Quebec offers a revealing case study. The province has strong universities, experienced clinical teams, biotechnology expertise and an established culture of publicly funded healthcare. It has also built considerable capacity in advanced therapies, including clinical research and cell manufacturing. Yet scientific strength does not automatically produce a smooth path from laboratory discovery to hospital treatment. Between those two points lies a complicated landscape of regulation, manufacturing, funding, intellectual property, workforce shortages, reimbursement decisions and hospital capacity.

Prof. Jean-Sébastien Delisle of Réseau ThéCell (the Quebec Network for Cell, Tissue, and Gene Therapy) and colleagues describe this landscape as one in which extraordinary opportunity exists alongside structural obstacles. The perspectives summarized here build on broad consultations led by ThéCell and Héma-Québec (the province’s blood agency) which coordinated discussions involving researchers, clinicians, patient representatives as well as public and private-sector organizations.

The challenge is more than simply inventing better therapies. It is to build a system capable of developing them, testing them, producing them and paying for them without allowing innovation to outrun access. This matters because advanced therapies do not behave like conventional medicines. A pill can often be manufactured in huge batches, stored, shipped and prescribed through familiar channels. A living cell therapy may instead begin with material collected from an individual patient, pass through specialized manufacturing under exacting quality controls, and return to a hospital where trained teams must administer it and manage potentially serious complications.

This complexity makes costs high, but cost is only part of the problem. Many advanced therapies are designed for relatively small groups of patients. A treatment may be medically transformative yet commercially difficult to sustain because the potential market is tiny while manufacturing and regulatory expenses remain substantial. In such cases, conventional pharmaceutical economics can produce a troubling paradox. A therapy may work, patients may need it, and clinicians may want to provide it, yet the business case may still be fragile. If a company withdraws a product or decides not to enter a small market, scientific success can coexist with practical unavailability.

That possibility changes the meaning of healthcare preparedness. A jurisdiction cannot assume that every effective therapy will simply appear on the market and remain there. It may need local or public production capacity, stronger partnerships with industry, and mechanisms for protecting access when commercial incentives are weak. Organizations like Héma-Québec that are responsible for the collection, processing and distribution of biological products, such as blood, cells and tissues, may also have an important role to play in supporting the development, manufacturing, logistics and equitable delivery of certain advanced therapies. This is particularly important for rare diseases, where small patient numbers can make traditional market models especially precarious.

Other countries provide clues about what alternatives might look like. France has developed public and hospital-based routes for producing advanced therapies, allowing academic medicine to play a role that extends beyond research alone. Australia has pursued a hybrid approach that combines hospital and university expertise with industrial partnerships, centralized production and experiments in more decentralized manufacturing. These systems differ, but they share an important principle: advanced therapies may require infrastructure that connects research, production, regulation and healthcare rather than treating each as a separate world.

For Quebec, this is both a warning and an opportunity. The province already has valuable ingredients. Its scientific and clinical expertise gives it a strong starting point, and its ability to launch clinical trials quickly can make it attractive to international developers. But global pharmaceutical decisions are often made elsewhere. Investment, manufacturing, supply chains and product launches can therefore depend on priorities beyond Quebec’s control. A strong research ecosystem does not necessarily guarantee long-term access for local patients.

The question, then, is not whether Quebec should try to manufacture every advanced therapy itself. That would be unrealistic and potentially wasteful. The more useful question is how to decide what should be produced locally, what should be imported, and what capacities must exist to ensure that patients are not left stranded when markets shift. Local production may make particular sense for highly personalized treatments, strategically important therapies or products whose commercial future is uncertain. Imported therapies may remain the sensible choice in many other cases.

Making those choices well requires something more sophisticated than a race for self-sufficiency. It requires a coordinated strategy that begins with patient need and considers the full value of a therapy, the capacity of hospitals, the availability of skilled workers, the resilience of supply chains and the realities of public budgets. It also requires recognizing that the real bottleneck may appear long before a treatment reaches a patient.

That bottleneck may be money, regulation, manufacturing space, staffing, or simply the time required for several institutions to make decisions in sequence. If regulators, health technology assessors, hospitals and payers begin serious discussions only after a clinical trial succeeds, years can pass while patients wait. A more responsive system would bring these groups together earlier, allowing developers to understand what evidence will be required and hospitals to anticipate what implementation will demand.

