One-Trial Requirement:
As of early 2026, the FDA has issued a new position allowing for the approval of some novel products based on one pivotal trial instead of two, provided the surrounding evidence ecosystem is persuasive.
Ultra-Rare Disease Framework:
A new framework has been launched to accelerate therapies for ultra-rare conditions, utilizing regulatory flexibility for individualized treatments.
PreCheck Pilot Program:
Starting in 2026, the FDA is implementing a program to conduct early inspections and reviews of new pharmaceutical manufacturing facilities to speed up domestic production.As of early 2026, the FDA has issued a new position allowing for the approval of some novel products based on one pivotal trial instead of two, provided the surrounding evidence ecosystem is persuasive.
In both Europe and the United States, clinical trials are essential for bringing new
treatments from the laboratory to the public. Each region follows strict scientific and
ethical standards to ensure the safety and effectiveness of new medicines. For
participants, that means protection and close monitoring at every phase — from early
testing to post-approval follow-up.
While both systems share the same goal of advancing health, Europe and the US differ in how trials are approved, regulated, and communicated. This can influence aspects from how easily you find a study to how your data is handled. In short, both regions are committed to making safer, more effective medicines available. They simply take different regulatory routes to get there. Expert guidance on clinical trial design specifically tailored for academic discoveries transitioning to first-in-human studies, with a focus on efficient protocols that meet FDA requirements
United States:
In the US, clinical research is regulated by the Food and Drug Administration (FDA) and monitored by Institutional Review Boards (IRBs), which safeguard participant rights and welfare. Before a study begins, sponsors must submit an Investigational New Drug (IND) application to the FDA, demonstrating that the trial is scientifically sound and ethically designed. This regulatory structure not only streamlines study initiation but also encourages early-phase, innovative research. Additionally, by ensuring studies reflect local patient needs, IRBs help volunteers feel more supported and heard. How clinical trials are designed, regulated, and conducted can differ between Europe and the US. From data protection to study procedures and accessibility, understanding these key differences offers valuable insights into what volunteering for a trial looks like — and how your experience may vary depending on where in the world you take part.
Regulatory Frameworks Europe
The Accelerating Clinical Trials in the EU (ACT EU) initiative has published a draft guidance document outlining how clinical trials should be conducted during public health emergencies. The guidance, now open for stakeholder consultation, is intended for sponsors and all parties involved in the design and conduct of clinical trials in the EU.
This is the first guidance on PHEs to reflect the EU’s current legislative framework as well as the guidelines from the International Council for Harmonization of Technical Requirements for Registration of Pharmaceuticals for Human Use (ICH) developed following the COVID-19 pandemic. It recommends a harmonized approach to ensure that clinical trials can be initiated, adapted and continued efficiently and safely when public health emergencies arise.
The guidance proposes regulatory mechanisms aiming to accelerate the authorization of new clinical trials and the approval of modifications to ongoing trials during a public health emergencies. Sponsors are encouraged to seek scientific advice from EMA’s Emergency Task Force (ETF) to ensure trials are well designed, efficient and capable of delivering meaningful data for regulatory assessment.
In Europe, the European Medicines Agency (EMA) and national authorities in each EU member state oversee clinical trials. The EU Clinical Trials Regulation (CTR 536/2014) streamlined the approval process. Now, the Clinical Trials Information System (CTIS) manages them centrally.
For volunteers, this centralized system makes participation easier to track and understand. The CTIS increases transparency, giving the public access to information on all authorized studies, including results once the trial is complete. It also ensures that informed consent, data protection, and ethical oversight are at the heart of every study, strengthening participant trust and safety.
The draft guidance for situations in which trial participants can be transferred across investigational sites. Adapted procedures or alternative approaches may be required due to the specific challenges posed by public health emergencies. The rights, safety, and well-being of trial participants and the generation of scientifically robust evidence remain central to regulatory decisions on clinical trials during such emergencies.
Australia stands out as a premier destination for biotechnology companies seeking to conduct clinical trials, thanks to compelling strategic advantages. The country’s robust healthcare infrastructure, highly skilled workforce, and stringent clinical trial environment provide a solid foundation. The regulatory framework, governed by the Therapeutic Goods Administration (TGA), is renowned for its efficiency—particularly the Clinical Trial Notification (CTN) scheme, which delivers expedited study approvals, often within weeks. This streamlined process empowers companies to launch trials with unmatched speed, accelerating time-to-market for breakthrough therapies. In addition, Australia’s diverse and stable population enables access to a broad spectrum of potential participants, maximizing the relevance and applicability of trial outcomes. Expert guidance on clinical trial design specifically tailored for academic discoveries transitioning to first-in-human studies, with a focus on efficient protocols that meet FDA requirements
Australia is increasingly celebrated as the destination of choice for early-stage oncology, immunology, and novel medical device trials. Sponsors seeking to unlock competitive market advantage are turning to Australian first-in-human (FIH) trials, drawn by these compelling features:
Rapid Startup: Australia empowers sponsors to progress from protocol development to first patient enrollment within just two to three months, while private sites can initiate even faster—often within six to eight weeks—giving partners a head start in the global race for innovation.
