized trials are not feasible. The guidance emphasizes targeting disease-causing abnormalities, using natural history data, and demonstrating clinical or biomarker benefit.
- FDA Commissioner's National Priority Voucher (CNPV) Pilot Program
Launched in June 2025, the CNPV pilot aims to accelerate review of drugs and biologics addressing public health crises, breakthrough therapies, unmet medical needs, domestic manufacturing, and affordability. In February 2026, the FDA approved the lung cancer drug Hernexeos (zongertinib) under the CNPV pilot program. The program aims to expedite approval of therapies addressing critical national health priorities, with the voucher offering benefits such as rolling review and shortened review timelines.
- FDA Updates CAR-T Trial Design and REMS Requirements
The FDA has signaled a major shift in CAR-T development, now generally requiring randomized trials with standard-of-care control groups and evidence of superiority over existing therapies, while equivalence or noninferiority must be strongly justified. At the same time, the agency has eased REMS requirements for approved autologous CAR-Ts, reducing logistical burdens for treatment centers and patients by shortening monitoring and driving restrictions, removing some onsite medication requirements, and updating labeling. These changes aim to maintain high evidentiary standards while streamlining access and supporting safe administration of CAR-T therapies.
- FDA Expands Use of Real-World Evidence (RWE)
In December 2025, the FDA removed a major barrier to using real-world evidence in drug and device applications by allowing submissions without identifiable individual patient data, provided the evidence is robust. This change enables the agency to consider large de-identified datasets—such as national registries, hospital systems, and claims databases—to support regulatory decisions, accelerating development and approval of therapies, including biologics and cell and gene therapies.
- FDA Declines Approval for EBV-Specific T-Cell Therapy
The FDA has declined to approve Atara Biotherapeutics’ EBV-specific T-cell therapy (Ebvallo (tabelecleucel)) for post-transplant lymphoproliferative disease (PTLD). Ebvallo (tabelecleucel) is an off-the-shelf allogeneic Epstein-Barr virus specific cytotoxic T-cell therapy. The product was approved in Europe as a treatment for PTLD. The reason provided by the FDA in its letter was that the single-arm ALLELE trial could no longer adequately provide evidence of efficacy and that its design, conduct and analysis could confound data interpretability. The decision impacts patients with PTLD, and the field more generally following AlloVir’s decision to discontinue its three Phase 3 posoleucel studies following its conclusion that the studies were unlikely to meet their primary endpoints (no safety concerns identified).
FACT
- FACT Draft Fourth Edition Common Standards
FACT has released the draft Fourth Edition of its Common Standards for Cellular Therapies for public comment (open until May 2026). The updated standards aim to reflect the evolving field and provide foundational quality principles applicable across cellular therapy products, development stages, and clinical applications.
UK
UK Government Plans to Phase Out Animal Experiments
The plan includes an end to regulatory testing on animals to assess the potential for new treatments to cause skin and eye irritation and skin sensitisation by the end of 2026.
By 2027 researchers are expected to end tests of the strength of botox on mice and to use only DNA-based lab methods for adventitious agent testing of human medicines – the process for detecting viruses or bacteria that might accidentally contaminate medicines.
By 2030 it will also reduce pharmacokinetic studies – which track how a drug moves through the body over time – on dogs and non-human primates.
View "Animal testing to be phased out faster as UK unveils roadmap for alternative methods" (Gov.UK).
In the UK there is a major campaign for a proposed legal reform, known as Owain’s law, to standardise tissue freezing so that brain cancer patients can have better and more equitable access to modern treatments and advanced research. It was named after Owain James, who died in June 2024 from glioblastoma. The Campaign for Owain’s Law was launched in 2025 by Owain’s wife Ellie James to address the issue of brain tissue being usually stored in the wrong way, using outdated methods that damage DNA, meaning it cannot be used for modern diagnostics, genome testing, or cutting-edge treatments. As a result, whether a patient can access the most advanced therapies often depends not on their clinical need, but on where they live and where they happen to be treated. The proposal hasn’t been passed into law yet, but is the subject of much public debate and support by brain tumour charities and the UK medical community.
Japan
“Japan’s Pharmaceutical Affairs Council, an advisory body within the country’s Ministry of Health, Labor and Welfare (MHLW), has recommended the conditional and time-limited marketing approval of two induced pluripotent stem cell (iPSC)-based therapies. If formally approved by MHLW, the decision would mark the first regulatory approval of iPSC-derived therapeutic products anywhere in the world. The first therapy, allogeneic iPSC-derived dopaminergic neural progenitor cells (Amchepry), developed by Sumitomo Pharma Co. and Racthera Inc., is designed for the treatment of Parkinson’s disease. The product consists of differentiated neural progenitor cells derived from donor iPSCs intended to restore dopaminergic signaling. The second therapy, ReHeart, developed by Cuorips Corporation, is an allogeneic iPSC-derived cardiomyocyte patch designed to treat severe heart failure due to ischemic cardiomyopathy. The therapy involves transplantation of cardiomyocyte sheets derived from iPSCs to support myocardial repair.”
Japanese Regulatory Panel Recommends Advancing World's First iPSC-Based Therapies
USA
Broad Institute Proposal to Make Cheaper and More Accessible Genetic Surgery
The Broad Institute has proposed a Centre for Genetic Surgery, a non-profit created by David Liu and colleagues at the Broad Institute. This comes in the wake of advancements in personalized gene editing therapies.