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Preparing for Clinical Trials in Rare Disease: Lessons in Global Regulatory Collaboration

Preparing for Clinical Trials in Rare Disease: Lessons in Global Regulatory Collaboration

Preparing for Clinical Trials in Rare Disease: Lessons in Global Regulatory Collaboration

Insights from the Network

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"For the regenerative medicine community, especially those working in rare diseases, these insights matter deeply. They remind us that strategic planning is as critical as scientific discovery. The success of a therapy doesn’t just depend on the science - it depends on navigating regulatory systems that ensure safety, consistency, and patient access."

– Patrick Bedford

Patrick Bedford, VP, Regulatory Affairs at Morphocell Technologies and Moderator, Canadian Rare Disease Symposium 2025

Watch the recordings

Both sessions from SCN’s Rare Disease webinar series are now available online.

February 2026

Reflections from Patrick Bedford, VP, Regulatory Affairs at Morphocell Technologies and Moderator, Canadian Rare Disease Symposium 2025

As we approach Rare Disease Day, it’s the perfect time to reflect on how far research has come in addressing rare diseases. Advanced cell and gene therapies offer promising treatments, but navigating the global regulatory landscape is critical to bringing these therapies to patients. I have a renewed understanding of how important it is to navigate global regulatory frameworks efficiently because I was very recently hospitalized with the effects of my own rare genetic disease, which has no treatment beyond careful management of physical activities. Thus, I have additional motivation to mark Rare Disease Day, highlight some of the comments from our Canadian Rare Disease Symposium regulatory speakers, and advocate for researchers to better understand how regulatory milestones line the path to successful clinical translation.

The Stem Cell Network (SCN) and National Research Council Canada (NRC) are committed to supporting rare disease research. As part of these efforts, I had the privilege of moderating the latest webinar in their rare disease series, Preparing for Clinical Trials in Rare Disease: Global Regulatory Insights, now available to watch online. This session builds on the earlier February 2024 webinar, which is also available for viewing. Together, these sessions provide practical guidance for researchers developing cell and gene therapies and highlight the importance of global regulatory engagement.

Researchers developing these therapies need more than just a basic understanding of the regulatory ecosystem. A treatment cannot reach patients without careful consideration from regulatory agencies, and the SCN and NRC webinar provided insights into how researchers should think about (and begin to navigate) this landscape at the earliest stages of product development. Hosting sessions like these highlights SCN and NRC’s ongoing commitment to supporting the research community in advancing treatments and cures.

In the October 2025 webinar, I moderated a conversation with regulatory experts from Health Canada, Japan’s PMDA, and the European Medicines Agency (EMA), as well as a fellow regulatory consultant who supports companies developing biotech drugs for rare diseases. Over the course of the session, it was apparent how aligned regulators truly are: This means that Canadian translational activities will fit seamlessly into global development plans (including European and Japanese regulators) and global translational activities will fit seamlessly into Canadian regulatory submissions.

Setting the stage: Why global perspective matters

In Canada’s growing cell and gene therapy ecosystem, discussions often centre on the U.S. Food and Drug Administration (presumably because real or perceived beliefs that investors only care about the world’s largest market). But as I reminded participants at the outset, there are other regulatory jurisdictions worth exploring. Each jurisdiction brings unique pathways and programs that can accelerate access for patients with rare diseases, and many jurisdictions have knowledgeable and experienced regulators (some of whom contribute to global regulatory standards such as International Council for Harmonization to which U.S. FDA adheres). It is entirely possible to reach target commercial markets FASTER by starting in jurisdictions like Canada.

As I put it to the audience: “We’ll have succeeded today if you leave thinking about a more nuanced global regulatory strategy – one that goes beyond target market size to include jurisdictions that can get your products to patients sooner.”  The panel that followed underscored this message powerfully.

Health Canada: Flexibility with scientific rigour

Dr. Omar Tounekti from Health Canada opened with a clear overview of how cell and gene therapy trials are reviewed in Canada (much like any other novel biologic drug), then focused on products targeting rare disease populations. While Canada lacks a formal orphan drug designation, there is significant flexibility within existing frameworks to accommodate small patient populations.

At Health Canada, Clinical Trial Applications (CTAs) are reviewed within 30 days, with room to justify limited batch or stability data when appropriate. Yet, as Dr. Tounekti reminded us, “Safety and quality are never compromised.” He walked us through the importance of detailed manufacturing documentation – everything from control strategies to certificates of origin – and highlighted that missing information on raw materials remains one of the most common pitfalls in cell and gene therapy submissions. The “regulatory flexibility” reflects what excited investors saw earlier this year when the US FDA issued its News Release “FDA Increases Flexibility on Requirements for Cell & Gene Therapies to Advance Innovation” (January 11, 2026) … it also probably sounds familiar to anyone who has worked to develop cell and gene therapies over the past decade or two. 

Dr. Tounekti also said, “Talk to us [Health Canada]. Come prepared with product-specific questions – that’s how we can add value.” For me, this perfectly captures Health Canada’s historical position and ongoing role: open, accessible, and science-driven, with a strong commitment to supporting innovation while maintaining the highest safety standards.

