Mining the Pipeline: Leading Investigational Products and Areas of Unmet Need

Stephanie Hawthorne, PhD
Published: Wednesday, Aug 07, 2013

Figure 1. Number of Trials Each Agent
is Participating In

table showing pivotal ongoing trials

Source: Kantar Health

Oncology continues to be a significant focus of development in the pharmaceutical market. The field is crowded, despite the fact that the agents in development target a broad range of tumor types. Kantar Health has profiled 69 pipeline agents that are in pivotal clinical development, in addition to the vast number of pivotal trials ongoing as part of lifecycle management (LCM) for launched oncology products. Here, we take a sneak peek focused on some of the late-stage drugs in development in the oncology pipeline.

In our analysis, over one-half of oncology pipeline agents possess novel mechanisms of action (MOA) and are vying to be first-in-class. Perhaps not surprisingly, the vast majority of these agents currently seeking first market entry are only being evaluated in one pivotal study (Figure 1). One-quarter of these pipeline agents are, however, being studied in multiple phase III trials (predominantly targeting multiple tumor types), with these multiple trials initiated before definitively established activity in patients.

Figure 2. Agents in Pivotal Trials,
by Market Size

table showing pivotal ongoing trials by market size

Source: Kantar Health

It’s worthwhile to note that these pipeline compounds are nearly equally being developed by “Big Pharma” and “Small Biotechs,” with only a handful in development by Midsize/ Emerging Pharma or as a collaborative effort. However, pipeline agents with multiple pivotal trials ongoing are heavily weighted toward those in development by Big Pharma or as part of a collaborative effort. Conversely, those agents with a single pivotal trial ongoing, trend toward those in development by smaller biotechs; this potentially reflects limited resources for R&D, especially considering that the mechanisms of action of two-thirds of these agents have the potential to support development in multiple indications.

Interestingly, the majority of the indications in which these 69 agents are being developed target rather small indications in terms of patient population size. As Figure 2 shows, 60% of the ongoing pivotal trials are being conducted in indications with a target population of less than 12,500 patients.1 This is in contrast to a decade ago, when most agents in pivotal development were targeting the largest patient populations, such as breast cancer, non-small cell lung cancer (NSCLC), and colorectal cancer (CRC).

Figure 3. Agents in Pivotal Trials,
by Unmet Need

table representing ongoing pivotal trials by unmet need

Source: Kantar Health

The level of unmet needs was calculated based on the number of existing treatment options in the target indication (with fewer treatment options correlating with higher need), the rate of treatment (with high non-treatment rates correlating with higher need), and efficacy outcomes achieved with current options (with short survival times correlating with higher need). High overall scores for the sum of these three attributes were considered to be a high unmet need, whereas low overall scores were considered to be a low unmet need.

Some of the smaller indications represent tumors in which the incidence of the disease overall is rather small, such as acute lymphocytic leukemia (ALL) and soft tissue sarcoma, whereas others represent biomarker subsets of larger tumor types, such as Met+ NSCLC or NRAS mutant melanoma. However, despite the oncology community’s growing focus on personalized medicine, only 22% of pipeline agents currently in pivotal trials are being developed in a biomarker-defined patient population. Two-thirds of ongoing pivotal studies with pipeline agents are being conducted in indications with relatively low unmet need (Figure 3).

Of course, all cancer indications have rather high unmet need compared to non-oncology indications due to the life-threatening nature of the disease; however, among the numerous cancer indications, the level of need may vary considerably.

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