Beyond the Molecule: Redesigning Pharma Innovation for Real-World Impact

Introduction: The Innovation Paradox

Pharma has never been more scientifically capable. Gene therapies are rewriting what we thought possible. AI is reshaping discovery and development. Precision medicine is turning blanket treatments into targeted solutions.

And yet—patients, payers, and health systems face a sobering reality. Too many breakthroughs don’t translate into timely, equitable access. Too many products stall in negotiations, face reimbursement hurdles, or fail to demonstrate value outside clinical trial conditions.

This is the paradox at the heart of pharma today: scientific success doesn’t guarantee societal success.

If we’re serious about improving global health outcomes, we need to think beyond the molecule. That means re-engineering how innovation is designed, developed, and delivered—starting not with the science alone, but with systems, access, and patients.

1. The Access Gap: Why It Matters More Than Ever

Science is advancing faster than systems

New modalities (cell therapies, RNA-based treatments, curative interventions) come with unprecedented promise—but also unprecedented cost, infrastructure requirements, and delivery challenges. Health systems aren’t always ready to absorb them.

Patients are waiting longer

Studies show that across Europe, for example, the time between EMA approval and patient access can vary by years depending on the country. For patients with life-limiting conditions, those delays are devastating.

Public trust is fragile

The pandemic revealed both the potential and pitfalls of pharma. On one hand, vaccines demonstrated what’s possible with urgency, investment, and collaboration. On the other, mistrust, misinformation, and uneven global access exposed the cracks in our approach.

If innovation cannot reach patients, it risks undermining the very trust and legitimacy the industry depends on.

2. Access-First Innovation: A New Mindset

Historically, pharma’s mindset has been “science first, access later.” Develop the product, prove the efficacy, then engage with regulators, HTA bodies, and payers to justify value.

That sequence no longer works.

Access-first thinking means:

  • Co-designing with payers and regulators. Instead of submitting dossiers at the end, involve them upfront in shaping endpoints, comparators, and evidence generation.
  • Rethinking trial design. Randomised controlled trials (RCTs) are critical but insufficient. Real-world evidence, pragmatic trials, and patient-centred outcomes need equal weight.
  • Planning affordability early. Too often, price is determined after years of R&D investment, with little room for flexibility. A directional access-first model considers budget impact, health equity, and long-term sustainability from day one.
  • Seeing innovation as system change, not just product launch. A one-off therapy that “cures” a disease changes not only patients’ lives but also the economics of health systems. Preparing for that disruption is part of the innovation itself.

This is not just a moral imperative—it’s a strategic one. Companies that fail to think this way risk seeing their products stall, rejected, or underutilised.

3. Leadership for a New Era: The Square Peg Advantage

Access-first innovation requires different leadership qualities than pharma has traditionally rewarded.

Specialists vs. Generalists

Pharma has long valued deep specialisation: the scientist who knows one pathway, the regulatory expert who masters one dossier format, the commercial lead who focuses on launch KPIs.

But the future belongs to versatile leaders—those who can move across science, systems, and strategy. People who are comfortable with ambiguity, skilled at collaboration, and unafraid to challenge silos.

These “square pegs in round holes” are often undervalued in traditional corporate structures. Yet they are precisely the leaders who can connect dots, foresee downstream access challenges, and build bridges across disciplines.

Three capabilities that matter most:

  1. Systems Thinking – understanding how payers, providers, regulators, and patients interconnect, and designing innovation with those systems in mind.
  2. Adaptive Collaboration – bringing stakeholders into the process early, and co-creating rather than negotiating after the fact.
  3. Courageous Storytelling – not just presenting data, but framing it in ways that resonate with policymakers, payers, and the public.

4. Practical Steps Towards Access-First Pharma

So what does this look like in practice? Here are directional steps pharma leaders can take now:

  1. Redefine success metrics. Shift from “Did we launch on time?” to “How quickly did patients get access?”
  2. Integrate payer/regulator voices into early-stage development. Pilot “co-creation boards” where HTA representatives input into trial designs.
  3. Invest in real-world data ecosystems. Build partnerships with health systems to collect evidence continuously, not just at launch.
  4. Embed affordability into portfolio strategy. Don’t leave pricing to the eleventh hour; pressure-test different access models before pivotal trials.
  5. Reward cross-functional talent. Redesign incentive systems to value collaboration and systemic impact, not just siloed KPIs.
  6. Communicate transparently. Trust is built not by perfection, but by openness about trade-offs, uncertainties, and long-term value.

5. The Future: Where Science Meets Systems

Imagine a world where:

  • A therapy is designed with the patient journey in mind from day one.
  • Regulators and payers co-shape endpoints to ensure evidence is both scientifically valid and system-relevant.
  • Innovation pipelines are judged not just by molecules discovered, but by patients reached.

That future is not utopian—it’s necessary.

As costs rise, populations age, and health systems strain, pharma’s licence to operate will increasingly depend not just on what we discover, but how we deliver it. The companies that lead in access-first innovation will not only succeed commercially, but also rebuild trust and legitimacy for the entire industry.

Conclusion

Pharma has the tools, talent, and technologies to change the future of health. But unless we embed access into the DNA of innovation, we risk creating miracles that never make it to the bedside.

It’s time to move beyond the molecule.

It’s time to think access-first.

And it’s time to empower a new generation of leaders—those who can bridge science and systems, strategy and society.

The question is not whether we can innovate. It’s whether we can innovate in ways that truly matter.

Over to you:
👉 What do you see as the biggest barrier to access-first innovation today—science, systems, or mindset?
👉 And how can we, as an industry, redesign incentives to reward true impact, not just launch?

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