Drug development continues to concentrate on a small set of well-known targets, creating a crowded landscape where many companies pursue similar pathways. While research into novel mechanisms continues, familiar targets often take priority, reflecting a cautious approach to scientific risk. This focus on the known shapes the industry’s strategies and sets the stage for a growing need to explore underutilised opportunities that could drive meaningful innovation.
The current landscape illustrates a clear tension where companies are striving to minimise early-stage risk, yet this cautious approach may limit the potential for truly differentiated therapies.
Fewer New Targets Entering Development
Despite the growth in research pipelines and early-stage investment, the introduction of new biological targets has slowed noticeably. This trend should be interpreted with some caution, as companies are increasingly choosing not to disclose targets early in development in order to protect competitive advantage, which may partly explain the apparent decline. What is clear and well-evidenced, however, is that crowding around a limited set of mechanisms has grown substantially. Moreover, despite a near-doubling of the overall R&D pipeline and consistent growth in early-stage venture capital investment, the number of novel targets entering the pipeline each year has fallen sharply – from around 100 pre-pandemic to approximately 30 in 2024.
The focus on predictable opportunities over fewer familiar mechanisms suggests that the industry has prioritised established mechanisms, reflecting a cautious approach to managing early-stage risk. This cautious stance has implications beyond the early stages of development. By concentrating on a narrow set of targets, companies risk crowding their pipelines and limit the variety of mechanisms available for exploration. Over time, this approach could make it more challenging to address unmet patient needs and reduce the likelihood of developing first-in-class therapies that offer significant clinical differentiation. This reinforces the importance of balancing early-stage risk with opportunities for greater therapeutic differentiation.
Balancing Risk and Reward in Target Selection
Choosing the right therapeutic target remains one of the most critical decisions in drug development. Companies must weigh the potential benefits of established targets against the promise and uncertainty of novel ones. Well-known targets often present lower early-stage risk due to existing knowledge and clinical familiarity, but they are frequently associated with crowded markets and limited differentiation. As a result, new therapies can struggle to stand out both clinically and commercially, even when the underlying science is strong.
In contrast, novel targets hold greater scientific uncertainty, but they also offer the potential for meaningful innovation and first-to-market advantage. Pursuing these targets can differentiate a therapy, create new treatment paradigms, and potentially unlock significant clinical value. In recent years, however, the industry appears to have shifted toward established pathways, prioritising lower-risk strategies over exploratory approaches. This preference has contributed to crowded development areas and a relative underinvestment in new mechanisms, potentially slowing the pace of breakthrough innovation.
Advancing Innovation Within Known Pathways
Even within familiar targets, innovation continues through multiple strategies. Companies may modify how a target is engaged, explore new therapeutic modalities or delivery methods, apply precision medicine techniques, or expand into additional disease areas. These approaches can improve efficacy, enhance patient outcomes, and extend the lifecycle of existing therapies.
However, focusing innovation within known pathways also reinforces pipeline concentration. The majority of resources remain directed toward a limited set of biological mechanisms, leaving other promising opportunities relatively untouched. This imbalance highlights the importance of strategically allocating resources to ensure that novel targets receive attention alongside refinement of established pathways. Without such a balance, the industry risks inefficiencies and may struggle to deliver therapies that offer meaningful differentiation.
The Need for a More Balanced Approach
The current state of biopharma research points to an imbalance between managing risk and pursuing innovation. While established targets remain important, overreliance on them may limit the industry’s ability to explore high-potential mechanisms. At the same time, a substantial portion of biological targets remains underexplored, representing a source of untapped opportunity.
Emerging technologies, including artificial intelligence and in silico experimentation, provide new ways to explore novel targets more efficiently and with lower risk. Alongside these tools, strategic collaboration among companies, research institutions, and emerging biotechs can further reduce uncertainty in early exploration and help accelerate progress.
By combining these approaches and rebalancing investment priorities to include both established and novel targets, the industry can unlock new opportunities for clinical impact, strengthen its pipelines, and position itself to deliver the next generation of transformative therapies.
Author Bios
Anne Dhulesia
Anne Dhulesia is a Partner in L.E.K. Consulting’s London office and a member of the European Life Sciences practice. Anne advises corporate clients on a wide range of assignments in the pharmaceuticals sector, including radiopharmaceuticals: market assessments, business plan development and definition of long-term growth strategies.
Ananth Srinivasan
Ananth Srinivasan is a Senior Consultant in L.E.K. Consulting’s New York office and a member of the firm’s Life Sciences Biopharma practice. Ananth supports clients across early R&D innovation, commercial opportunity assessments and portfolio strategy
Matt Mancuso
Matt Mancuso is a Managing Director and Partner in L.E.K. Consulting’s Boston office and a member of the Life Sciences practice. Matt has experience in oncology and nononcology opportunity assessment and target identification, including leveraging commercial, scientific and advanced bio- and chemo-informatic analysis.
Pierre Jacquet, M.D., Ph.D.
Pierre Jacquet, M.D., Ph.D., is a Managing Director and Vice Chairman of L.E.K. Consulting’s Global Healthcare practice. Based in Boston, Pierre has more than 20 years of experience in corporate and business unit strategy consulting and in M&A advisory services.
Ricardo Brau
Ricardo Brau is a Managing Director and Partner in L.E.K. Consulting’s Boston office. Ricardo leads the firm’s Life Sciences Biopharma practice and has experience across most therapeutic areas and industry segments, in both large and emerging biopharma companies.
