Clinical Research Informer

STAT+: The FDA has approved a new Alzheimer’s drug, but wide access may depend on CMS easing restrictions

STAT News

JANUARY 6, 2023

Such decisions are based on myriad factors, starting with the average $26,500 price tag. But there are other considerations, including the quality of the clinical trial data, side effect concerns, the patient population for which the medicine is approved, and budgetary constraints.

Drugs

Drugs Clinical Trials Clinical Trials Medicine

Grand Rounds Biostatistics Series January 5, 2024: Methods for Handling Missing Data in Cluster Randomized Trials (Rui Wang, PhD; Moderator: Fan Li, PhD)

Rethinking Clinical Trials

JANUARY 9, 2024

For missing completed at random (MCAR) models, the missing process will be independent of all the covariate treatments and outcomes so that the observed data actually represent the underlying population you’re making an inference about. Most of an analysis can proceed using complete data, so this isn’t an issue.

Trials

Grand Rounds April 14, 2023: RECOVER in Action – Status of Clinical Trial Protocols (Kanecia Zimmerman, PhD, MD, MPH)

Rethinking Clinical Trials

APRIL 18, 2023

This broad set of clinical conditions and varied underlying causes underscore the need for testing a broad portfolio of therapeutic agents. The 5 trials, RECOVER-VITAL, RECOVER-AUTONOMIC, RECOVER-SLEEP, RECOVER-NEURO, and RECOVER-ENERGIZE, will enroll about 2,600 total participants, and each trial will include between 25-100 sites.

Clinical Trials

Clinical Trials Clinical Trials Trials Immune Response

Grand Rounds December 15, 2023: Diversifying Clinical Trials: A Path Forward (Roxana Mehran, MD, FACC, FAHA, MSCAI, FESC)

Rethinking Clinical Trials

DECEMBER 20, 2023

It is important to note when you look at the ARIC surveillance data, there is consistently higher risk in black females and males across all ages. Yet enrollment of ethnic minorities in NIH clinical trials and for trials studying approved devices and drugs remains low.

Clinical Trials

Clinical Trials Clinical Trials Trials Cardiology

Grand Rounds September 22, 2023: Integrating Community Health Workers into Team-Based, Early Childhood Preventative Care (Tumaini Rucker Coker, MD, MBA)

Rethinking Clinical Trials

SEPTEMBER 28, 2023

The Parent-Focused Redesign for Encounters, Newborns to Toddlers (PARENT) study was a randomized controlled trial of PARENT verses usual care for parents with infants 12 months and younger over a 12-month study period. The first year was spent allowing the clinics to make the project their own, with input from parents and clinicians.

Trials

Grand Rounds March 1, 2024: Effect of an Intensive Food-As-Medicine Program on Health and Health Care Use: Evidence from a Randomized Clinical Trial (Joseph Doyle, PhD)

Rethinking Clinical Trials

MARCH 6, 2024

Schell Professor of Management and Applied Economics MIT Sloan School of Management Slides Keywords Food-as-Medicine, Randomized Clinical Trial, Diabetes Key Points Diabetes is common and costly. Clinic staff included a dietitian, a nurse and a community health worker. The trial was for adults with HbA1c greater than 8.0,

Clinical Trials

Clinical Trials Clinical Trials Medicine Trials

Grand Rounds June 30, 2023: Decentralized Trials – From Guidance to Reality & What’s Left (Adrian Hernandez, MD, MHS; Pamela Tenaerts, MD, MPH; Craig Lipset, MPH)

Rethinking Clinical Trials

JULY 6, 2023

Over the past several years, a variety of factors have accelerated the need for decentralized trials, including the push to make trials more accessible, the increased speed of science, the possibility of environmentally conscious trials, and the need to be flexible in a rapidly changing world.

Trials

Trials Clinical Trials Clinical Trials Licensing

Grand Rounds May 12, 2023: Design and Pragmatic Trial of COACH: A Patient Portal/EHR Information System for Home Blood Pressure Monitoring in Hypertension (Richelle J. Koopman, MD, MS)

Rethinking Clinical Trials

MAY 19, 2023

Colwill Professor and Vice Chair Department of Family and Community Medicine University of Missouri Slides Keywords Electronic Health Record, Pragmatic Clinical Trial Key Points Patients bring patient-generated home blood pressure data into the clinical workflow. The primary outcome is blood pressure at 6 months.

