Disease-Target Selection Screen
Prioritize diseases where science, technology, need, and impact align
- Difficulty
- Moderate
- Time to result
- ~weeks to results
- Steps
- 4
- Confidence
- 90%
The Disease-Target Selection Screen evaluates a prospective research area through four linked filters: prior scientific knowledge, enabling technology, unmet patient need, and the possibility of meaningful impact. Existing research provides a foundation rather than forcing the team to begin from zero. Technology must either already exist or appear feasible for the organization to develop. The disease must impose a significant burden that current care does not adequately address. Finally, the team asks whether its capabilities can realistically change outcomes. The screen produces a focused research portfolio by excluding attractive but poorly supported opportunities and prioritizing diseases where evidence, tools, need, and organizational capability reinforce one another.
Origin
Extracted from Masters in Business, in a sponsored episode describing how Vertex Pharmaceuticals selects challenging diseases for research.
Core principles
- 01Build on credible prior research
- 02Require usable or developable technology
- 03Prioritize substantial unmet need
- 04Pursue targets where meaningful impact appears possible
How to run it
- 1
Map the Scientific Foundation
Determine whether a credible history of research explains enough of the disease to support a focused investigation. Identify the strongest evidence, unresolved questions, and relevant biological mechanisms.
Pro tip Distinguish foundational evidence from assumptions that have merely been repeated.
Watch out A large literature does not necessarily mean the underlying mechanism is well understood.
- 2
Evaluate Enabling Technology
Identify tools that can interrogate or modify the relevant mechanism. If the technology does not exist, assess whether developing it is technically and economically realistic.
Pro tip Favor tools that connect biological hypotheses to measurable outcomes.
Watch out Do not treat speculative technology as though it were already validated.
- 3
Confirm Unmet Need
Assess the number of affected patients, severity of the condition, and limitations of current treatments. Establish whether better care would materially improve patients' lives.
Pro tip Include tolerability, addiction risk, and treatment burden when evaluating existing options.
Watch out Prevalence alone does not establish that the proposed approach addresses the most important need.
- 4
Test the Potential for Impact
Ask whether the organization has the expertise and resources to make a meaningful difference. Advance the target only when scientific feasibility and patient value plausibly converge.
Pro tip State the expected patient-level change before committing major resources.
Watch out Avoid selecting a disease solely because it is scientifically interesting.
In the wild
Pain has centuries of treatment history, emerging knowledge about pain-sensing proteins, and technology capable of investigating signal transmission. Tens of millions of people experience acute or chronic pain, while commonly used opioids carry tolerability and addiction concerns. These factors create a case for researching non-opioid approaches that act on the underlying transmission mechanism.
→ Pain passes the screen as an area with scientific foundations, enabling research tools, substantial unmet need, and potential patient impact.
Common mistakes
Starting With Technology Alone
A novel tool does not justify a disease program unless it addresses a well-supported mechanism and a meaningful patient need.
Equating Market Size With Unmet Need
A common disease may still be a weak target if existing treatments work well or the proposed intervention offers little improvement.
Ignoring Organizational Fit
A promising disease can consume resources without producing results when the team lacks the capabilities required to affect it.
Is it for you?
Best for
It is best for biotechnology leaders and research teams allocating limited resources across possible disease targets.
Not ideal for
It is not ideal for projects driven primarily by short-term commercial opportunity without sufficient scientific evidence.
From the transcript
“There's a long history of research to build on, there's new technology available or Vertex thinks they can develop it. There's a large unmet need…”
“The real question in our mind is can we succeed? Can we make a difference?”
From the episode
Targeting Pain (Sponsored Content)