AIMS Service Category

Development Rationale and Goal Alignment

Align disease, patient population, and pipeline selection into one development logic.

AIMS reviews the candidate mechanism, available evidence, disease characteristics, target population,
and competitive landscape to define why this disease, why this patient group, and why this pipeline should be prioritized.

TPP Indication Rationale Target Population Rationale Positioning Statement Value Proposition
Why it matters

A coherent rationale keeps downstream evidence generation focused.
When the disease, target population, and pipeline rationale are not aligned early, nonclinical studies, early clinical trials, TPP, and partnering messages may move in different directions.

Recommended for companies with a candidate asset
but an unclear development direction.

This category supports clients that have a mechanism or candidate asset but cannot yet explain, in a coherent development logic, which disease and patient group should be prioritized and why the pipeline should be selected by investors or partners.

AS IS
  • The candidate has technical strengths, but the first development disease is unclear.
  • Multiple indications are possible, but the entry indication and expansion path are not separated.
  • The target patient population is broad or vague, making early clinical value difficult to define.
  • Differentiation versus competitors is abstract and weak for investor or partner communication.
TO BE
  • A disease rationale linked to medical unmet needs and the candidate mechanism is defined.
  • The entry indication and follow-on expansion opportunities are separated.
  • The target population and early development direction are made concrete.
  • Differentiation logic that can feed the TPP and license-out message is established.

What decisions are clarified?

This category organizes three early development questions: why this disease, why this patient population,
and why this pipeline. When these answers connect, evidence generation and external communication become clearer.

Why this disease?

Link treatment gaps, disease burden, and limitations of current care to the need for new drug development.

01

Why this patient group?

Define the patient population and treatment setting in which pipeline value can be demonstrated first.

02

Why this pipeline?

Connect the mechanism, available evidence, formulation, modality, and feasibility to the selected rationale.

03

Service modules

Service modules are organized around the actual decisions clients need to make at the beginning of development.

Disease and Pipeline Rationale

This service connects treatment gaps, limitations of current care, the chosen development approach, mechanism, evidence, and formulation/modality/route considerations into a clear answer to “why this disease and this pipeline.”

Core question
Why should this pipeline be developed in this disease?
Why this service matters
Even strong nonclinical results may not convince investors, partners, or regulators unless they are
linked to a clear disease and pipeline rationale.

What can go wrong
if this is not addressed?

01

Weak disease rationale

Medical unmet needs and candidate evidence may remain disconnected.

02

Weak development approach rationale

It may be difficult to explain why a FiC, BiC, reformulation, or combination strategy is appropriate.

03

Scattered evidence generation

Performance claims may remain undefined, leading to unfocused study planning.

Multidisciplinary participation

The radial chart shows participating expertise only. RACI roles and contributions are shown in the table.

Multidisciplinary participation graph
Accountable Responsible Consulted Informed

Role by expertise

RoleExpertiseContribution
AClinical Development

Owns the disease selection and clinical development rationale.

RNonclinical Development

Connects mechanism, efficacy, safety, and disease model evidence to the development logic.

CCMC / Formulation

Advises whether formulation, modality, and route fit the product value.

CDisease Expert Network

Advises treatment gaps and patient unmet needs in clinical practice.

CRegulatory Strategy

Assesses whether the rationale can be explained to regulators.

CBusiness Development

Advises value logic from investor and license-out perspectives.

IClinical Pharmacology /
MIDD

Identifies points that may require quantitative evidence later.

When clients need this service

Client situationAIMS value
The asset exists but the disease selection rationale is hard
to explain externally.Structures the development rationale linking unmet needs and candidate
evidence.
The value against current therapies or competing pipelines is unclear.Analyzes limitations of standard care and competitive assets.
The formulation, modality, route, or combination strategy needs to be
linked to the rationale.Clarifies why the selected development approach is appropriate.
Investor or partnering discussions require a concise pipeline rationale.Develops an externally usable value proposition.

Workflow

01

Define disease context

Summarize treatment gaps
and unmet needs.

02

Analyze current care

Review standard of care
and competitor limitations.

03

Connect pipeline evidence

Link mechanism, efficacy,
safety, and modality
evidence.

04

Formulate the rationale

Translate the logic
into language for external
stakeholders.

Representative deliverables

Development rationale report

A document linking disease selection, unmet needs, current treatment gaps, and pipeline evidence.

Pipeline value proposition summary

A concise message for investors and partners.

Development issue map

Key points by FiC, BiC, reformulation, combination, or other development approach.

Evidence-to-be-generated list

Performance claims that should feed the evidence generation roadmap.

Relevant standards by service

AIMS defines the standards and guideline anchors that should be considered for each service, rather than treating them as optional references.

ServiceRelevant standardsHow they are applied
Disease and Pipeline RationaleICH E8(R1), E6(R3)Align clinical development purpose, quality-by-design thinking, and stakeholder communication.
Entry Indication and Target SegmentICH E8(R1), E17, E5(R1)Support population, endpoint, and global development considerations.
Positioning and DifferentiationICH E8(R1), E9(R1), E17Link clinical objectives, estimands/endpoints, and global strategy to the TPP.

Core terms

Medical unmet needs

Disease burden or treatment gaps not adequately addressed by current care.

Entry
indication

The first indication in which the pipeline is developed to demonstrate value.

TPP

A target product profile describing intended indication, population, efficacy, safety, dosing, and differentiation.

Value
proposition

A concise rationale explaining why the asset matters to patients, clinicians, investors, or partners.