Trials

September 30, 2022: CTTI’s Digital Health Trials Hub Recommendations and Resources to Run Your Digital Health Trial (Marianne Chase, MGH, CTTI Team Lead, Jörg Goldhahn, ETH Zurich, CTTI Team Lead)

Rethinking Clinical Trials

OCTOBER 4, 2022

Senior Director Clinical Trial Operations. Neurological Clinical Research Institute. Clinical Trial Transformation Initiative (CTTI); Decentralized Clinical Trials (DCTs); Digital endpoints; Developing novel endpoints. Decentralized trials require novel technology and digital endpoints.

Trials

Trials Clinical Trials Clinical Trials Production

Grand Rounds January 20, 2023: Collaborative Pragmatic Trials in Action: EVOLVE-MI (Mikhail Kosiborod, MD)

Rethinking Clinical Trials

JANUARY 26, 2023

This study is looking at a particular subpopulation, where there is a lack of outcomes data. EVOLVE-MI is a pragmatic, effectiveness outcome trial of Evolocumab dosed within 10 days of a myocardial infarction (MI). The recruitment rate is higher than what you would expect in a normal ACS trial.

Trials

Grand Rounds March 24, 2023: From Observational Studies to Pragmatic Clinical Trials: (Almost) A Decade of Research in PCORnet® (Erin Holve, PhD, MPH, MPP; Russell Rothman, MD, MPP; Schuyler Jones, MD; Neha Pagidipati, MD, MPH)

Rethinking Clinical Trials

MARCH 28, 2023

It is an independent, nonprofit, research institute and leading funder of patient-centered comparative clinical effectiveness research (CER). It has grown to be a major force for supporting pragmatic research, both real-world evidence research and supporting observational research and pragmatic clinical trials. enabled research.

Clinical Trials

Grand Rounds January 12, 2024: Design and Implementation of a Weighted Lottery to Equitably Allocate Scarce Covid-19 Resources (Erin K. McCreary, PharmD, BCIDP)

Rethinking Clinical Trials

JANUARY 17, 2024

UPMC did not allow patients to receive experimental COVID-19 therapies outside of the context of a clinical trial and used the REMAP-CAP platform, a global pragmatic adaptive trial platform, in all clinic sites. UPMC set clear criteria based on the published data at the time for who could and could not receive Remdesivir.

Clinical Trials

Clinical Trials Clinical Trials Antibody Trials

Grand Rounds October 13, 2023: Incorporating Social Determinants of Health Into PCORnet (Keith Marsolo, PhD)

Rethinking Clinical Trials

OCTOBER 18, 2023

Speaker Keith Marsolo, PhD Associate Professor Department of Population Health Sciences Duke University School of Medicine Slides Keywords PCORnet, Common Data Model, EHR, Social Determinants of Health Key Points There are many different definitions of social determinants of health. For example, consider food security.

Clinical Trials

Clinical Trials Clinical Trials Clinical Research Research

Grand Rounds September 29, 2023: Navigating the Use of Patient-Reported Outcomes in Research and Practice: The PROTEUS Consortium (Claire Snyder, PhD; Norah Crossnohere, PhD; Anne Schuster, PhD)

Rethinking Clinical Trials

OCTOBER 2, 2023

The PROTEUS Consortium’s objective is to ensure that patients, clinicians, and other decision-makers have high-quality PRO data from clinical trials and clinical practice to make the best decisions they can about treatment options.

Clinical Trials

Clinical Trials Clinical Trials Research Trials

Grand Rounds August 18, 2023: Opportunities to Improve Clinical Trials: Draft Revision of the ICH Good Clinical Practice Guideline (E6R3) (Prof Sir Martin Landray, FMedSci; M. Khair ElZarrad, PhD, MPH)

Rethinking Clinical Trials

AUGUST 28, 2023

Food and Drug Administration (FDA) Slides Keywords Pragmatic trials; Research; Guidance, Regulatory, Data Governance Key Points The clinical trial enterprise needs modernization. Time, cost and failure to recruit trial participants are significant barriers that must be addressed.

Clinical Trials

Clinical Trials Clinical Trials Trials Development

Grand Rounds August 18, 2023: Opportunities to Improve Clinical Trials: Draft Revision of the ICH Good Clinical Practice Guideline (E6R3) (Prof Sir Martin Landray, FMedSci; M. Khair ElZarrad, PhD, MPH; Adrian F. Hernandez, MD, MHS)

Rethinking Clinical Trials

AUGUST 28, 2023

Food and Drug Administration (FDA) Slides Keywords Pragmatic trials; Research; Guidance, Regulatory, Data Governance Key Points The clinical trial enterprise needs modernization. Time, cost and failure to recruit trial participants are significant barriers that must be addressed.

