Earlier Than We Think.
The ultimate aim of drug development is not discovery for discovery’s sake. It is to solve a real healthcare problem and to ensure that the solution reaches as many patients as possible.
For that to happen, a medicine must achieve regulatory approval and sustainable access. These are not sequential hurdles—they are deeply interconnected. And that means discussions around access should not start at the end of development, but from the very beginning of the evidence plan.
Why “Early” Matters
In a fast-moving development environment, early-stage scientific teams often—understandably—focus on proof of concept, mechanism of action, and early efficacy. But success in today’s landscape requires more:
- Understanding the burden of disease and unmet need
- Knowing what competitors and standards of care already offer
- Selecting the right patient populations
- Incorporating patient-reported outcomes (PROs) that matter in real life
- Anticipating regulatory and payer evidence expectations
This is why early and regular touchpoints with regulatory and access-savvy colleagues are critical. Evidence plans should not be static documents, but living strategies, revisited as science, policy, and the external environment evolve.
With the introduction of the EU Joint Clinical Assessment (EU JCA), this need for early, forward-looking planning is now more important than ever.
The Shift Toward Integrated Evidence
The pharmaceutical industry is increasingly moving toward Integrated Evidence (IE) approaches. Historically, evidence generation was compartmentalized and sequential—clinical development first, access evidence later. This often resulted in misaligned priorities, gaps, and late-stage surprises.
An Integrated Evidence Plan (IEP) is designed to identify evidence needs across all stakeholders—internal and external—early in the development process.
Importantly, it balances short-term development goals with long-term success across the product life cycle.
Influencing Study Design for Long-term Success
Starting evidence generation early allows critical data elements to be embedded directly into clinical trial design—rather than added later through costly post-approval studies.
This includes:
- Comparative effectiveness signals
- Meaningful PROs
- Real-world–relevant endpoints
- Health system and cost considerations
When evidence is integrated from the outset, companies can build a coherent value narrative that supports the asset throughout its lifecycle—rather than retrofitting evidence to close gaps after approval.
Comparative Evidence: Still a Major Gap
As Naci and colleagues note, despite progress, fewer than half of new drugs in Europe and the US have robust comparative data at the time of approval. Even when active-comparator trials exist, they often fail to inform real-world decision-making.
In their landmark Lancet paper, Naci et al. (2020) argue that regulatory systems must create stronger incentives to generate comparative evidence useful to patients, clinicians, and payers. Their proposed principles—ranging from clearer labeling to payer-driven incentives—remain highly relevant in today’s accelerated approval environment.
The Takeaway
Access is not an “end-of-pipeline” topic. It is a design principle.
Early, integrated evidence planning:
- Improves study relevance
- Reduces uncertainty at approval
- Supports faster and smoother access decisions
- Ultimately helps medicines reach patients who need them – sooner
In a world of EU JCA, accelerated pathways, and increasing scrutiny on value, starting early is no longer optional.
This article was prepared with AI-assisted editing to improve clarity and structure. All perspectives and interpretations are grounded in published evidence and professional experience.
#IntegratedEvidence #EvidenceGeneration #MarketAccess #RegulatoryStrategy
#EUJCA #DrugDevelopment #PatientCentricity #HTA #LifeCycleManagement #AIassistedEditing
📘 DiMasi et al. (2025) highlight that integrated evidence approaches can significantly improve decision quality and development efficiency when implemented early and consistently.
Therapeutic Innovation & Regulatory Science, 59:808–816
https://doi.org/10.1007/s43441-025-00778-y
As highlighted in Forbes Business Council (2024), early evidence planning strengthens payer confidence, accelerates access discussions, and reduces downstream friction. https://www.forbes.com/councils/forbesbusinesscouncil/2024/12/16/why-developing-an-evidence-generation-plan-early-in-the-asset-life-cycle-of-pharmaceutical-products-is-critical/
📄 Generating comparative evidence on new drugs and devices before approval
The Lancet, Volume 395, Issue 10228, 986–997
https://www.thelancet.com/article/S0140-6736(19)33192-2/fulltext
