Miguel Forte, President, ISCT, board member of ARM and CEO of Kiji Therapeutics
Operational changes may be even more important than policy changes. One would be faster clinical trial approval and start-up. This is critical to accelerate evidence generation and enable project progression and funding. Another would be greater pragmatism and a broader view when making Health Technology Assessment decisions so that multiple perspectives on evidence could be considered, such as small sample sizes and single arm trials, particularly in orphan indications.
Payment models should better reflect the long-term value of these therapies, including future cost savings, while supporting more flexible health technology assessment and reimbursement decisions.
Daria Donati, Chief Scientific Officer of Genomic Medicine at Cytiva
I would prioritize greater global harmonization of regulatory and quality frameworks for advanced therapies.
Today, companies often have to navigate fragmented requirements across regions, which slows development and adds cost without necessarily improving patient safety.
A more aligned framework, particularly around comparability, analytics, and manufacturing changes would:
Enable faster iteration and innovation
Reduce duplication of effort
Accelerate patient access globally
Given the international nature of this field, global regulatory alignment could have an outsized impact on both innovation and patient access.
Daniel Vitt, Chief Executive Officer of Immunic Therapeutics
The clinical development burden has room for improvement. I'd expand the use of biomarker-based trial endpoints and conditional approvals. If we can demonstrate a clear therapeutic effect through validated endpoints and biomarkers, there could be an opportunity to enable earlier approval, followed by confirmation of long-term clinical outcomes post-approval in Phase 4 and real-world settings.
This approach could significantly reduce the time and cost required to bring new medicines to patients, while still maintaining high standards of safety and efficacy. It would also allow innovation to reach patients faster, particularly in areas with high unmet medical need. And, finally, this would also add more competition from smaller companies and make R&D investments much more attractive.
Earlier access combined with continued evidence generation could make drug development more efficient without compromising scientific rigor.
Ali Pashazadeh, CEO of Treehill Partners
Mandate that CROs provide an independent strategic assessment of the study they are being asked to run, separate from the commercial pitch to win the business. At the moment, the incentives in the outsourcing industry are structured around executing whatever the client brings to them as efficiently as possible – and with the industry having a large-pharma outsourcing legacy, that is understandable where “no questions asked” is usually the customer’s prerogative. Nobody is incentivized to say: this is the wrong study. But with biotech on the one hand being one of – if not the most – critical source of large pharma’s future blockbusters beyond their current patent cliffs, and on the other hand notoriously being short of capital and expertise, the approach needs to be different. If a structural separation existed between the advisory function and the operational delivery function – within the same organization – but both were mandated to be included, we could see a meaningful reduction in avoidable clinical failures. The automotive industry does not allow the manufacturer to certify its own safety. Drug development market practice should not allow the service provider to leave invalidated the strategic rationale of the program it is commercially incentivized to execute. For the time being, the buck stops with management and boards – whilst investors lean back asking companies to show them how their path to commercial success is more financially attractive on a risk-adjusted basis than running a similar program through a Chinese competitor.
Renee Aguiar-Lucander, CEO, Hansa Biopharma
I would reduce the regulatory focus on demanding a randomized trial in Phase 1/2 in rare diseases and make greater use of real-world evidence. We have learned so much over the past 30 years; we should use this knowledge more effectively. Phase 3 would still rely on randomized controlled trials.
Michael May, CEO, CCRM
Cell and gene therapies are arguably the most complex medicines ever developed, and their complexity will continue to grow. Consequently, it is likely that their mass adoption will require the greatest amount of human collaboration ever seen in therapeutic development. This will require regulatory policy (and other activities) to evolve so that prior collective knowledge and experience can be leveraged in the approval process. It will be unsustainable if personalized medicines or gene therapies for rare disorders are not able to leverage collective, modular development and shared submissions.
Rab Prinjha, Chief Research and Development Officer, Curve Therapeutics
I would love to see policies enacted that make it easier and cheaper to recruit talent internationally and reduce barriers that prevent the very best scientists from moving between companies, countries, and continents.
Jane Rhodes, Chief Executive Officer, AstronauTx
One policy with significant potential is the Mansion House Compact, which aims to unlock greater investment in UK biotech and the life sciences. This is currently voluntary and reinforcing aspects of this policy through legislation, making is more directly relevant to life science investment could accelerate and amplify the impact releasing more growth capital and enabling biotech growth in the UK.
