Patient-Centered Cancer Drug Development: Clinical Trials, Regulatory Approval, and Value Assessment

2019 ◽  
pp. 374-387 ◽  
Author(s):  
Bishal Gyawali ◽  
Thomas J. Hwang ◽  
Kerstin Noelle Vokinger ◽  
Christopher M. Booth ◽  
Eitan Amir ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Drug Development ◽  
Cancer Biology ◽  
Drug Approval ◽  
The United States ◽  
Rapid Expansion ◽  
Cancer Drug ◽  
Trial Participation ◽  
Value Assessment ◽  
The Impact

Historically, patient experience, including symptomatic toxicities, physical function, and disease-related symptoms during treatment or their perspectives on clinical trials, has played a secondary role in cancer drug development. Regulatory criteria for drug approval require that drugs are safe and effective, and almost all drug approvals have been based only on efficacy endpoints rather than on quality-of-life (QoL) assessments. In contrast to Europe, information regarding the impact of drugs on patients’ QoL is rarely included in oncology drug labeling in the United States. Until recently, patient input and preferences have not been incorporated into the design and conduct of clinical trials. In recent years, a more in-depth understanding of cancer biology, as well as regulatory changes focused on expediting cancer drug development and approval, has allowed earlier access to novel therapeutic agents. Understanding the implications of these expedited programs is important for oncologists and patients, given the rapid expansion of these programs. In this article, we provide an overview of the role of QoL in the regulatory drug–approval process, key issues regarding trial participation from the patient perspective, and the implications of key expedited approval programs that are increasingly being used by regulatory bodies for cancer care.

Changes in numbers of randomized (RCT) versus non-randomized (NRCT) clinical trials from 2004-2016: Evidence of shifting cancer drug development pathway.

2017 ◽  
Vol 35 (15_suppl) ◽  
pp. 2543-2543
Author(s):  
Laura Vidal Boixader ◽  
Kelly Kevelin Curtis ◽  
Jim Wahl ◽  
Nicholas Kenny ◽  
Keren Rachel Moss
Keyword(s):  
Clinical Trials ◽  
Drug Development ◽  
Cancer Biology ◽  
Drug Approval ◽  
Cancer Drug ◽  
Phase 2 ◽  
The Past ◽  
Anti Cancer ◽  
High Antitumor Activity ◽  
Drug Approval Process

2543 Background: Trials using randomized designs have been conducted for decades to demonstrate efficacy of novel anti-cancer drugs (NACD). Recently, several NACD have shown high antitumor activity in early phase studies, prompting suggestions that NRCT could expedite drug development. We sought to determine what changes have occurred in numbers of NACD RCT vs NRCT conducted from 2004-2016. Methods: We reviewed a database of NACD clinical trials conducted by INC (excluding phase I and I/II trials) and classified them by RCT vs NRCT, grouped by year (≤ 2010 or > 2010). We queried Citeline Trialtrove database for industry sponsored, phase 2 trials (P2T) initiated from 2006-2016 and examined numbers of RCT vs NRCT by year.A more detailed analysis of non-small cell lung cancer (NSCLC) clinical trials based on drug type category - Immunooncology (IO) vs. all other mechanims of action (NIO) was performed. Results: 190 INC-conducted trials were reviewed. 58 trials (31%) were performed ≤ 2010 and 132 trials (69%) > 2010. Over this period, NRCT (n = 107, 56%) outnumbered RCT (n = 83, 44%). Whereas RCT outnumbered NRCT from 2004-2010 (74% vs 52%), after 2010, NRCT outnumbered RCT (58% vs 42%). Citeline Trialtrove search revealed 4776 industry sponsored P2T initiated from 2006-2016. The total number of P2T started annually was highest in 2007 (n = 621), decreasing to a low of 375 in 2016. The proportion of phase 2 RCT demonstrated an increase from 27% (n = 166) in 2006 to a peak plateau of 37-39% from 2011-2014, followed by a drop to 33% in 2015 and 29% in 2016. Among IO studies, RCT declined in 2015-6 vs. previous years, and a decreased for all NACD in 2016 vs. previous years also was noted. For studies in NSCLC, declines in RCT were evident from 2015-6 vs. previous years ( 45% in 2007-14 vs. 25% in 2015-6). Conclusions: Our data indicate a trend toward fewer trials of NACD using randomized designs and more studies using non-randomized designs, with overall fewer P2T initiated in the past year. This change reflects shifts in NACD development pathways, related to a better understanding of cancer biology, drive to develop personalized treatment and a more flexible regulatory drug approval process.

