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Oncology Sub-Indication Phase Transition Success Rates and LOA Oncology drugs were further categorized into two main types of cancer: solid tumors and hematological cancers. Solid tumors had twice as many transitions in the data set (2,283 vs. 805), but only half the LOA from Phase I vs. hematological cancers (4.0% vs. 8.1%). These are shown in Figure 7 in more detail. Phase Success Advanced or Suspended Phase Success Advanced or Suspended Phase Success Advanced or Suspended Phase Success Advanced or Suspended Phase Success Oncology 1222 62.8% 1416 24.6% 349 40.1% 176 82.4% Solid 860 64.1% 1055 23.0% 260 34.2% 108 79.6% Hematologic 327 61.8% 341 28.7% 78 52.6% 59 86.4% LOA n Likelihood of Approval Phase LOA LOA n Phase LOA LOA n Phase LOA LOA n Phase LOA Oncology 3163 5.1% 1941 8.1% 525 33.0% 176 82.4% Solid 2283 4.0% 1423 6.3% 368 27.3% 108 79.6% Hematologic 805 8.1% 478 13.1% 137 45.4% 59 86.4% Phase I to Phase II Phase II to Phase III Phase III to NDA/BLA NDA/BLA to Approval Phase I to Approval Phase II to Approval Phase III to Approval NDA/BLA to Approval 0% 10% 20% 30% 40% 50% 60% 70% Phase III Success Rate Oncology Indications Hematologic Cancers CLL/SLL – NHL Multiple Myeloma Indolent NHL AML Solid Tumors Renal Cell Cancer Breast Cancer Melanoma Prostate Cancer Colorectal Cancer NSCLC Ovarian Cancer Gastric Cancer Pancreatic Cancer Figure 7. Phase transition success rates and LOA for Oncology indications with corresponding n values. ‘Advanced or Suspended’ refers to the total number of transitions used to calculate each success rate, with the n value noted in the text. The LOA n value is the total ‘Advanced or Suspended’ transitions of all phases used to calculate LOA. ‘Phase Success’ is the probability of successfully advancing to the next phase, whereas ‘Phase LOA’ is the probability of FDA approval for drugs in this phase of development. 14 | BIO Industry Analysis Phase II transition success rates by sub-indication tended to range close to the overall 25% Oncology calculation for Phase II, +/-10%. Narrowing in on Phase III transition success rates, only 34.2% of the 260 drug programs in solid tumor cancers were deemed sufficiently successful to file an NDA/BLA with the FDA. This was the underlying cause for the 2x difference we see in overall LOA, as the hematological cancer programs recorded a 52.6% success rate in Phase III. Since Phase III was identified as the weakest phase for Oncology, Phase III transition success rates for a number of major oncology sub-indications were included in Figure 7. The solid tumor Phase III transition success rate (34.2%, n=260) ended up as the lowest for any of the major disease categories studied. Solid tumor drugs for pancreatic cancer seemed to have the toughest challenges in Phase III studies (13.3%, n=15). However, Phase III success rates for Ovarian and Gastric cancers also fell below 30%.