Author Interviews, Heart Disease, JAMA / 10.09.2019

MedicalResearch.com Interview with: [caption id="attachment_51215" align="alignleft" width="200"]Nariman Sepehrvand, MD Research Associate & PhD Candidate Canadian VIGOUR Centre, and Department of Medicine, University of Alberta, Edmonton, Canada Dr. Sepehrvand[/caption] Nariman Sepehrvand, MD Research Associate & PhD Candidate Canadian VIGOUR Centre, and Department of Medicine, University of Alberta, Edmonton, Canada Medical Research: Can you tell us a little bit about the background of this study? Dr. Sepehrvand: As you know the traditional randomized clinical trials (RCT) have been criticized from time to time for the lack of generalizability, high costs and lengthy processes. Pragmatic trials with the primary goal of informing patients, clinicians, healthcare administrators and policy-makers about the effectiveness of biomedical and behavioral interventions have the potential to address those shortcomings by enrolling a population representative of the populations in which the intervention will be eventually applied to and by streamlining and simplifying the trial-related procedures. We knew about the challenges that trialists encounter in the design and implementation of pragmatic trials, so we were wondering how pragmatic or explanatory are cardiovascular (CV) RCTs and if there has been any change over time!
Annals Internal Medicine, Author Interviews, Heart Disease / 29.07.2019

MedicalResearch.com Interview with: [caption id="attachment_50478" align="alignleft" width="153"]Jeffrey L Jackson, MD, MPH Medical College of Wisconsin Milwaukee, Wisconsin Dr. Jackson[/caption] Jeffrey L Jackson, MD, MPH Medical College of Wisconsin Milwaukee, Wisconsin MedicalResearch.com: What is the background for this study? What are the main findings? Response: Unfortunately,  most systematic reviews exclude non-English trials, mostly for convenience, but nearly all systematic reviews wind up excluding at least 1 non-English trial.  We looked at whether this was justified, since Google Translate is a free and easily usable platform.  We had native-language speakers in 9 languages (Chinese, French, German, Italian, Japanese, Korean, Romanian, Russian and Spanish) abstract data and had another researcher abstract all the articles using Google Translate. We found that there was over 90% agreement and that the few differences were due to human error, not to problems with the translations.
Author Interviews, Cancer Research, FDA, JAMA / 01.04.2019

MedicalResearch.com Interview with: [caption id="attachment_48283" align="alignleft" width="158"]Emerson Chen, MDChief Fellow, Hematology-Oncology, PGY-6Oregon Health & Science University Dr. Chen[/caption] Emerson Chen, MD Chief Fellow, Hematology-Oncology, PGY-6 Oregon Health & Science University MedicalResearch.com: What is the background for this study? What are the main findings? Response: Many cancer drugs are approved annually giving the appearance of innovation; however, some drugs may have been approved because of a lower bar. Use of lesser endpoints like response rate (how tumor shrinks) and progression-free survival (how tumor has delayed growth) have been proposed to speed trials when compared against traditional endpoints like overall survival (how long patients might live). Using published trials that led to cancer drug approval from 2006 to 2017, we estimated how long it would take to get each of these three endpoints across all cancer drugs and indications to see how much time we could save by using these weaker but faster endpoints. We see that many trials using overall survival used less time than anticipated, and many trials using response rate or progression-free survival actually took quite a bit of time.  In part that is due to researchers needing to document the duration of the response. But, whatever the reason, the time to get each of the three endpoints is actually more similar than different, and we estimate that our current use of  these faster endpoints are saving us only 11 months compared to using only overall survival.
Author Interviews, Duke, Heart Disease, JAMA / 21.03.2019

