Analogue analysis in rare diseases plays a central role in how we think about value, evidence and positioning for orphan therapies. It shapes pricing strategy, HTA positioning and value communication; yet in rare diseases, analogue assessment is rarely straightforward.
Unlike established therapy areas with clearly defined treatment pathways, orphan drug development often lacks a single obvious reference point. Evidence standards vary, endpoints may be unfamiliar to payers, and patient populations are small or heterogeneous.
Analogue assessment for orphan drugs is more like assembling a puzzle: no individual piece gives you the full picture, and the way the pieces are chosen and fitted together ultimately determines the quality of the answer.
Fortunately, I’ve always been a fan of puzzles. I find the process of laying out the pieces, testing different combinations, and slowly arriving at something coherent deeply satisfying. Perhaps that’s why rare diseases have come to occupy so much of my work.
A structured, question-led analogue scorecard approach can improve transparency, defensibility and strategic alignment when selecting comparators for rare disease HTA and pricing decisions.
Why Analogue Analysis Is More Complex in Rare Diseases
In common diseases, analogue selection typically follows predictable patterns. Standards of care are well established. Historical precedents exist. Payers are familiar with clinical endpoints and benchmarks.
In rare diseases, those anchors are often absent. The challenges orphan medicines face are quite different from those in non-orphan indications:
- Limited or evolving standard of care
- Small and heterogeneous patient populations
- Novel or surrogate endpoints
- Uncertainty around long-term durability of benefit
- Variable HTA precedents across markets
As a result, searching for a single “perfect” analogue can be misleading, if not impossible. At best, it risks oversimplifying the value story. At worst, it leads teams to abandon analogue research altogether because nothing seems to fit.
For orphan medicines, analogue analysis must be multi-dimensional.
Analogue assessment as a puzzle, not a match
Thinking about analogue research as a puzzle helps reframe the task. Rather than identifying one closest match, effective analogue analysis assesses multiple potential comparators across defined strategic dimensions. The objective is not to identify the best analogue overall, but the most informative analogue for a specific decision.Teams often default to referencing high-profile orphan drug successes. However, using a single comparator to inform pricing or HTA strategy risks oversimplification. One analogue may be helpful for understanding evidence standards. Another may offer insight into endpoint relevance. A third may help frame expectations around target population size or unmet need.
No single comparator answers everything: I’ve lost count of the number of times I’ve had to gently wave Zolgensma away as an analogue. Not because it isn’t a remarkable success story, but because it simply isn’t the right reference for every situation.
The strength of analogue assessment lies in structured comparison, not intuitive selection.
A Question-Led Framework for Comparator Selection
Effective analogue analysis begins with a clearly defined strategic question:
- Are we assessing pricing potential?
- Are we testing payer acceptability of the evidence package?
- Are we informing clinical development strategies?
- Are we shaping value communication?
Different questions require different lenses. A structured analogue scorecard makes this explicit.
The Four Dimensions of a Rare Disease Analogue Scorecard
Adjusting the Strategic Lens
As previously mentioned, the same analogue may be highly informative in one context and less relevant in another. A defining feature of a structured analogue analysis scorecard is dynamic weighting depending on the strategic question.
For example:
- When assessing pricing and value framing, nature and magnitude of benefit may carry greater weight.
- When evaluating HTA risk, the evidence package may be the dominant consideration.
- When informing development strategy, population definition and endpoint alignment may take precedence.
Weighting does not produce a single definitive answer. Instead, it clarifies why a comparator is relevant for a particular strategic question, and makes it clear why the same analogue may be highly relevant in one context and much less so in another. This improves cross-functional alignment and strengthens internal and external defensibility.
Practical Implications for Rare Disease Market Access Strategy
In practice, a structured analogue scorecard approach enables:
- More transparent and defensible comparator selection
- Clear articulation of strategic assumptions
- Stronger alignment between global and local teams
- Better justification of pricing recommendations
- Improved anticipation of HTA scrutiny
Importantly, it does not remove uncertainty. It makes uncertainty explicit, structured and easier to navigate.
From Analogue Research to Strategic Capability
In rare diseases, analogue analysis should not be treated as a shortcut to pricing; it is a strategic capability.
When applied rigorously, analogue assessment supports:
- Early value assessment
- Evidence generation planning
- Pricing corridor modelling
- HTA scenario planning
- Value communication strategy
For orphan medicines, where precedent is limited and scrutiny high, structured analogue analysis provides discipline without oversimplification. The result is not a single “right” analogue, but a more thoughtful, transparent, and credible decision-making process
Download the Rare Disease Analogue Scorecard
If you would like a structured template for applying this framework, a downloadable rare disease analogue scorecard and example analysis is available on request.
Download the resource
About the author
Isabel is a Partner at Access Infinity and has supported pharma companies in developing their Pricing & Market Access strategy for products across all major therapeutic areas. She’s worked with several orphan drug manufacturers from the earliest stages of drug development through to launch readiness, to ensure the value of their therapies is recognised by payers.
If you have a question or a project in mind, get in touch with Isabel by emailing isabel@accessinfinity.com or via our contact us page.
Frequently Asked Questions
Analogue analysis in rare diseases is a structured framework used to evaluate comparable orphan medicine launches across defined dimensions to inform pricing, HTA strategy and value positioning.
Rare diseases often lack established standards of care, involve small patient populations and use novel endpoints, making direct comparison complex and multi-dimensional.
HTA bodies typically evaluate the competitive landscape, nature of benefit, robustness of evidence, and clarity of target population when assessing comparability.
No. A structured approach evaluates multiple comparators across defined dimensions, weighted according to the strategic question being addressed.