Clinical Trials

Clinical Trials Clinical Trials Trials Development

Grand Rounds August 25, 2023: Pragmatic Trial of an EHR Application to Display Real-time PRO Data: Successes and Challenges (Gabriela Schmajuk, MD, MS)

Rethinking Clinical Trials

AUGUST 30, 2023

To try to bridge the gap between PRO collection and meaningful use during clinical encounters, the RA PRO dashboard was developed. The RA PRO dashboard was tested as part of a stepped wedge, cluster randomized trial at the clinician level. 552 unique patient participated in the trial.

Trials

Trials Clinical Research Coordinator Medicine Clinical Trials

Grand Rounds January 26, 2024: Advancing the Safe, Effective and Equitable Use of AI in Healthcare (Mark Sendak, MD, MPP; Suresh Balu, MD, MBA)

Rethinking Clinical Trials

JANUARY 31, 2024

The second key decision point is development and adaptation, which is either building or adapting an external solution for internal clinical use. The next key decision point is clinical integration. – How do you get reliable information and data for care that is received outside of Duke?

Production

Production Development Clinical Trials Clinical Trials

Grand Rounds March 10, 2023: Estimands in Cluster-Randomized Trials: Choosing Analyses that Answer the Right Question (Brennan Kahan, PhD)

Rethinking Clinical Trials

MARCH 15, 2023

An estimand is a precise description of the treatment effect that a researcher aims to estimate from the trial. The difference relates to how data is weighed. Two estimands will differ when there is informative cluster size in a trial. Two estimands will differ when there is informative cluster size in a trial.

Trials

Grand Rounds February 24, 2023: S2302 Pragmatica-Lung: New Directions for Decreasing Burden and Increasing Inclusion in NCTN Clinical Trials (Konstantin Dragnev, MD; Karen Reckamp, MD, MS)

Rethinking Clinical Trials

MARCH 1, 2023

To reduce the burden on sites, the study has simplified data reporting by reducing time points data, reducing the number of forms that need to be submitted, and reducing the number of data elements within a form. One network is the lead, but the trial will be available in all four U.S.

Clinical Trials

Clinical Trials Clinical Trials Trials Packaging

Grand Rounds December 1, 2023: Guidelines for Design and Analysis of Stepped-Wedge Trials (James P. Hughes, PhD)

Rethinking Clinical Trials

DECEMBER 6, 2023

Hughes, PhD Professor Emeritus of Biostatistics, University of Washington Slides Keywords Design, Analysis, Stepped-Wedge Trial Key Points Stepped-wedge design is typically run by clusters that are randomized. Another issue to think about with stepped-wedge trials is potential sources of variation. Speaker James P.

Trials

Biogen, Eisai Alzheimer’s drug lecanemab fast tracked by FDA

pharmaphorum

DECEMBER 24, 2021

The anti-amyloid drug, which also claimed a breakthrough tag from the FDA in July, is being developed for the treatment of early-stage Alzheimer’s disease. In a statement, Biogen and Eisai said that the FDA has agreed that Clarity AD “can serve as the confirmatory study to verify the clinical benefit of lecanemab.”

Drugs

Drugs FDA Approval Trials Marketing

Grand Rounds October 6, 2023: Hybrid Studies Should Not Sacrifice Rigorous Methods (David M. Murray, PhD; Moderator: Jonathan Moyer, PhD)

Rethinking Clinical Trials

OCTOBER 11, 2023

Through this initiative, 7 Clinical Centers are expected to test an evidence-based, multi-level intervention designed to reduce or eliminate cardiovascular and/or pulmonary health disparities. They also helped review each Clinical Center’s protocol before it went to the DSMB and NHLBI for transition to the UH3 phase.

Clinical Trials

Grand Rounds May 19, 2023: Aspirin or Low-Molecular-Weight Heparin for Thromboprophylaxis After a Fracture (Robert O’Toole, MD)

Rethinking Clinical Trials

MAY 25, 2023

A pilot study, called the ADAPT trial, demonstrated feasibility, similar in-patient compliance and similar post-discharge adherence for the 2 options. The pragmatic randomized control trial was designed from a hospital policy perspective. This inspired the research team to shift the primary outcome to death.