scholarly journals Clinical, Pathological, and Ethical Considerations for the Conduct of Clinical Trials in Dogs with Naturally Occurring Cancer: A Comparative Approach to Accelerate Translational Drug Development

ILAR Journal ◽  
2018 ◽  
Vol 59 (1) ◽  
pp. 99-110 ◽  
Author(s):  
Daniel Regan ◽  
Kelly Garcia ◽  
Douglas Thamm
Keyword(s):  
Clinical Trials ◽  
Drug Development ◽  
Translational Research ◽  
Cancer Biology ◽  
Comparative Approach ◽  
Cancer Drug ◽  
Comparative Oncology ◽  
Pet Dogs ◽  
Naturally Occurring

Abstract The role of comparative oncology in translational research is receiving increasing attention from drug developers and the greater biomedical research community. Pet dogs with spontaneous cancer are important and underutilized translational models, owing to dogs’ large size and relative outbreeding, combined with their high incidence of certain tumor histotypes with significant biological, genetic, and histological similarities to their human tumor counterparts. Dogs with spontaneous tumors naturally develop therapy resistance and spontaneous metastasis, all in the context of an intact immune system. These fundamental features of cancer biology are often lacking in induced or genetically engineered preclinical tumor models and likely contribute to their poor predictive value and the associated overall high failure rate in oncology drug development. Thus, the conduct of clinical trials in pet dogs with naturally occurring cancer represents a viable surrogate and valuable intermediary step that should be increasingly incorporated into the cancer drug discovery and development pipeline. The development of molecular-targeted therapies has resulted in an expanded role of the pathologist in human oncology trials, and similarly the expertise of veterinary pathologists will be increasingly valuable to all phases of comparative oncology trial design and conduct. In this review, we provide a framework of clinical, ethical, and pathology-focused considerations for the increasing integration of translational research investigations in dogs with spontaneous cancer as a means to accelerate clinical cancer discovery and drug development.

scholarly journals Surrogate endpoints for early-stage breast cancer: a review of the state of the art, controversies, and future prospects

2021 ◽  
Vol 13 ◽  
pp. 175883592110595
Author(s):  
María Gion ◽  
José Manuel Pérez-García ◽  
Antonio Llombart-Cussac ◽  
Miguel Sampayo-Cordero ◽  
Javier Cortés ◽  
...  
Keyword(s):  
Breast Cancer ◽  
Clinical Trials ◽  
Drug Development ◽  
Hormone Receptor ◽  
Early Stage ◽  
Drug Approval ◽  
Cancer Drug ◽  
Surrogate Endpoints ◽  
Long Term Outcomes

Drug approval for early-stage breast cancer (EBC) has been historically granted in the context of registration trials based on adequate outcomes such as disease-free survival and overall survival. Improvements in long-term outcomes have made it more difficult to demonstrate the clinical benefit of a new cancer drug in large, randomized, comparative clinical trials. Therefore, the use of surrogate endpoints rather than traditional measures allows for cancer drug trials to proceed with smaller sample sizes and shorter follow-up periods, which reduces drug development time. Among surrogate endpoints for breast cancer, the increase in pathological complete response (pCR) rates was considered appropriate for accelerated drug approval. The association between pCR and long-term outcomes was strongest in patients with aggressive tumor subtypes, such as triple-negative and human epidermal growth factor receptor 2 (HER2)-positive/hormone receptor-negative breast cancers. Whereas in hormone receptor-positive/HER2-negative EBC, the most accepted surrogate markers for endocrine therapy–based trials include changes in Ki67 and the preoperative endocrine prognostic index. Beyond the classic endpoints, further prognostic tools are required to provide EBC patients with individualized and effective therapies, and the neoadjuvant setting provides an excellent platform for drug development and biomarker discovery. Nowadays, the availability of multigene signatures is offering a standardized quantitative and reproducible tool to potentiate the efficacy of standard treatment for high-risk patients and develop de-escalated treatments for patients at lower risk of relapse. In this article, we first evaluate the surrogacies used for long-term outcomes and the underlying evidence supporting the use of each surrogate endpoint for the accelerated or regular drug approval process in EBC. Next, we provide an overview of the most recent studies and innovative strategies in a (neo)adjuvant setting as a platform to accelerate new drug approval. Finally, we highlight some clinical trials aimed at tailoring systemic treatment of EBC using prognosis-related factors or early biomarkers of drug sensitivity or resistance.