MedicalResearch.com Interview with: Renato D. Lopes MD, MHS, PhD Professor of Medicine Division of Cardiology Duke University Medical Center Duke Clinical Research Institute Alexander C. Fanaroff, MD, MHS Division of Cardiology and Duke Clinical Research Institute Duke University, Durham, North Carolina MedicalResearch.com: What is the background for this study? What are the main findings? Response: About ten years ago, a group of researchers examined the evidence supporting guideline recommendations in cardiology for the first time. Quite surprisingly, they found that only 11% of recommendations in American College of Cardiology/American Heart Association (ACC/AHA) guidelines were supported by evidence from randomized controlled trials, the highest level of evidence. The researchers called for greater collaboration among investigators and funders in identifying key research questions, development of streamlined clinical trial methods, and expansion of funding for clinical research. Over the past 10 years, some of these steps have been taken, but it is unclear how the evidence supporting guideline recommendations has changed. We therefore analyzed the 51 current cardiovascular guideline documents -- 26 from the ACC/AHA and 25 from the European Society of Cardiology (ESC) -- including 6,329 recommendations. Overall, 8.5% of recommendations in ACC/AHA guidelines and 14.3% of recommendations in ESC guidelines were supported by evidence from randomized controlled trials. When looking specifically at guidelines that have been updated, we found no significant changes in the proportion of recommendations supported by evidence from randomized controlled trials.
Author Interviews, JAMA, Pain Research, Technology / 06.09.2018

MedicalResearch.com Interview with: [caption id="attachment_44273" align="alignleft" width="150"]Richard L Kravitz, MD, MSPH Professor, General Internal Medicine Director, UC Center Sacramento Dr. Kravitz[/caption] Richard L Kravitz, MD, MSPH Professor, General Internal Medicine Director, UC Center Sacramento MedicalResearch.com: What is the background for this study? What are the main findings?  Response:  The study was designed to address tso problems. The first is that many patients with chronic pain struggling to find a workable regimen. The second is more general. Patient sometimes I hesitate to participate in clinical research because they right away do not see the relevance I directly to them selves. And have one trials are away I’m addressing both problems. 
Author Interviews, BMJ / 27.07.2018

MedicalResearch.com Interview with: [caption id="attachment_43533" align="alignleft" width="166"]Trudo Lemmens (LicJur, LLM bioethics, DCL) Professor and Scholl Chair in Health Law and Policy Faculty of Law, University of Toronto Toronto Ontario, Canada Prof. Lemmens[/caption] Trudo Lemmens (LicJur, LLM bioethics, DCL) Professor and Scholl Chair in Health Law and Policy Faculty of Law, University of Toronto Toronto Ontario, Canada  MedicalResearch.com: What is the background for this study? Response: The study is part of a series of articles organized in collaboration between the BMJ and the Pan American Health Organization, with particular involvement of members of PAHO’s Advisory Committee on Health Research. The goal of the series is to highlight some of the key accomplishments in the PAHO region in the last decades following PAHO’s 2009 regional policy on research for health, the first WHO regional policy that aimed at contributing to WHOs Stragegy on Research for Health. One of the issues that have been identified in the last decades as an important challenge, particularly for research related to pharmaceutical product and medical device development, is the lack of transparency of clinical trials data. This has been associated with problems of hiding and misrepresentation of research results, duplication of research, and misleading or even outright fraudulent presentations of findings in medical publications. Various remedies have been proposed to address these problems, from trial registration, to results reporting, to broader data sharing that enables independent researchers to verify and challenge research results. I have been involved through PAHO and in my own research in the promotion of transparency measures at the national and international level. PAHO has been playing a leading role in this in the region, so it seemed more than fitting to have an article in the series on what has been accomplished and what challenges remain.
Author Interviews, Breast Cancer, Cancer Research, Yale / 28.01.2018