Trials

Trials Clinical Trials Clinical Trials Compliance

Grand Rounds June 16, 2023: BeatPain Utah: Partnering With Community Health Centers Within a Socio-Technical Framework (Julie Fritz, PT, PhD, FAPTA; Guilherme Del Fiol, MD, PhD)

Rethinking Clinical Trials

JULY 9, 2023

Clinical research can exacerbate disparities, because clinical trials typically are based in urban, academic medical centers, underrepresent diverse populations, and overlook community engagement strategies in trial planning and design. The clinic’s priorities should drive the design of the trial. “We

Clinical Trials

June 24, 2022: FDA Draft Guidance on Real-World Evidence (John Concato, MD, MS, MPH)

Rethinking Clinical Trials

JUNE 29, 2022

Big data; Real-word evidence; Real-world data; 21st Century Cures Act; FDA Draft Guidance. Big Data, a term first used in the 1990s, leverages modern technology to increase the quantity, forms, speed, and capability to manipulate large-scale data. Food and Drug Administration (FDA). Key Points. Discussion Themes.

Big Data

Big Data Clinical Trials Clinical Trials Drugs

Grand Rounds August 19, 2022: Inclusion and Diversity in Clinical Trials: Actionable Steps to Drive Lasting Change (Gerald Bloomfield, MD, MPH; Michelle Kelsey, MD)

Rethinking Clinical Trials

AUGUST 23, 2022

There is a national priority to increase diversity in clinical trials, from regulatory, funding agencies, industry, and others, so that the participant population reflects the U.S. The Duke Clinical Research Institute (DCRI) organized and sponsored a Think Tank to address diversity in clinical trial research.

Clinical Trials

Clinical Trials Clinical Trials Trials Medicine

Cartherics grants licence for CTH-004 to Shunxi

Pharmaceutical Technology

MAY 1, 2023

CTH-004 is developed by genetically altering patient T cells for inserting a chimeric antigen receptor (CAR) to target a marker (TAG-72) on ovarian cancer cells and delete genes which are involved in T cell function suppression. Additionally, both the companies will share CTH-004 pre-clinical and clinical data in future.

Gene Therapy

Gene Therapy Gene Development Clinical Trials

Grand Rounds July 15, 2022: Overview of Accelerating COVID-19 Therapeutic Interventions and Vaccines (ACTIV) Public-Private Partnership and Lessons Learned (Stacey J. Adam, PhD)

Rethinking Clinical Trials

JULY 22, 2022

There were four ACTIV fast-track focus areas: vaccines, preclinical, clinical trial capacity, and therapeutics – clinical. Candidate agents were triaged based on concurrent clinical trials, completion of a multiple ascending dose study, and availability of preclinical data before being scored based on predefined criteria.

Vaccination

Vaccination Vaccine Clinical Trials Clinical Trials

Grand Rounds May 5, 2023: All of Us Research Program: Improving Health Through Diverse Technology, Huge Cohorts, and Precision Medicine (Joshua C. Denny, MD, MS)

Rethinking Clinical Trials

MAY 10, 2023

Participants have access to their information, data will be access broadly for research purposes, and security and privacy will be of highest importance. All of Us has a philosophy to get data to researchers quickly. The Research Workbench data browser is available with no log in and you get aggregate results.

Medicine

Medicine Research Clinical Trials Clinical Trials

Grand Rounds July 22, 2022: ACTIV-6: 1-Year Later and Trial Results for Ivermectin-400 and Inhaled Fluticasone (Susanna Naggie, MD, MHS)

Rethinking Clinical Trials

JULY 27, 2022

Vice Dean for Clinical Research. The key clinical questions of the ACTIV-6 study are: how to help someone feel better faster with newly diagnosed mild-moderate COVID-19 and how to prevent hospitalizations or death in someone with newly diagnosed mild-moderate COVID-19? We did not see as much diversity in this decentralized trial.

Trials

Trials Pharmacy Medicine Clinical Trials

Grand Rounds September 16, 2022: Using Nationwide Registries to Conduct Pragmatic Randomized Trials: The DANFLU Program (Tor Biering-Sørensen, MD, PhD, MPH)

Rethinking Clinical Trials

SEPTEMBER 22, 2022

Professor in Translational Cardiology and Pragmatic Randomized Trials. Head of Center for Translational Cardiology and Pragmatic Randomized Trials. DANFLU-1, Pragmatic Clinical Trials. The DECODE trial used registry data to identify patients with chronic kidney disease. Tor Biering-Sørensen, MD, PhD, MPH.