Enrollment disparities in cancer clinical trials leading to FDA approvals between 2008 and 2020.

2021 ◽  
Vol 39 (15_suppl) ◽  
pp. e18501-e18501
Author(s):  
Ryan Huu-Tuan Nguyen ◽  
Yomaira Silva ◽  
Vijayakrishna K. Gadi
Keyword(s):  
Prostate Cancer ◽  
United States ◽  
Clinical Trials ◽  
Drug Approval ◽  
The United States ◽  
Cancer Clinical Trials ◽  
Cancer Prevalence ◽  
Fda Approval ◽  
The Us ◽  
Enrollment Data

e18501 Background: Cancer clinical trials based in the United States (US) have lacked adequate representation of racial and ethnic minorities, the elderly, and women. Pivotal clinical trials leading to United States Food and Drug Administration (FDA) approval are often multi-national trials and may also lack generalizability to underrepresented populations in the United States. We determined the racial, ethnic, age, and sex enrollment in pivotal trials relative to the US cancer population. Methods: We reviewed the FDA’s Drug Approvals and Databases for novel and new use drug approvals for breast, colorectal, lung, and prostate cancer indications from 2008 through 2020. Drugs@FDA was searched for drug approval summaries and FDA labels to identify clinical trials used to justify clinical efficacy that led to FDA approval. For eligible trials, enrollment data were obtained from FDA approval summaries, FDA labels, ClinicalTrials.gov, and corresponding journal manuscripts. Enrollment Fraction (EF) was calculated as enrollment in identified clinical trials divided by 2017 SEER cancer prevalence. All data sources were publicly available. Results: From 2008 through 2020, 60 drugs received novel or new use drug approval for breast, colorectal, lung, or prostate cancer indications based on 66 clinical trials with a total enrollment of 36,830. North America accounted for 9,259 (31%) enrollees of the 73% of trials reporting location of enrollment. Racial demographics were reported in 78% of manuscripts, 66% of ClinicalTrials.gov pages, and 98% of FDA labels or approval summaries. Compared with a 0.4% enrollment fraction among White patients, lower enrollment fractions were noted in Hispanic (0.2%, odds ratio [OR] vs White, 0.46; 95% confidence interval [CI], 0.43 to 0.49, P< 0.001) and Black (0.1%, OR 0.29; 95% CI 0.28 to 0.31, P< 0.001) patients. Elderly patients (age ≥ 65 years) were less likely than younger patients to be enrollees (EF 0.3% vs 0.9%, OR 0.27; 95% CI 0.26 to 0.27, P< 0.001) despite accounting for 61.3% of cancer prevalence. For colorectal and lung cancer trials, females were less likely than males (EF 0.7% vs 1.1%, OR 0.66; 95% CI 0.63 to 0.68, P< 0.001) to be enrolled. Conclusions: Black, Hispanic, elderly, and female patients were less likely to enroll in cancer clinical trials leading to FDA approvals from 2008 to 2020. Race and geographic enrollment data were inconsistently reported in journal manuscripts and ClinicalTrials.gov. The lack of appropriate representation of specific patient populations in these key clinical trials limits their generalizability. Future efforts must be made to ensure equitable access, representation, and reporting of enrollees that adequately represent the US population of patients with cancer.

Patient care and clinical trials in gynecological oncology: Implications of the COVID-19 pandemic.

2021 ◽  
Vol 39 (15_suppl) ◽  
pp. 5564-5564
Author(s):  
Sara Nasser ◽  
Christina Fotopoulou ◽  
Murat Guktekin ◽  
Desislava Dimitrova ◽  
Philippe Morice ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Medical Care ◽  
Patient Care ◽  
Healthcare Professionals ◽  
Clinical Pathways ◽  
Trial Participation ◽  
Early Stage Disease ◽  
Gynecological Oncology ◽  
Cardiopulmonary Support ◽  
The Impact

