For decades health technology assessment (HTA) has played a central role in determining whether new treatments are reimbursed and made available to patients. Today, HTA decision-making is evolving to incorporate more structured input from a larger set of stakeholders, including patients, clinicians, payers, and policymakers, each of whom evaluates therapies through a different lens. Given the growing influence of diverse stakeholders, it is critical that manufacturers use a tailored approach to engage with them early and strategically.
Companies that understand the type of evidence decision-makers need, demonstrate the value of their products in ways that resonate, coordinate internally to speak with one voice, and build support for innovative treatments throughout the HTA process are more likely to succeed in reaching more patients more quickly.
HTA is no longer just a technical exercise
In recent years, HTA has shifted from a purely technical assessment of clinical and cost-effectiveness to a decision-making process shaped by multiple stakeholder perspectives and health system priorities. Although strong clinical and economic evidence remains essential to secure reimbursement and patient access, it is no longer sufficient on its own. Factors such as political priorities, budget constraints, health system pressures, and public expectations increasingly influence pricing and reimbursement decisions.
At the same time, different stakeholders – patients, clinicians, policymakers, and payers – often apply different definitions of value when evaluating therapies. While payers may prioritize affordability and budget impact, patients and clinicians may place greater emphasis on health outcomes, quality of life, ability to carry out everyday activities, and overall unmet need among those with a particular disease or condition.
For pharmaceutical companies, these dynamics mean that it is more important than ever to engage stakeholders early to determine evidence requirements, value drivers, and system priorities before formal HTA submission. An effective stakeholder engagement strategy requires coordination across a company’s internal functions, including government affairs, public affairs, medical affairs, outcomes research, and commercial teams, to ensure that evidence generation, policy positioning, and value communication are consistent and credible throughout the access journey.
Tailor value stories to different stakeholder groups
A common pitfall for pharmaceutical manufacturers is assuming that a single value narrative will resonate with all stakeholders. The table below summarizes different groups’ priorities regarding the value of innovative therapies and recommendations for communicating the right value story:
| Patient advocacy groups | Clinicians | Policymakers | Payers | |
| Priority focus | Quality of life, unmet need, and outcomes captured through patient-reported measures | Clinical relevance and impact on treatment pathways in real-world practice | System-level impact, including how the therapy compares to alternatives and fits within broader health priorities, and whether it’s a good use of limited health system resources | Affordability, budget impact, and predictability of use over time |
| Recommended value story | Demonstrate meaningful improvements in daily life and functional outcomes, and be transparent about access considerations | Show where the therapy fits in clinical practice and how it changes treatment decisions for specific patient groups | Position the therapy within broader system priorities and demonstrate value relative to competing health investments | Provide clear assumptions on patient population, uptake, and total cost, and address budget uncertainty directly |
| Key question | “Will this meaningfully improve patients’ daily lives?” | “Would I use this in practice, and for which patients?” | “Is this the best use of limited health system resources?” | “What will this cost, and can we manage it?” |
| Recommended engagement approach | Engage early with patient groups to ensure outcomes reflect what matters most to patients | Align early with clinicians on evidence, endpoints and comparators used in practice | Connect the value narrative to health system priorities and policy objectives | Be transparent about cost, utilization, and uncertainty, and demonstrate understanding of budget constraints |
Disease area changes the stakeholder equation
Stakeholders’ priorities also vary depending on disease area – and engagement strategies must adapt accordingly. What payers, clinicians, and patient groups care about for people being treated for cancer will not necessarily translate to rare diseases or chronic conditions. There is no “one size fits all” HTA engagement playbook.
For example…
- In assessing oncology therapies, HTA bodies and payers closely examine whether these products deliver meaningful clinical benefit compared with existing treatment options. Clinicians may see value in evidence showing that a treatment can delay the cancer from progressing, particularly in certain types of cancer where treatment options are changing quickly. However, payers often need stronger evidence that these benefits translate into outcomes such as helping patients live longer, improving quality of life, or reducing pressure on the health system.
Recommendations for pharma: Support claims of value with evidence that reflects real-world clinical benefit and demonstrates how the therapy improves patient care relative to the current standard of care.
- When examining treatments for chronic diseases, patients and clinicians often prioritize long-term health outcomes, quality of life, adherence, and caregiver burden. Payers, however, tend to focus more on the sustained financial impact on the health system, including reductions in hospitalizations, complications, or long-term treatment costs. Traditional economic models do not always capture these dimensions effectively.
Recommendations for pharma: Generate evidence showing how their products improve outcomes that matter to patients living with a chronic disease as well as reduce healthcare utilization and long-term system costs.
- Rare diseases are often a special HTA challenge because they affect small patient populations (although with great unmet need), clinical trials have few participants and, therefore, limited data, and evidence is based on surrogate or single-arm studies. The small patient populations and limited clinical evidence create uncertainty about a treatment’s true effectiveness.
While all stakeholders recognize these constraints, their tolerance for uncertainty varies. Patients emphasize unmet need and urgency; while payers focus on the reliability of evidence and financial implications of high-cost therapies.
Recommendations for pharma: Focus on generating evidence to manage uncertainty about a product’s effectiveness over time. For example, commit to establishing disease registries or conducting post-launch studies. Or take a bolder approach, such as structuring outcome-based managed entry agreements where reimbursement or pricing is linked to whether the therapy achieves agreed upon outcomes in real-world use.
Engage early to shape HTA outcomes
Another shift in the current HTA environment is the timing of engagement. Increasingly, opportunities for influence are happening earlier in the assessment process through formal scientific advisory groups and more structured stakeholder input. Companies that engage only at the point of dossier submission are often managing risk too late. In contrast, those that align early on endpoints, comparators, and evidence plans can manage stakeholder expectations better and strengthen credibility with decision-makers.
Speaking with one voice is critical
Internal coordination within companies is essential to develop a consistent and credible value narrative. Government affairs, market access, medical affairs, and commercial teams must align on priorities, messaging, and timing of communications. Evidence generation plans, policy positioning, and stakeholder engagement strategies should be developed in parallel rather than in silos, where inconsistencies can undermine trust with external stakeholders.
Leading manufacturers are increasingly coordinating cross-functional teams and taking an integrated approach earlier in the product development process to better anticipate stakeholder concerns, align evidence strategies, and communicate value consistently across markets. In the continually evolving HTA environment, internal alignment is not only operationally important, but a strategic advantage.
The real differentiator is engagement, not just evidence
As HTA frameworks mature globally, clinical effectiveness and analytical rigor remain essential, but they are not enough. Manufacturers that succeed in this changing environment recognize that definitions of value vary across stakeholders, therapeutic areas, and the criteria used to evaluate new therapies.
Rather than relying solely on technical strength of the evidence at submission of a dossier, companies are investing in early, tailored stakeholder engagement strategies to guide evidence generation plans and build credibility throughout the HTA process.
For leaders in government affairs and market access, the implication is clear: strategic stakeholder engagement is no longer a supporting activity, but a core component of successful market access. Ultimately, stronger alignment across stakeholders will lead to more favorable and faster reimbursement decisions and ensure that patients can benefit more quickly, consistently, and equitably from innovative therapies.