Trials

Trials Cardiology Vaccination Vaccine

Grand Rounds December 16, 2022: The Use of EHR-Agnostic Clinical Decision Support to Prevent Thromboembolism in Hospitalized Medically Ill Patients (Alex C. Spyropoulos, MD, FACP, FCCP, FRCPC; Jeffrey Solomon, BFA)

Rethinking Clinical Trials

FEBRUARY 9, 2023

The study team’s health informatics group developed a novel clinical decision support (CDS) tool, called IMPROVE-DD VTE CDS, which can be integrated into different electronic health record (EHR) systems. In doing this, they were able to replicate the data among different populations.

Clinical Trials

Clinical Trials Clinical Trials Trials Development

Grand Rounds July 14, 2023: Lessons From the COORDINATE-Diabetes Trial (Christopher B. Granger, MD; Neha J. Pagidipati, MD, MPH)

Rethinking Clinical Trials

JULY 19, 2023

However, these therapies are highly underused in routine clinical practice. The objective of COORDINATE-Diabetes trial was to improve the implementation and adoption of these therapies by testing the impact of a clinic-level, multifaceted intervention on the prescription of 3 key groups of evidence-based therapies.

Trials

Trials Cardiology Medicine Nurses

Sarepta’s Elevidys Reaches Finish Line as First Gene Therapy Approved for Duchenne Muscular Dystrophy

XTalks

JUNE 28, 2023

The approval is restricted to ambulatory patients within this narrow age range due to uncertainty around its effectiveness in older children, which Sarepta hopes to clarify in a confirmatory trial. Additionally, the FDA said a “clinical benefit of Elevidys, including improved motor function, has not been established.”

Gene Therapy

Gene Therapy Gene Protein FDA Approval

Global (68M) cardiomyopathies market projected to grow to $9.63bn by 2031

Pharmaceutical Technology

DECEMBER 15, 2022

This extrapolation leverages data from GlobalData’s World Markets Healthcare and PharmOnline International databases to expand the number of markets in GlobalData’s patient-based sales forecast model from seven major markets (7MM) to 68 markets (68M). of sales in 2021 and 2031 respectively. of global cardiomyopathies sales in 2021 and 31.2%

Marketing

Marketing Sales Clinical Trials Clinical Trials

Grand Rounds August 4, 2023: AI & ML: Want to Play a Game? (Eric Perakslis, PhD)

Rethinking Clinical Trials

AUGUST 10, 2023

” The key to quality of AI and utilizing AI in a beneficial way relates to the quality of the data. It’s important for organizations to recognize this and upgrade their data in order to these technologies more effectively and efficiently. This can be useful in the context of clinical trials.

Clinical Trials

Clinical Trials Clinical Trials Trials Medicine

Grand Rounds April 21, 2023: Personalised Cooler Dialysate for Patients Receiving Maintenance Haemodialysis (MyTEMP): A Pragmatic, Cluster-randomised Trial (Amit Garg, MD, MA, FRCPC, FACP, PhD; Stephanie N. Dixon, PhD MSc)

Rethinking Clinical Trials

APRIL 27, 2023

This change in practice is based on data that suggests the at cooler (vs. The MyTEMP trial is a pragmatic, cluster randomized controlled trial in Ontario, Canada, to determine if adopting a default center-wide policy of personalized cooler dialysate is superior to a standard temperature dialysate of 36.5 degrees Celsius.

Trials

Grand Rounds October 21, 2022: Disinformation, Cyberthreat, and Choice: Protecting Patients and Clinical Research From the Digital Triple Threat (Eric Perakslis, PhD; Andrea Downing)

Rethinking Clinical Trials

OCTOBER 27, 2022

Duke Clinical Research Institute. Data Security, Disinformation, Cyberthreat. It was started when Andrea discovered a vulnerability in Facebook’s group platform that allowed data to be scraped outside of the group. The problem is patients can’t predict benefit or harm based on how their data is used. Eric Perakslis, PhD.

Clinical Research

Clinical Research Research Clinical Trials Clinical Trials

Grand Rounds September 8, 2023: The DEVICE Trial: An Embedded, Pragmatic Trial of Emergency Airway Management (Matthew Prekker, MD, MPH; Jonathan Casey, MD, MSc)

Rethinking Clinical Trials

SEPTEMBER 13, 2023

The DEVICE trial hypothesized that the use of a video laryngoscope will increase the incidence of successful intubation on the first attempt. The trial operated under an IRB waiver of informed consent with a patient information sheet. Discussion Themes -Why did the RSI trial follow a different path – not a waiver of consent?

Trials