5564 Background: This is a prospective international Survey to evaluate the impact of the COVID-19 Pandemic on the management of patients with gynecological malignancies from the multidisciplinary physicians' perspective, with particular focus on clincial infrastructures, and trial participation. Methods: The anonymous online survey consisted of 53 COVID-related questions. It was sent to all healthcare professionals in gynaecological oncology centres across Europe and the Pan-Arabian region from April 2020 to October 2020. All healthcare professionals treating women with gynecological cancers were able to participate in the survey. Results: A total of 243 answers were collected from 30 different countries. The majority (73%) of participants were gynecological oncologists from university hospitals(71%) with at least an Intensive care unit with cardiopulmonary support available at their institutions. Most institutions continued to perform elective surgeries only for oncological cases (98%). Patients had to wait on average 2 weeks longer for their surgery appointments compared to previous years(range 0-12 weeks). Cases that were prioritised for surgical intervention across all tumors (Ovarian, Endometrium, Cervical) were early stage disease (74%), primary situation (61%), and good ECOG status (63%). The radicality of surgery did not change in the majority of cases (78%) across all tumor types. During the pandemic, only 38% of clinicians stated they would start a new clinical trial. 45% stated the pandemic has negatively impacted the financial structure and support for clinical trials. 79% do not routinely screen patients included in trials for SARS CoV2. Overall, approx. 20% of clinicians did not feel well informed regarding clinical pathways for COVID-19 patients throughout the pandemic. The majority preferred regular updates and training via Webinars (75%), followed by tumorboards and interdisciplinary conferences (45%). 30% of clinicians stated that they are currently experiencing difficulties in providing adequate medical care due to staff shortage. Conclusions: Despite well-established guidelines for patient care and performing clinical trials in gynecological oncology, the COVID-19 pandemic has impacted clinical research, and financial structures. Longer waiting times for operative interventions, less support for clinical trials and concerns regarding provision of adequate medical care and triaging patients are very real. This survey underlines the necessity for building robust emergency algorithms tailored to gynecological oncology patients in the future.

National survey on the effect of oncology drug shortages in clinical practice: A Hematology Oncology Pharmacy Association (HOPA) survey.

2021 ◽  
Vol 39 (15_suppl) ◽  
pp. e13609-e13609
Author(s):  
Sarah Hudson-Disalle ◽  
David L. DeRemer ◽  
Larry W Buie ◽  
Mark Hamm ◽  
Jeffrey Pilz ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Supportive Care ◽  
Cancer Patients ◽  
Medication Errors ◽  
The United States ◽  
Adverse Outcomes ◽  
Common Disease ◽  
Drug Shortages ◽  
Oncology Drug ◽  
The Impact

e13609 Background: Drug shortages are a clear and growing challenge. Prominent shortages included oncology medications and supportive care products essential for the care of cancer patients. Oncology drug shortages often result in disruptions in the timing of chemotherapy treatments, alterations in the dose or regimen administered, or even missed doses when alternative agents are unavailable. The purpose of this survey was to characterize the impact of oncology drug shortages across the United States, including the experiences of health care organizations, resource implications, and the impact on patient safety, patient care, and clinical trials. Methods: A 34-item online survey was distributed to HOPA membership of the Hematology Oncology Pharmacy Association to gather information on shortages of oncology drugs (i.e., all drugs essential in the care of cancer patients, including supportive care agents. Results: Sixty-eight organizations completed the survey; almost all completed by pharmacists, and analysis completed. Sixty-three percent of institutions reported one or more drugs shortages a month, with a 34.33% increase in 2019 from 2018. Sixty four percent of responded had incurred increased costs from oncology drugs shortages, with 7% noting reimbursement issues when switched to brand name therapies due to shortages. Treatment delays, reduced doses or alternative regimens were reported by 74.63% of respondents. The most common disease states which causes a dose delay of treatment included Acute Lymphocytic Leukemia, Lymphoma and Multiple Myeloma with dose reductions noted in 36.36%, 36.36 and 15.91%. The top five oncology drugs on shortage included epirubicin, flutamide, decitabine, mechlorethamine, dactinomycin with the top 5 supportive care drugs on shortage being noted as hydrocortisone, bivalirudin, promethazine, mycophenolate sodium and scopolamine. Respondents noted medication errors related to oncology drug shortages at 4.48%, with noted errors including incorrect conversion from iv to oral etoposide and incorrect EMR drug builds. Oncology Drug shortages impacted clinical trials in 13.4% of respondents in which 54.55% of respondents noting patients not being enrolled in clinical trials. Conclusions: A survey of US oncology pharmacists and technicians indicated that oncology drug shortages occurred frequently in 2020. Shortages led to delays in chemotherapy and changes in treatment or omission, complicated clinical research and increased the risk of medication errors and adverse outcomes.