MedicalResearch.com Interview with: [caption id="attachment_39559" align="alignleft" width="130"]Lajos Pusztai, M.D, D.Phil. Professor of Medicine Director of Breast Cancer Translational Research Co-Director of the Yale Cancer Center Genetics, Genomics and Epigenetics Program Yale School of Medicine New Haven, CT  06511 Dr. Pusztai[/caption] Lajos Pusztai, M.D, D.Phil. Professor of Medicine Director of Breast Cancer Translational Research Co-Director of the Yale Cancer Center Genetics, Genomics and Epigenetics Program Yale School of Medicine New Haven, CT  06511 MedicalResearch.com: What is the background for this study? Response: Overall, about 85% of newly diagnosed stage I-III breast cancer patients will not die of their disease, and this roughly equates to an 85% cure rate. Of course cure rates are higher for stage I cancers and lower for stage III cancers. An 85% overall cure rate is good but not good enough, we continuously try to develop new therapies hoping to push these rates to 90%...,95%...etc. However, it is not possible to cure a patient twice over. For example, if surgery plus endocrine therapy cures all patients, the addition of chemotherapy cannot improve on it no matter how effective it is. If surgery plus endocrine therapy cures 95%, adding the perfect chemo to this treatment can only bring about a 5% improvement, and very good chemo that would push cure from 95% to 97%, would require a very large trial including many thousands of patients. This is an increasingly common scenario in modern breast cancer adjuvant trials (where the goal is to improve survival and cure); the control arm that receives the current standard of care invariably does better than expected and the experimental arm only improves outcome by 1-3% that does not reach statistical significance.  The painful conclusion from these trials is that we do not know if the new drug actually works or not because there were not enough events to demonstrate an effect. Of course, a lot of patients in the study were also exposed to a new drug with all of its associated toxicities who could not possibly benefit from it.
Author Interviews, ESMO, Pharmacology / 09.10.2016

MedicalResearch.com Interview with: [caption id="attachment_28678" align="alignleft" width="144"]Paolo Bossi MD Medical Oncologist Head and Neck Cancer Department  IRCCS Istituto Nazionale dei Tumori Foundation Milan, Italy Dr. Paolo Bossi[/caption] Paolo Bossi MD Medical Oncologist Head and Neck Cancer Department IRCCS Istituto Nazionale dei Tumori Foundation Milan, Italy MedicalResearch.com: What is the background for this study? What are the main findings? Response: Precise, clear and unbiased reporting of adverse events (AE) is essential to ensure safety of the new drugs. It is crucial also in engaging patients and physicians in a shared decision making: before starting a new treatment I need to discuss with my pt what are the expected benefits and what the toxicities of a new drugs. However, in parallel to the discovery and development of new drugs, little attention has been paid to modernization of the way of collecting toxicities. This line of reasoning is particularly true for new or "relatively new" drugs, such as immunotherapy (IT) and targeted agents (TT). So, we analysed all the trials that lead to the approval of TT or IT from 2000 – 2015 retrieved by FDA database.
Author Interviews, Biomarkers, UCSF / 10.05.2015

Alan H.B. Wu, PhD, DABCC Professor Laboratory Medicine Chief, Clinical Chemistry Laboratory University of California San Francisco, CAMedicalResearch.com Interview with: Alan H.B. Wu, PhD, DABCC Professor Laboratory Medicine Chief, Clinical Chemistry Laboratory University of California San Francisco, CA Medical Research: What is the background for this study? What are the main findings? Response: Clinical trials are conducted for validation of novel biomarkers.  There is considerable heterogeniety in the quality design and documentation of findings. This can have a major impact on the conclusions rendered.
Author Interviews, General Medicine, Race/Ethnic Diversity / 29.12.2013

Aisha T. Langford, PhD, MPH Postdoctoral Fellow VA Health Services Research and Development Service (HSR&D & U-M Center for Bioethics and Social Sciences in Medicine (CBSSM) 2800 Plymouth Road, NCRC Building 16, Room 400S-15 Ann Arbor, MI 48109MedicalResearch.com Interview with: Aisha T. Langford, PhD, MPH Postdoctoral Fellow VA Health Services Research and Development Service & U-M Center for Bioethics and Social Sciences in Medicine Ann Arbor, MI 48109 MedicalResearch.com: What are the main findings of the study? Dr. Langford: The main and perhaps most interesting finding was that there were no racial/ethnic differences in cancer clinical trial enrollment, refusal rates, or "no desire to participate in research" as the reason given for clinical trial refusal; however, patients over the age of 65 had lower odds of being enrolled in a clinical trial. Additionally, higher odds of having physical/medical conditions were associated with older age, males, and non-Hispanic blacks.