Developing a strategic approach to indication expansion assessments

Challenge 

Our client, a leading pharmaceutical company, was in the midst of a clinical drug development for a rare disease indication. When evaluating scientific literature, they noticed that the drug might also have the potential to be effective for the treatment of other medical conditions including rare, specialty and broad indications.   

This discovery presented an opportunity to create a product lifecycle management strategy and overcome industry obstacles such as the so-called “patent cliff” – the loss of drug exclusivity and the associated sharp decline in revenues upon the expiry of the patent. At the same time, the broad spectrum of indications demanded a close assessment of the value that the drug might add both from patient and commercial perspectives.   

Proactive planning and cross-functional collaboration are imperative to create an indication expansion strategy that drives both growth and earnings in the long-term. Therefore, our client’s R&D, commercial and medical teams had to fully understand the potential impact of this promising drug and adjust their development strategy to address areas where patients’ unmet needs and burden of disease are highest without sacrificing the commercial viability and attractiveness.  

The following key questions needed to be addressed for finding an optimal and sustainable solution:  

• How can we identify indication expansion possibilities? 
• What are they key opportunities and risks associated with each one of them? 
• How can we define a roadmap for the successful expansion strategy for this drug?  

Solution 

In order to help our client answer to these key questions, we opted for a four-step-approach.  

Strategic guiding principles 

It was very important to our client that the expansion strategy was based on the company’s core framework consisting of values and principles that put the patient first. For the assessment of expansion opportunities, we therefore defined a set of strategic guiding principles that were aligned with those core values. 

Assessment framework definition 

For the development of an assessment framework, our goal was to integrate the commercial and patient perspectives with scientific and clinical rational. We therefore integrated these three dimensions and corresponding metrics into the framework to help analyse the opportunities and risks of the different expansion possibilities – qualitatively and quantitatively. 

• Patient value: Burden of disease, unmet needs 
• Commercial value: Revenue potential, strategic value, competitive landscape, market access dynamics 
• Scientific fit: Clinical confidence, compound fit 

These dimensions were crucial to fostering understanding and alignment between the different internal stakeholders on risks and opportunities. Accordingly, they were an important tool for our client when making trade-off decisions, while at the same time ensuring that these decisions were in line with the company’s values.  

Expansion opportunity assessment 

Working within the framework, different expansion opportunities were screened and assessed. A series of agile workshops with internal stakeholders such as R&D, medical and commercial teams provided valuable insights for both qualitative and quantitative assessment.  

Additionally, together with the client we gathered market insights from key opinion leaders in the different disease areas. These interviews helped gain a better understanding of the unmet needs and burden of patients and health practitioners.   

Assumptions used for the quantification of the expansion opportunities were informed through interviews with internal specialists, external key opinion leaders as well as secondary research. By modelling business impact and financial projections, we assisted our client in gaining a better understanding about the commercial value and feasibility of the different expansion scenarios.   

Prioritisation of indication expansion opportunities and strategic roadmap definition 

In order to effectively harness the insights obtained in the interviews, through our quantitative modelling as well as information gained through the opportunity and risk assessment within the new framework, we developed three categories from high to low commercial priority according to which the different indications could be clustered. Based on this mapping of the expansion opportunities we formulated different strategic scenarios. They included important considerations regarding patient value, price differential, product differentiation, timing of expansion, regulatory aspects and the minimisation of cannibalisation.  

Result

The assessment and the strategic roadmap with its framework enabled our client to gain a clearer understanding on how to prioritise the different indication expansion opportunities and how to further explore them. Moreover, with the help of the guiding principles and the three dimensions patient value, commercial value and scientific fit the pharmaceutical company also had an important tool at hand for patient-centric ethical decision-making. 

The information gathered and strategic insights gained throughout the assessment process allowed the company to develop a strategy to maximise the value of their asset while ensuring long-term success of the drug for the rare medical condition and beyond. For this, we also provided our client with guidance on where to focus their efforts in the coming months to further explore the opportunities from a clinical development standpoint.