Impact of Peritoneal Dialysis Dose Guidelines on Clinical Outcomes

2005 ◽  
Vol 25 (3_suppl) ◽  
pp. 95-98 ◽  
Author(s):  
David N. Churchill
Keyword(s):  
Clinical Trials ◽  
Peritoneal Dialysis ◽  
Clinical Practice ◽  
Adverse Effects ◽  
Randomized Clinical Trials ◽  
Intervention Group ◽  
The United States ◽  
Dialysis Dose ◽  
Middle Molecules ◽  
The Impact

The objective was to review the rationale for the Kidney Disease Outcomes Quality Initiative (K/DOQI) recommendations for adequacy of peritoneal dialysis and to evaluate the impact of these recommendations on clinical practice and patient survival. The K/DOQI recommendations were based on large observational studies; the target weekly Kt/V value of 2.0 assumed equivalence of peritoneal and renal clearances. This assumption is no longer considered correct. The impact on clinical practice was evaluated by an examination of temporal trends before and after publication of the guidelines in 1997. In the United States and The Netherlands, there had been a trend toward increased delivered total Kt/V prior to 1997, and there was no acceleration in this trend after 1997. Two randomized clinical trials have implemented these guidelines with increased peritoneal Kt/V (or creatinine clearance) used to achieve the K/DOQI target in the intervention group. This was not associated with improved survival, compared to a lower Kt/V, in either of the randomized clinical trials. Among the explanations for the failure to improve outcome are potential adverse effects of increasing the dialysis dose. These include increased intraperitoneal pressure associated with increased exchange volume, failure to increase clearance of middle molecules, and increased exposure to glucose. Strategies that increase peritoneal clearance without exposure to these potential adverse effects include more-frequent exchanges rather than increased exchange volume, and decreased exposure to glucose and glucose degradation products. Pending such studies, current K/DOQI guidelines should be updated in a timely manner.

scholarly journals Patient Income Level and Cancer Clinical Trial Participation

2013 ◽  
Vol 31 (5) ◽  
pp. 536-542 ◽  
Author(s):  
Joseph M. Unger ◽  
Dawn L. Hershman ◽  
Kathy S. Albain ◽  
Carol M. Moinpour ◽  
Judith A. Petersen ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Clinical Trials ◽  
Universal Access ◽  
Participation Rate ◽  
Treatment Decision ◽  
Trial Participation ◽  
Lower Income ◽  
Clinical Trial Participation ◽  
Clinical Trial Results ◽  
The Impact

Purpose Studies have shown an association between socioeconomic status (SES) and quality of oncology care, but less is known about the impact of patient SES on clinical trial participation. Patients and Methods We assessed clinical trial participation patterns according to important SES (income, education) and demographic factors in a large sample of patients surveyed via an Internet-based treatment decision tool. Logistic regression, conditioning on type of cancer, was used. Attitudes toward clinical trials were assessed using prespecified items about treatment, treatment tolerability, convenience, and cost. Results From 2007 to 2011, 5,499 patients were successfully surveyed. Forty percent discussed clinical trials with their physician, 45% of discussions led to physician offers of clinical trial participation, and 51% of offers led to clinical trial participation. The overall clinical trial participation rate was 9%. In univariate models, older patients (P = .002) and patients with lower income (P = .001) and education (P = .02) were less likely to participate in clinical trials. In a multivariable model, income remained a statistically significant predictor of clinical trial participation (odds ratio, 0.73; 95% CI, 0.57 to 0.94; P = .01). Even in patients age ≥ 65 years, who have universal access to Medicare, lower income predicted lower trial participation. Cost concerns were much more evident among lower-income patients (P < .001). Conclusion Lower-income patients were less likely to participate in clinical trials, even when considering age group. A better understanding of why income is a barrier may help identify ways to make clinical trials better available to all patients and would increase the generalizability of clinical trial results across all income levels.