Regulatory Efficiency: Without the need for a United States Investigational New Drug (IND) submission, sponsors benefit from Australia’s streamlined pathways, accelerating the commencement of early-phase research.
Cost-Effectiveness: The Australian Government’s attractive tax incentive—up to 43.5% on eligible R&D expenditures—delivers substantial value, supporting innovators to maximize investment returns.
Focus on Safety: State-of-the-art Phase I units in key cities ensure a highly controlled, technology-driven environment for safely monitoring healthy volunteers or targeted patient populations, upholding the highest standards in patient care and safety.
China
China is aggressively accelerating clinical trials for Advanced Therapy Medicinal Products, including cell and gene therapies, through 2026, aiming to synchronize with global markets. Revisions to the Drug Administration Law, effective May 2026, slash approval times for urgently needed drugs to 30 days or less, expand conditional approvals, and boost the acceptance of overseas clinical data
Key 2026 Initiatives for Accelerating ATMPs
· Faster Regulatory Approvals: The National Medical Products Administration (NMPA) aims to reduce clinical trial approval times for innovative drugs to approximately 30 working days, with further plans for a 20-day timeline in specific cases.
· Conditional Approval Expansion: Regulations now allow for conditional sales approval based on interim results, enabling earlier market access to ATMPs before Phase 3 trials are fully completed.
· Global Data Acceptance: China is broadening the recognition of overseas clinical trial data to reduce the need for redundant studies.
· Regulatory Updates & Data Protection: New implementing regulations (effective May 15, 2026) strengthen intellectual property and data protection, incentivizing innovation in the biotech sector.
· Focus on Breakthrough Therapies: The revised policies strongly support breakthrough, high-value therapies, including CAR-T and antibody-drug conjugates (ADCs).
Impact on Drug Development in China
The reforms aim to narrow the gap between clinical studies and market authorization, with some studies showing a 50-70% increase in speed from discovery to investigational new drug (IND) application. These changes are particularly relevant for CAR-T therapies, as China leads the world in the number of active projects. Strategic Considerations for 2026
· Marketing Authorization Holder (MAH) System: The MAH system requires robust, full-lifecycle oversight of drug development and manufacturing.
· Supply Chain Support: The use of Free Trade Zones (FTZs) is encouraged to streamline logistics for imported clinical trial materials.
· Enhanced Surveillance: Strict post-marketing surveillance and data traceability are required to maintain quality.
The question is: should I make my drug in China since the production time is shorter than in the US the important question is ,once made, will the FDA approve it?
Yes, the FDA approves drugs manufactured in China, provided they meet strict U.S. safety, efficacy, and quality standards. The FDA now often requires multi-regional clinical data, not just Chinese-only trials, for drug approval. Chinese-made products must comply with the same U.S. laws as domestic ones.
Approval Trends: While some, such as Beigene’s zanubrutinib, have been approved, others (e.g., Hutchmed’s surufatinib, Eli Lilly’s sintilimab) were rejected, often due to a lack of diverse, multi-regional clinical data.
The FDA is concerned about data integrity and the applicability of China-only trials to the U.S.
Drugs from China, especially oncology drugs, face increased FDA scrutiny over trial diversity. China supplies many pharmaceutical ingredients, accounting for a significant share of U.S. drug master files. The FDA continues to review and accept applications from China, but they must meet rigorous multi-site study criteria rather than rely on single-country trials.
NOTE: Oncology Clinical Trials in ASIA
The 2026 ASCO Breakthrough meeting will take place from June 25–27, 2026, at the Raffles City Convention Centre in Singapore, featuring in-person and online attendance. Co-hosted by the Singapore Society of Oncology, this event focuses on oncology advancements in Asia, featuring keynotes on precision public health and multiomics diagnostics.
American Society of Clinical Oncology – ASCO +3
Key Details for 2026 ASCO Breakthrough:
- Dates: June 25–27, 2026.
- Location: Raffles City Convention Centre, Singapore.
- Keynote Speakers: Dr. Nora Pashayan (“Optimizing Cancer Screening Via Risk Stratification”) and Dr. Yuk Ming Dennis Lo (“Multiomics and Non-Invasive Diagnostics for Cancer Detection and Treatment Personalization”).
- Abstract Deadline: Feb 24, 2026.
- Early Registration Deadline: May 20, 2026.
- Focus: Bridging clinical care with technological innovation in Asia, featuring specialized sessions and networking.
- Program Committee: Chaired by Melvin Chua, MBBS, PhD, FASCO.
American Society of Clinical Oncology – ASCO +7