Over the past few months, both the United States FDA and Health Canada have updated their guidance on expanded / early access. Although these changes haven’t radically rewritten the rulebook, they have raised the bar on transparency, structure, and data expectations for expanded access.
For Sponsors planning or running programs in 2026, this is a good moment to sanity check whether your approach is still fit for purpose.
1. FDA’s 2025 Expanded Access Q&A – What Actually Changed?
In October 2025, FDA finalized its revised guidance “Expanded Access to Investigational Drugs for Treatment Use: Questions and Answers”, replacing the 2017 version. The Federal Register notice of availability followed on 17 November 2025.
The key points are outlined below:
- Public expanded-access policies are now non-negotiable
The guidance incorporates statutory requirements from the 21st Century Cures Act and FDA Reauthorization Act of 2017 (FDARA): Sponsors of certain investigational drugs must have a publicly available expanded-access policy, once the program reaches defined regulatory milestones (e.g. key trials starting or expedited designations granted). The policy must be easily accessible for patients and physicians and is typically posted on a company’s website.
- Clearer expectations on when and how to use each pathway
FDA expands its Q&A around single-patient (including emergency), intermediate-size, and treatment protocols and INDs – including practical advice on when each is appropriate, how to submit (e.g., use of Form 3926 for individual patients) and how to ensure expanded access does not interfere with the primary clinical development plan.
- More detail on ethics, consent and IRB involvement
The revised document spells out expectations on informed consent, including differences between emergency and non-emergency use, and reiterates that IRB review is still expected, even for single-patient access, in most circumstances.
What does this mean for Sponsors?
- Providing readily available, up-to-date information around your expanded access intentions is key – the FDA will be looking at how clearly and fairly you explain your policy to patients, investigators, and advocacy groups.
- Internal SOPs, website content, and training decks that still mirror the 2017 guidance are now out of date; regulators, institutions and patients will be working off the 2025 document.
- The guidance reinforces the idea that expanded access is a structured, governed program, not an exception granted on a case-by-case basis.
2. Health Canada’s New “Expanded Access Clinical Trials” Framework
In September 2025, Health Canada released a new guidance package on “expanded access clinical trials” – a regulated pathway that sits alongside, but distinct from, the long-standing Special Access Program (SAP).
Instead of treating group access as a series of SAP requests, Health Canada now sates: if a Sponsors wants to provide access to a group of patients in Canada, you are required to run a formal expanded access clinical trial with a Clinical Trial Application (CTA) under regulation (Part C, Division 5).
Key features:
- Group access = a proper trial
- Individual patients continue to use SAP.
- For a defined cohort (e.g. all eligible patients with a condition), Sponsors are expected to set up an expanded access clinical trial, with a protocol, ethics review and 30-day CTA review, similar to an interventional clinical trial.
- Stronger focus on fairness, transparency and risk management
The guidance contains specific sections on transparency, equity and fairness in how patients are identified and prioritized. It also outlines how to justify the rationale for offering access in Canada (e.g. disease severity, lack of treatment alternatives, development stage of the drug), ensuring the trial does not undermine recruitment to confirmatory studies.
- Decentralization and evidence generation explicitly encouraged
Health Canada highlights decentralized and hybrid approaches, local Healthcare Provider involvement and data reliability in real-world conditions. Expanded access trials are expected not only to provide treatment, but potentially to complement and contextualize data from confirmatory studies.
What does this mean for Sponsors?
- For multinational EAPs, you can’t simply “copy-paste” a US-style protocol into Canada. Group access there is likely to look and feel like an additional clinical trial arm, with its own CTA and oversight.
- On the positive side, Health Canada is implicitly endorsing expanded access as a legitimate evidence-generating tool, provided design, equity and data quality are properly addressed.
3. Practical Implications for your 2026 Expanded Access Strategy
Across both regulators, a few clear themes emerge. As you plan for 2026, it’s worth pressure-testing your approach against the following:
1. Refresh your governance and public-facing policy
- Align your global expanded-access policy and website wording with the 2025 FDA guidance and Health Canada’s language on fairness and transparency.
- Ensure your policy answers the questions patients and investigators will have: who is eligible, how enrollment decisions are made, and what happens if demand exceeds supply.
2. Design programs as programs – not ad-hoc exceptions
- Move away from responding to isolated requests and towards structured frameworks: single-patient, cohort and full program models, with clear intake criteria and documentation.
- For Canada, decide early whether you anticipate enough demand to justify an expanded access clinical trial, and build that into your global planning.
3. Embrace “access + evidence”, not access or evidence
- Both FDA and Health Canada acknowledge that expanded access can generate useful supportive data if designed correctly.
- Consider how you can harmonize data elements across your pivotal studies, EAPs and post-authorization work to create a coherent evidence package – without overburdening sites or patients.
4. Plan country-specific variants from the start
- In 2026, a typical global strategy may need to reconcile:
- US Expanded Access INDs (single-patient, intermediate-size, treatment IND)
- Canadian expanded access clinical trials vs SAP
- EU national compassionate use / MAPs under an EMA/HTA lens
- Building these pathways in from the protocol concept stage, rather than bolting them on later, will save time and avoid awkward re-work with regulators and ethics committees.
5. Support investigators and sites with practical tools
- The regulatory bar is higher, but the clinician time has not expanded. Providing templates, checklists, centralized regulatory support and patient-friendly explanations will make the difference between a program that works on paper and one that works in real life.
How WEP Clinical can help
If you’re revisiting your expanded access plans for 2026, this is an ideal time to connect with our expert expanded access team. We can help you to:
- Benchmark your current policy and program designs against the new FDA and Health Canada expectations.
- Stress-test whether your global plan is operationally realistic across the US, Canada, Europe, Asia, and other priority markets.
- Explore opportunities to collect Real-World Data in your EAPs to support clinical trial data and evidence packages.
- Identify where decentralized or hybrid models could widen access without overcomplicating oversight.
If you would like to connect to discuss how we can support your team, please reach out to us – [email protected]