P003 INFLAMMATORY BOWEL DISEASE PATIENTS’ PERSPECTIVES OF CLINICAL TRIALS

2020 ◽  
Vol 26 (Supplement_1) ◽  
pp. S53-S53
Author(s):  
David Rubin ◽  
Laurent Peyrin-Biroulet ◽  
Walter Reinisch ◽  
Swati Tole ◽  
Laura Sullivan ◽  
...  
Keyword(s):  
Health Care ◽  
Clinical Trial ◽  
Clinical Trials ◽  
Health Care Providers ◽  
Online Survey ◽  
The United States ◽  
Trial Participation ◽  
Pharmaceutical Manufacturer ◽  
Care Providers ◽  
Inflammatory Bowel

Abstract Background Despite recent progress in treatment for inflammatory bowel diseases (IBD), there is a need for therapies with long-term efficacy and improved safety. Clinical trials in IBD face challenges with patient recruitment because of study designs, competitive or overlapping trials, and a limited number of eligible patients. We sought to better understand patients’ motivations, awareness of, and experience with IBD clinical trials. Methods We conducted an international survey of adult patients with IBD consisting of 2 components. The quantitative component, a 15-minute online survey, was completed by all patients. A qualitative component, a 30-minute telephone interview, was completed by a subset of patients from the United States (US). All percentages indicate results from the online survey. Results 226 patients (mean age, 41.9 y) completed the online survey. Survey respondents included patients with ulcerative colitis (52%) and Crohn’s disease (48%) from the US (n=100, 21 of whom underwent a phone interview), Brazil (n=26), Canada (n=25), France (n=25), Germany (n=25), and Spain (n=25). Ninety-six percent of respondents reported at least a basic understanding of clinical trials, and 34 (15%) were current or past clinical trial participants. Patients reported learning about trials through 1 or more sources (could select as many as applied): health care providers (42%), pharmaceutical manufacturer websites (31%), social media (30%), online support groups (28%), and foundations (18%-23%). In the survey, patients rated conversations with health care providers most helpful, but patients who were interviewed revealed that most physicians often do not initiate conversations about clinical trials, and patients typically do not ask. Primary motivators for trial participation (rated from “does not encourage me at all” to “encourages me very much”) included altruistic goals of advancing medicine (67%), potentially mitigating risks of uncontrolled IBD such as colon cancer (59%), and access to treatment options that could improve quality of life (59%) or would otherwise be unaffordable (52%). Major barriers to participation (rated from “does not discourage me at all” to “discourages me very much”) included invasive screening and monitoring (35%), concern over receiving placebo (35%), or suboptimal treatment (33%), and concerns about posttrial access to study medication (27%). The majority (68%) reported that being in a clinical trial means being a “guinea pig” for an experimental treatment. Conclusion Opportunities to improve patients’ clinical trial experience in IBD include better communication with health care providers and improved patient education about clinical trial design and ethics. Ultimately, a better understanding of the patient perspective will be important for more informed patients and potentially higher recruitment and enrollment.

Drug Development in the Era of Personalized Oncology: From Population-Based Trials to Enrichment and Prescreening Strategies

2012 ◽  
pp. 168-172
Author(s):  
Rodrigo Dienstmann ◽  
Jordi Rodon ◽  
Josep Tabernero
Keyword(s):  
Clinical Trials ◽  
Drug Development ◽  
Large Scale ◽  
Human Genetics ◽  
Tumor Biology ◽  
Drug Approval ◽  
Population Based ◽  
Tumor Subtypes ◽  
Recent Success ◽  
Personalized Oncology

Overview: Recent advances in tumor biology and human genetics along with the development of drugs for specific targets hold promise for an era of personalized oncology treatment. Routine use of modern technologies, such as large-scale genome sequencing, will help to unravel the specific biology of each tumor. Adding a rigorous genomic view could determine key genetic events, critical dependencies, and stratification of patients in early clinical trials. Integrating biomarker development into the early testing of novel agents might provide clinically relevant therapeutic opportunities for patients with advanced-stage cancer and also accelerate the drug-approval process. After recent success stories of therapies targeting driver molecular aberrations in genetically defined tumor subtypes, innovative clinical trials based on a strong biologic hypothesis are expected to bring further excitement to the field. In this article, we describe a new trend in biomarker-driven early drug development using enrichment and prescreening strategies. Technical and logistical obstacles that may hinder progress of this approach will be discussed, along with ethical and economic concerns.

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