Case Studies:  Business Development



Payer Strategy/
Reimbusement Planning



Portfolio Planning/
Indication Scans

1. Case Study

Demand modeling for a novel RNA technology in a mass market: using conjoint to inform partnership discussions

The Problem

  • A small, privately held clinical stage biotech sought to quantify the dynamics at play in a mass market cardiovascular indication in the US, with an emphasis on a recently launched franchise that they would be competing against with their RNA technology

  • Their goal was to inform a launch forecast, clinical development decisions, and help in partnership negotiations

What We Did

  • Designed and executed an Internet survey with academic and community cardiologists in the US
  • Used conjoint to create a user-friendly “what-if” scenario modeling tool to forecast scenarios across various patient and physician segments in 2 and 3 and 4 product markets
  • Uncovered the dynamics behind a key relationship between 2 product attributes that had previously been hidden, leading to a re-thinking of clinical development

Our Results and Insights

  • Identified key attributes at play; structured conjoint scheme to run parallel studies spanning 3 patient types
  • Designed a concise conjoint grid spanning attributes and levels of highest competitive importance
  • Modeled a series of 2, 3, and 4 product markets across various patient types, uncovering unexpected pockets of opportunity
  • Conducted sensitivity analyses to uncover hidden relationships between product attributes across segments
  • Integrated sensitivity analyses across patient segments in visually compelling ways
  • Synthesized data into a series of actionable insights; uncovered the dynamics behind a key relationship between 2 attributes that had previously been hidden, leading to a re-thinking of clinical development

2. Case Study

Commercial diligence for licensing: Global forecast modeling for a phase 3 asset in an orphan pediatric CNS disease

The Problem

  • A mid-sized public biotech company was conducting due diligence on a potential in-licensing asset and commissioned a global research study to understand the demand for a key late stage asset with 2 launch indications
  • In addition, they wanted a global launch forecast and valuation tool for scenario planning

What We Did

  • Qualitative primary research with KOL physicians and dietitians in 8 countries involved in 2 rare pediatric neurological conditions
  • Built a cohort-level demand forecast with customizable assumptions (spanning awareness, diagnosis rate, compliance, persistence, and share) by year and by country
  • Client used this NPV forecast as the basis for their final negotiations

Our Results and Insights

  • Mapping the patient segments laid the groundwork for defining the opportunity
  • Patient lifecycle is complex; demand was a function of patient age and severity
  • Given the familiarity with the mechanism of action, respondents identified a number of patient segments where Product X would be a natural fit
  • Country differences regarding Product X were stark between the US/ Europe and South America
  • Using the qualitative research, a country-level forecast and NPV tool was built with customizable assumptions for scenario planning
  • The NPV output reflected different uptake rates based on launch timing and geography

3. Case Study

Determining global prevalence of a niche market with poor epidemiological understanding

The Problem

  • A mid-sized pharmaceutical client was in a quandary: they had a compelling asset with a well-defined clinical target, but had only a very rough estimate of the market size due to poor epidemiology coverage in the medical literature
  • To advance partnership discussions, they sought to estimate the global genetic and diagnosed prevalence of this rare condition using an efficient, cost-effective custom methodology

What We Did

  • Conducted a 10-minute survey among n=500 “all-comers” across 3 specialties and 4 countries; supplemented this with rigorous secondary research
  • Quantitative techniques were used to extrapolate prevalence to real-world while stratifying for patient age and severity and eliminating duplicate patients who are comanaged by multiple physicians
  • Aquest arrived at an accurate figure for global diagnosed prevalence that allowed the client to adequately forecast and advance partnership discussions

Our Results and Insights

  • Survey provided a quantitative understanding of the patient journey which allowed us to correct for double counting at the country-level
  • Using a battery of diagnostic questions we were able to estimate both the genetic prevalence and the diagnosed prevalence to high accuracy
  • We concluded with high precision (and by geography) that doctors treat 8 unique patients in a 3-month period
  • A final summary of prevalence by patient segment and by country was obtained, allowing the client to adequately forecast and advance partnership discussions

4. Case Study

Preparing for a partnership: Clinical development strategy and forecasting for novel prostate cancer molecule

The Problem

  • A privately-held, development stage pharmaceutical company needed a strategic partner and, in preparation for this, wanted to clarify the current and future treatment landscape of prostate cancer, and forecast revenue across several potential indications for its novel asset
  • The disease state is competitive, with multiple other products expanding their indications and targeted therapies expected to become more prominent

What We Did

  • Reviewed the current standards of care and identified treatment gaps and clinical development opportunities for potential indications alone or as a combination therapy
    Based on these potential indications, we identified patient segments and potential market sizing based on approvals.
  • Using this, we forecast a range of peak year revenue based on variable clinical trial success and patient uptake

Our Results and Insights

  • Mapped the expected future treatment landscape, showing both line extensions within the same class and the emergence of targeted therapies
  • Identified potential market size based on targeted indications
  • Used completed and ongoing clinical trials in the therapeutic area as an analogue for expected clinical trial design
  • Forecast peak year revenue based on variable clinical trial success and uptake across the targeted indications

5. Case Study

Ex-US opportunity assessment

View the Case

A mid-sized pharmaceutical company decided to take a fresh look at potential agents to in-license. Their question was simple yet challenging: are there any attractive opportunities among compounds currently marketed in ex-US territories that have never been reviewed by the FDA? Aquest built a sophisticated data gathering and screening tool (keying off such value drivers as strength of clinical evidence, therapeutic area attractiveness, commercial potential, regulatory pathway risk, and IP status). A process that started with over 10,000 compounds resulted in a prioritized list of 10 promising agents, several of which were selected for more intensive diligence.

6. Case Study

Drug delivery technology assessment

View the Case

A drug delivery company with an innovative technology platform sought assistance in identifying new and commercially promising small molecules and proteins to integrate with their technology. Aquest worked closely with an internal client team to develop a screening methodology based on several key determinants of value (technical feasibility, clinical development cost and timelines, regulatory risk, commercial potential, loss of exclusivity, and partnerability) and generated a prioritized list of candidates the client used to defend their investment decisions at the next board meeting.