Build the evidence payers need. Learn how to identify evidence gaps, anticipate payer and HTA requirements, and build a plan before your options narrow.
Building the Evidence That Payers Care About
A Practical Guide to Building an Evidence Strategy That Optimizes Reimbursement
A strong clinical trial can demonstrate that a therapy is safe and efficacious. However, payers and health technology assessment (HTA) bodies ask different questions: does the therapy provide enough value, compared with available options, to support coverage and reimbursement?
Evidence strategy helps a company anticipate that question and build the right evidence before key development decisions are fixed.
Regulatory approval and payer access are separate decisions.
Payers often need comparative, economic, real-world, and patient-reported evidence beyond the data required for approval.
Planning before Phase 3 creates more options and reduces the need for costly evidence generation later.
A useful evidence plan prioritizes gaps by business impact, timing, and feasibility.
Evidence strategy is the plan for generating and using information that supports decisions across a product's lifecycle. It begins with the needs of the decision-makers who will evaluate, pay for, prescribe, and use the therapy. It then compares those needs with the evidence the clinical development program is expected to produce.
The result is a clear view of what is covered, what is missing, and when each gap should be addressed.
This connects clinical development, health economics and outcomes research (HEOR), medical affairs, market access, and commercial planning around one set of priorities.
A practical strategy should identify priority decision-makers and markets, define their likely evidence needs, assess the planned evidence package, and prioritize the remaining gaps. Each recommended activity should have a clear purpose, owner, timing, and link to a future decision.
A study earns its place by improving a coverage, reimbursement, pricing, or treatment decision.
Your company has just received positive Phase 2 data and is gearing up for a Phase 3 filing. At some point someone (your board of directors, your newly hired Head of Commercial, your investors) asks a question nobody has prepared an answer for:
"What are the payers going to need?"
This is a common position to be in. Scientific teams routinely spend years carefully designing trials and hitting their primary endpoints without anyone checking whether the resulting data will support a payer decision.
Evidence strategy is the work of getting from "our drug works" to "payers will cover our drug at a price that keeps the lights on."
How does the therapy perform against the treatment used in current practice (standard of care)?
Does treatment improve symptoms, daily functioning, or quality-of-life in ways that matter to patients?
How could the therapy affect total cost of care, health care use, or value for money?
Do outcomes hold across a broader and more varied patient population than those in the clinical trial?
How large is the unmet need for patients, caregivers, health systems, and society?
Each phase closes a set of choices. Knowing which ones close when is what makes early planning worth the effort.
Outcome measures
Comparison and patients
The pivotal trial
The value story
Which COA and PRO measures the trial will use, and whether they suit your patients.
The comparison, dose, and patient group the pivotal trial will use.
Which results are measured, and for how long, once the pivotal protocol is final.
At launch you can only argue from data you already have.
The next page covers what to settle before the pivotal trial begins.
Alkemi helps life sciences companies translate payer and HTA requirements into a focused evidence plan. We assess the clinical program and external landscape, identify the gaps that matter, and recommend practical actions across clinical trials, HEOR, real-world evidence, and patient research.
This gives teams a shared roadmap for investing in evidence that supports both development and access.
The pivotal trial is the one meant to support approval. It is usually Phase 3, but in rare disease and some cancers a Phase 2 trial is the pivotal one, which moves every decision below earlier.
Which clinical outcome assessments (COAs) and patient-reported outcome (PRO) measures the trial will use. Choosing and preparing them takes time, so this work starts well before the protocol.
The comparison, dose, and patient group. Payers compare against what doctors prescribe today, which is not always what a regulator will accept.
Which additional results to collect, how often, and for how long. This is usually where the quality-of-life claims come from.
Which results the label can mention, and what regulators will accept as proof of benefit.
What the cost and value analysis will need to address, and where that data comes from.
Which comparisons and patient groups payers and HTA bodies look for.
These decisions are usually written down in one place, as an Integrated Evidence Generation Plan: what evidence the program will produce, who each piece is for, when it is needed, and who owns it.
One plan keeps clinical development, regulatory, HEOR and market access working from the same list.
Evidence planning is most valuable before the Phase 3 protocol is finalized. At that point, the team may still be able to adjust comparators, endpoints, patient-reported outcome measures, subgroups, and follow-up periods.
Later planning can still improve the launch evidence package. Teams may be able to strengthen analysis plans, conduct burden-of-illness or patient preference research, develop economic models, or build a parallel real-world evidence program.
The available choices narrow over time, which makes early prioritization important.
Faster approval leaves less time, so payer evidence has to be built alongside the filing rather than after it.
Regulators may accept an early marker of benefit that payers do not, which puts more weight on the COA and PRO plan.
The confirmatory trial becomes the main evidence payers see, so design it for both audiences.
Launching on limited data usually means committing to collect real-world evidence later.
Alkemi turns payer and HTA requirements into an evidence plan a development team can act on. We assess the clinical program and the external landscape, rank the gaps that matter, and set out practical work across trials, HEOR, real-world evidence, and patient research, including support on study design.
Most engagements start with a strategic assessment of four to six weeks. You finish it with a prioritized list of evidence activities, each with timing, an owner, and an estimate of what it costs. We are 16 people, so you work with the people doing the thinking rather than a team you never meet.
A question we hear from biotech CEOs and heads of development: "Do we need to hire an HEOR team, or can we outsource this?" It depends mostly on where the program sits.
A full-time HEOR hire is usually premature. What helps is a partner who can set the evidence plan, choose the outcome measures, and tell you which questions to take to regulators and payers while the answers can still change the program.
You probably want both. Someone internal who owns the plan for the long run, and an external partner who has watched this happen before and knows which gaps show up at submission.
Late, but recoverable. A parallel real-world evidence program and focused economic work can still close gaps. It costs more and moves faster than it should. Call earlier if you can.
A literature review catalogues what has been published. An evidence gap assessment compares that body of work against what specific decision-makers will require at the time of your launch, which is a different exercise and usually a more uncomfortable one.
Knowing where the gaps sit is what changes how you spend the next two years.
Start with a strategic assessment. This is typically a 4-6 week engagement where an external partner reviews your clinical program, target product profile, and the competitive and payer landscape. That provides you with a prioritized list of evidence activities with timelines and estimated investment.
It works as a blueprint: you are not committing to the studies yet, only establishing what they will have to cover.
Rare disease works differently. Payers generally understand that you won't have large randomized controlled trials with 10-year follow-up. But you still need to demonstrate clear unmet need, meaningful clinical benefit, and a defensible price. Real-world evidence, patient registries, and natural history studies carry more weight here. And patient advocacy counts for a great deal.
Sometimes the most powerful evidence is a parent who can explain what this disease does to a family.
Probably not. You may have less flexibility but there's usually room to add secondary endpoints, pre-specify subgroup analyses, or layer in a real-world evidence strategy that runs parallel to your trial.
Assuming it is too late and doing nothing guarantees the gap stays open.
At Alkemi, we help biotechnology and pharmaceutical companies build evidence strategies that connect clinical development to commercial success. We're 16 people, not 16,000. That means you work directly with the people who actually do the thinking.
If you're sitting on positive Phase 2 data and wondering what payers will need in your Phase 3 or if you're six months from filing and realizing you have gaps, get in touch. We have worked with companies in both situations and can tell you what to do next.
