Impact of Precision Medicine on Efficiencies of Novel Drug Development in Cancer

Abstract

Precision medicine (PM) offers opportunities for reducing the costs, burdens, and time associated with drug development. We examined time, number of trials, indications tested, and patient burden needed to achieve first U.S. Food and Drug Administration license for all five novel anticancer PM drugs and all 10 novel non-PM drugs receiving U.S. Food and Drug Administration approval during 2010–2014. The 15 drug portfolios encompassed 242 trials: 87 for PM drugs and 155 for non-PM drugs. Embase and MEDLINE databases were searched for all prelicensure clinical trials, and data on time, patient numbers, indications tested, and total treatment-emergent grade 3–5 adverse events were measured from the first trial of each drug. We did not find patterns suggesting greater efficiencies in PM compared with non-PM. Gains in efficiency for PM drug development may be offset by challenges with recruitment.

Patient Participation in Clinical Trials of Oncology Drugs and Biologics Preceding Approval by the US Food and Drug Administration

“…It differs from prior studies that confined their assessment to successful drug development efforts. 7 , 8 We estimated that 12 217 patients were required to advance a new oncology drug from initial test of efficacy to regulatory licensure. However, many drugs that gain licensure have limited clinical impact.…”

“…Previous studies of patient enrollment have shown that a median of 1708 patients participated in clinical trials per European Medicines Agency new molecular entity approval 7 ; a median of 2316 patients were studied per US Food and Drug Administration (FDA) approval of a precision medicine oncology drug. 8 However, these studies restricted their analyses to approved drugs and thus do not account for the large numbers of patients participating in unsuccessful drug development efforts. Estimates that include all patient study participants can make more visible the degree to which private drug development efforts build on public volunteerism.…”

“…Drug development also requires that physicians and patients invest substantial cognitive resources in explaining and understanding study protocols. Previous studies of patient enrollment have shown that a median of 1708 patients participated in clinical trials per European Medicines Agency new molecular entity approval; a median of 2316 patients were studied per US Food and Drug Administration (FDA) approval of a precision medicine oncology drug . However, these studies restricted their analyses to approved drugs and thus do not account for the large numbers of patients participating in unsuccessful drug development efforts.…”

“…Patient Participation in Clinical Trials of Oncology Drugs and Biologics Before FDA Approval trials was 158 810; 47 913 (30.2%) were enrolled in trials testing drugs that gained FDA approval, and 110 897 (69.8%) were enrolled in trials that did not. The median (IQR) patient enrollment per drug was 389 (152-1402) patients, and the median (IQR) number of trials per drug was 6(3)(4)(5)(6)(7)(8)(9)(10)(11)(12)(13)(14). For drugs that achieved regulatory approval, the median (IQR) number of patients was 3776 (1582-5779); for those that did not achieve approval, it was 328 (131-937) (eTable 3 in the Supplement).…”

Health Care Resource Use at End of Life in Patients with Advanced Lung Cancer

“…Cancer treatment costs account for a substantial part of health care expenditure [1]. The costs have increased due to advancements in novel drugs and therapy innovations, such as genomic testing, in the last decade [2]. It is difficult to compare results from different countries directly as the absolute cost of cancer treatment is influenced by each country's socioeconomic environment, health care system and income level [3].…”