Chapter 3 50 WEIGHING POSITIVE PHASE II RESULTS FOR EXPANDED ACCESS Novel agents are often lauded as ‘life-changing’ or ‘breakthrough’ when showcased at scientific conferences.1,2 These events often serve as the initial introduction of new drugs to physicians, and the positive portrayal in posters or abstracts may later lead to potential requests for expanded access. We wondered whether abstracts presenting ‘positive’ outcomes at the major global hematology conference, the meetings of the American Society of Hematology (ASH) of phase II drugs eventually were associated with the initiation of a phase III investigations and potentially subsequent drug approvals. For this research, we tabulated all abstracts on hematological malignancies of three consecutive years of ASH meetings, 2013-2015. Abstracts were categorized as ‘positive’, ‘negative’, or ‘inconclusive’ (Table 1). Positive abstracts featured terms such as ‘encouraging’, ‘promising’, ‘could represent a novel therapeutic option’ and ‘warrants investigation in a randomized trial’. ‘Negative’ abstracts included terms such as ‘does not support further research’ and ‘demonstrates no clinical activity’. The remainder were scored as inconclusive. We also assessed the reliability of ‘positive’ abstracts by determining if the results were published in peer-reviewed journals with the ‘positive’ outlook maintained, and if a phase III trial was initiated with the described drug. We found that the large majority of presented abstracts were ‘positive’ (76%), and that results from both ‘positive’ and ‘negative’ abstracts were published at a similar rate in peer reviewed journals (83% and 86% respectively). And although results are initially presented as promising, only 47% of regimens described in ‘positive’ abstracts progressed to phase III. Table 1: Research characteristics according to abstract conclusion as presented at ASH 2013 to 2015 OVERALL INCONCLUSIVE NEGATIVE POSITIVE YEAR 2013 124 20 (16%)* 11 (9%) 93 (75%) 2014 128 16 (13%) 13 (10%) 99 (77%) 2015 116 3 (3%) 13 (11%) 100 (86%) SAMPLE SIZE 41 (25-63)† 41 (26-69) 29 (21-53) 41 (25-63) PUBLISHED 302 (82%)‡ 28 (72%) 32 (86%) 242 (83%) PHASE III 161 (44%) 13 (33%) 11 (30%) 137 (47%) TOTAL 368 39 37 292 *N (percent per year). †Median (interquartile range). ‡N (percent per abstract conclusion).
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