The clinical value of drugs under development in hematological malignancies 53 3♣ Results Twenty-eight agents were steered towards phase III, after three phase II trials on average, across a variety of different potential targets (Figure 2). In this Figure, one also sees the multiplicity of phase II trials prior to progressing to phase III research. This underlines the uncertainty inherent to early-stage clinical development, in which the exact dosage, setting, and patient populations that might benefit from investigational drugs still has to be determined. Eight targeted drugs were FDA approved. Of these, none had been proven to improve quality of life at time of approval. Systematic evaluation through the ESMO-MCBS v1.1 indicated that two out of eight drugs provide substantial benefit, but three out of eight approved products had not established a clinically relevant gain in overall survival at the time of approval (see Table 2). Ongoing development of targeted agents and application in combination with chemotherapy raise new perspective for patients with AML, and provide them with some survival benefit. However, objectively, only 16.8% of 167 targeted agents (potentially provided to patients through expanded access) moved to phase III and of the eight FDA approved drugs, a minority provides a substantial clinical benefit. Thus, returns on targeted therapy research remain lean for AML patients, and so potentially does expanded access to these drugs. Accessing drugs in early stages of development, whether it is through trial participation or through expanded access pathways, usually simply equates to accessing ineffective therapies.
RkJQdWJsaXNoZXIy MTk4NDMw