Value of Disease Leadership for Biopharma Companies in Today's Environment

Biopharma companies that contemplate whether to strive for disease leadership often question whether it’s imperative for long-term success. It is a well-known fact that disease leadership, like therapeutic area leadership, offers significant direct and indirect benefits. The direct benefits range from delivering better solutions for patients to maximizing portfolio potential, while indirect benefits range from engaging research community to gaining better access to solutions. The decision to establish leadership in any disease is complex as it requires long-term commitment.


What does it take to establish disease leadership?

Though it’s difficult to be prescriptive in terms of what works and what doesn’t in each disease area, the foundational building blocks of what is needed to establish a disease platform is well understood. It starts with a fundamental question of “What’s the nature of the disease and is this an area of interest for the company?”. Significant unmet needs continue to exist in many diseases including chronic conditions where cures are expected given the rapid developments in science and technology. The first step is to gain a deeper understanding of the unmet needs of patients in the disease area of interest and see whether the company has the wherewithal to address these needs.

Based on extensive consulting work that we have done in this area for pharma and biotech companies, we have concluded that there are four core components of disease strategy that must be addressed to lay the groundwork for establishing a platform for leadership. 

  1. Building the Scientific Platform:  With the rapid pace of innovation, researchers are finding breakthroughs in fundamental science to gain a deeper understanding of the pathophysiology of diseases and how best to target these diseases. In order to stay ahead of the curve, it is imperative that companies build a robust scientific platform (translational research, precision medicines) via collaborative networks (academic and research institutions) to harness information and to develop new technologies (diagnostics / biomarkers / delivery systems / drugs).  Many successful biopharma companies have built R&D ecosystems with internal and external collaborations to find solutions for complex diseases.  

  2. Strengthening the Portfolio: Most companies start with one asset in the chosen disease area and look for life cycle opportunities to expand the potential for this asset.  Relying on the success of any one drug is always risky as the franchise may not be sustainable after patent expiry.  It is imperative to build a portfolio of novel best-in-class products that are highly differentiated to make the franchise sustainable in the long run which in turn allows companies to make strategic investments to target diseases with the highest unmet needs.  

  3. Developing Personalized Solutions: Though pharmacological intervention is key in most cases, it is equally important to focus on comprehensive personalized solutions to deliver quality (clinical and health economic) outcomes.  This approach often requires creative thinking to develop solutions to address areas of the disease continuum (diagnosis, treatment, and treatment monitoring). In fact, it becomes an obligation for companies to show commitment in developing these comprehensive solutions to attain leadership.

  4. Ensuring Market Access to Solutions:  Engaging policy makers and advocacy groups lays the ground work for seeking optimal market access and is key for long term success.  In this increasingly challenging healthcare environment, Biopharma companies have to articulate the value of these solutions (cost-effectiveness) to payers to get reimbursement. The HEOR platforms are key to develop data supporting the value story and to gain alignment with payors and health authorities. In many rare diseases, the treatment guidelines are often unclear or don’t exist in which case it becomes important to work with the physician community to develop these guidelines and work with payers / policy makers to adopt them.

Why is it so challenging to attain disease leadership?

There are numerous challenges that companies must overcome as they try to attain disease leadership. First, companies must secure the resources to execute a strategy with a long-term investment mindset as pressure from short-term investors often outweigh long-range objectives.  Second, building the required R&D competencies to lay the proper foundation often requires early success with at least one anchor product, the absence of which makes it difficult for management to stay focused on long-term plans. Third, leadership must foster a patient-centric culture to instill empathy and social responsibility to motivate stakeholders. 

In order to be successful, Biopharma executives must continuously assess tradeoffs associated with key investment decisions while ensuring appropriate risk management mechanisms to avoid common pitfalls.  Disease leadership is an aspiration for most companies and we think that it’s an important goal to have to make a meaningful impact on patients’ lives and to create long-term value for stakeholders.



Author, Subbarao Jayanthi is Managing Partner. 


RxC International is a premier life sciences management consulting firm. RxC collaborates with clients to identify and develop growth opportunities. The firm leverages consulting partners and advisers to combine strategic and operational expertise to bring multiple perspectives to every engagement. The firm has deep expertise in corporate strategy, new product strategy, and commercial excellence.

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