Bruce Levine, Barbara and Edward Netter Professor in Cancer Gene Therapy, University of Pennsylvania Perelman School of Medicine
I would stop regulating and reimbursing advanced therapies as if they were ordinary pharmaceuticals. Cell and gene therapies are living, durable, complex medicines. They need rigorous standards, but also appropriately calibrated and ethical trial designs, adaptive manufacturing rules, real-world evidence systems, and payment models that recognize long-term value. Otherwise, we will keep proving that these therapies work while failing to get them to patients.
Jerry McMahon, CEO, STORM Therapeutics
I would continue reducing reliance on large animal testing as more predictive, human-relevant models become available. Advances in in vitro systems, organoids, and computational approaches are starting to provide more translatable insights. Supporting broader adoption of these approaches could improve efficiency and contribute to more informed decision-making in drug development.
Claudia Zylberberg, PhD, co-founder and board chair of ARScience Bio, founder and chair of Akron Bio, and co-founder and board chair of Kosten Digital
I would focus less on creating new regulations and more on modernizing the frameworks we already have.
Today, drug pricing, reimbursement, and healthcare economics are under intense scrutiny, yet many of the policies governing how innovative therapies are evaluated, reimbursed, and adopted were designed for a different era of medicine. Advanced therapies, precision medicine, and AI-enabled healthcare require new ways of thinking about value, outcomes, and patient access.
A thoughtful push from policymakers, regulators, and payers to update these frameworks could create the guardrails needed for innovation to thrive while ensuring accountability and sustainability.
The reality is that the science is moving faster than the systems designed to support it. As a result, we risk creating remarkable innovations that patients cannot access efficiently or affordably.
I also believe we need greater alignment among regulators, payers, providers, manufacturers, and patients. Too often, each stakeholder is optimizing for a different objective. Progress will accelerate when we start measuring success by impact rather than process.
The future is not about having more agents, more platforms, or more complexity. It is about creating greater impact for patients. Policies that reward outcomes, encourage collaboration, and support responsible innovation would go a long way toward helping this industry reach its full potential.
Jason Bock, PhD, CEO of CTMC
I would create a more explicit regulatory framework for iterative manufacturing improvement in cell therapy.
Everyone agrees that cell therapy manufacturing needs to become faster, more automated, more consistent, and less expensive. But the regulatory risk of changing a process during development — and especially later in development — can be substantial. That creates a paradox: the field needs innovation in manufacturing, but companies are often afraid to introduce improvements because comparability can be difficult, expensive, and uncertain.
I would like to see clearer, more predictable pathways for platform-based manufacturing changes, especially when companies can demonstrate strong analytical understanding and product comparability. This does not mean lowering standards. Patients need safe, potent, well-controlled products. But we need regulatory mechanisms that encourage continuous improvement rather than locking early, imperfect processes in place.
A better framework would accelerate the adoption of automation, improved analytics, closed processing, shorter culture systems, and lower-cost manufacturing. That would help patients because it would make therapies faster to deliver, easier to scale, and more economically sustainable.
Ali Tavassoli, Professor of Chemical Biology, University of Southampton; and Former Chief Scientific Officer and Co-founder, Curve Therapeutics
Stronger pull incentives for areas of clear market failure. There are whole categories of medicine where the science is tractable, the patient need is undisputed, and the commercial model simply does not work; antimicrobial resistance most obviously, but also rare pediatric diseases, neglected tropical diseases, and pandemic preparedness. In each case, the conventional volume-based pricing model that funds most of pharma fails to align with what society actually needs. A novel antibiotic that works is, paradoxically, one we want to use as little as possible. A medicine for a rare pediatric disease serves a population too small to support the cost of development under normal economics. The result is that companies have systematically exited these spaces, and the pipelines are dangerously thin at exactly the moment we need them to be deep.
The fix is pull mechanisms that guarantee revenue, subscription-style payments, market-entry rewards, and priority review vouchers. We need systems that decouple the financial return from the volume sold. The UK's NHS antimicrobial subscription pilot was an important proof of concept, and similar models are being developed elsewhere. What is needed now is for this kind of structure to become the global default in areas of market failure rather than a handful of national experiments. Policy can fix markets where markets cannot fix themselves, and that is where governments have the most leverage to accelerate progress in the medicines we most urgently need.
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