While “come early, come often” sounds highly desirable coming from a regulator, I must caution early-stage developers not to approach Health Canada without sufficient preparation:  One should always “do their homework” prior to engaging regulators. This involves (a) preparing to describe key development decisions based on existing regulatory expectations (principles, guidance, and precedents), (b) developing sophisticated regulatory narratives (rationales in understandable, familiar, and coherent formats), and (c) asking meaningful, actionable, and informed questions that de-risk key decisions. Remember, regulators are not on your payroll! Their insights and advice are integral but do not consider implications beyond product safety, quality, and efficacy (e.g., they intentionally disregard your reality from a capabilities, cost, or timeline perspective, which fall outside their mandate focused on safety, quality and efficacy), and as a patient awaiting gene therapy I would argue that we should not ask them to provide remedial regulatory training on these topics (otherwise wait times grow for everyone). 

Japan’s PMDA: Partnership and patient access

Next, Dr. Yoshiaki Maruyama from Japan’s PMDA shared a detailed look at Japan’s structured and proactive regulatory landscape. Since 2014, Japan has implemented a Sakigake designation that offers a unique conditional and time-limited approval pathway, a model that enables earlier patient access while maintaining rigorous post-market surveillance. Notably, 80% of Japan’s approved cell and gene therapy products target orphan diseases.

Dr. Maruyama emphasized the PMDA’s philosophy of “thinking together against challenges,” highlighting its consultation program that provides early feedback on research and development strategies, often with government cost-sharing to reduce barriers. I was particularly struck by Japan’s focus on predictability: standardized review periods, transparent biosafety protocols, and clear expectations for developers. It’s a system that was built and implemented as a world first to encourage innovation without compromising regulatory credibility for ensuring safety, quality, and efficacy.

The European perspective: Harmonization and transparency

We also heard from Dr. Ilona Reischl, Chair of the EMA’s Committee for Advanced Therapies. In her candid and insightful style (which I always find refreshing and highly engaging!), she demystified the European Union’s highly coordinated but intricate system.

Dr. Reischl reminded us that clinical trials in Europe must pass through a joint review process across member states – one that integrates both regulatory and ethics assessments (while U.S. and Canadian regulators delegates ethics to IRBs/REBs, which must be satisfied separately before the trial commences). The result, she said, is a “single decision per member state,” underpinned by transparency and patient protection that justifies EU member state confidence and produces data that can be reviewed by the EMA at the commercial stage of development.

What resonated with me most was her caution that “getting your trial approved doesn’t mean you have the right data for marketing authorization.” This might be the most important message for researchers and investors alike, and it is globally applicable.  Europe’s earlier requirement to manufacture at licensed GMP facilities (even for first-in-human studies) may seem burdensome at first, but as Dr. Reischl explained, it helps to avoid technology transfers from first-in-human study facilities at critical timepoints in development and associated comparability issues that can derail products later. Her closing thought resonated with me: “The sooner you meet the requirements, the better – because you’re dealing with complexities that can’t be fixed after the fact.” I think cell and gene therapy companies developing products for rare diseases are well served by “front-loading” their efforts to the extent possible.

From insight to action: Building a global strategy

Finally, Dr. Sarmitha (Sam) Sathiamoorthy, President of Aspire Bio, helped translate these regulatory principles into practical strategy. Speaking from the consultant’s lens, she urged developers to “build a global regulatory strategy early- and invest in pre-submission meetings.” Her advice was pragmatic and grounded in experience, and can be summarized as a practical checklist:

  • Early engagement with regulators are an investment that prevents costly setbacks – you should not treat them as optional.
  • Non-clinical, manufacturing, and clinical plans must be aligned from the outset – especially since a single production lot may serve multiple trial phases.
  • You should choose jurisdictions based on science, feasibility, and patient reach, not just investor expectations.

Dr. Sathiamoorthy’s closing comment captured the spirit of the discussion: “Doing the work up front isn’t a waste of money – it’s how you de-risk your entire program.”

What I took away

As moderator, what struck me most was the shared ethos across all our speakers, which did not exist 10+ years ago and has evolved over time: regulators are not gatekeepers – they are partners. Whether in Canada, Japan, or Europe, each jurisdiction is committed to fostering innovation through flexibility, harmonization, and early collaboration. They all WANT us to succeed in treating rare disease populations. I couldn’t agree more.

For the regenerative medicine community, especially those working in rare diseases, these insights matter deeply. They remind us that strategic planning is as critical as scientific discovery. The success of a therapy doesn’t just depend on the science – it depends on navigating regulatory systems that ensure safety, consistency, and patient access.

Looking ahead

This discussion reaffirmed my belief that Canada has a vital role to play as both a testing ground and a global collaborator in cell and gene therapy development. Health Canada’s openness, combined with SCN’s network of researchers, clinicians, and trainees, creates fertile ground for advancing these therapies responsibly and efficiently.

SCN will continue to convene these conversations – bringing together international perspectives that help our community prepare for translation and clinical readiness. The path to rare disease treatment is complex, but by working together and learning from global partners, we can make it a little clearer – and bring hope to patients who need it most. I know that as a rare disease patient waiting for gene therapies to target my disease, I am grateful to SCN and NRC for the role they play. I am also grateful to regulators because I know when it’s my turn and someone proposes to cure me, I need to trust that they developed a safe, high quality, efficacious product whose benefits outweigh their risks.