Financing also needs to reflect the unusual nature of these treatments. Their costs often arrive upfront, while their benefits may unfold over years or decades. Yet long-term benefits can also be uncertain because some treatments are so new. This makes evaluation difficult. Paying immediately for promised lifetime benefits carries risk, but refusing access until decades of evidence accumulate defeats much of the purpose of innovation.

One response is to make reimbursement more flexible and connect access with continued evidence gathering. Patients could receive promising treatments while health systems collect real-world information about effectiveness, safety, quality of life and resource use. Such an approach would turn routine care into a source of learning, provided that data are collected consistently and patients remain central to decisions. Pan-Canadian registries could help to create the larger evidence base that is particularly important when individual diseases affect only small numbers of people.

The patient perspective is crucial here. Advanced therapies force difficult judgments about uncertainty, risk and value, and those judgments cannot be made entirely from institutional or economic viewpoints. Someone living with a severe disease may accept risks that seem unacceptable to a person evaluating treatment from a distance. Patients therefore need a meaningful role in decisions about approval, reimbursement and implementation, rather than being consulted only after the basic structure has already been designed.

The same principle applies to equity. A therapy is not genuinely accessible merely because it has regulatory approval. If receiving it requires travelling hundreds of kilometres, relocating temporarily, finding a specialist centre or waiting while hospitals struggle with capacity, access remains unequal. A sustainable strategy must consider where therapies are produced and administered, how patients reach specialized centres, and when cooperation across provincial or national borders offers a better solution.

Building capacity also means investing in people. Cell and gene therapies require expertise spanning laboratory science, manufacturing, clinical medicine, regulation, engineering and health economics. A sophisticated production facility is of limited value without enough qualified staff to operate it, just as excellent researchers can struggle to move discoveries toward patients without regulatory and manufacturing support. Integrated platforms could bring these skills together and give academic teams, hospitals and young companies a clearer route from an idea to a usable therapy.

There is also a danger in building expertise without creating careers that can sustain it. Advanced therapies depend on people who can move comfortably between research and clinical care, or between academic science, manufacturing and regulation. Yet conventional institutions do not always recognize these hybrid roles. Clinician-researchers, for example, may contribute to developing treatments and redesigning care while working within systems built around more traditional measures of clinical activity. Creating stable career paths, specialized training and opportunities to work across institutional boundaries would help Quebec retain expertise that might otherwise migrate elsewhere. Infrastructure, after all, includes laboratories and equipment, but it also encompasses the people who know how to make them useful.

For Prof. Jean-Sébastien Delisle and colleagues, the broader lesson emerging from this work is ultimately about coordination. Quebec does not lack talent or ambition. What it needs is a system that connects its strengths. The collaboration that underpinned this reflection, bringing together ThéCell, Héma-Québec, researchers, clinicians and other stakeholders, illustrates the type of collective approach that may be required to ensure that scientific advances translate into sustainable patient access. Regular dialogue among governments, researchers, clinicians, industry and patients could help the province to anticipate scientific developments instead of reacting after they arrive.

There is also a deeper idea behind these practical recommendations. Access to scientific progress is more than a commercial question. The framework developed around advanced therapies links innovation to the rights to science, health and non-discrimination. That perspective changes the standard by which success is judged. The goal is not simply to produce more discoveries, attract more investment or approve more products. It is to create conditions in which scientific progress can become meaningful improvement in people’s lives.

That will require difficult choices. Not every therapy can be produced locally. Public resources are finite, and enthusiasm for innovation cannot replace careful evidence. But caution can exist without inertia. A health system can be rigorous while becoming faster, more coordinated and more willing to learn.

The arrival of cell and gene therapies therefore poses a test larger than any single treatment. Modern medicine is gaining the ability to manipulate the building blocks of life with extraordinary precision. The institutions surrounding medicine must now become equally inventive. The next breakthrough may already exist in a laboratory, a manufacturing facility or a clinical trial. Whether it becomes a breakthrough for patients will depend on what happens next.