CTTI News

Getting Down to the Bottom Line: The Financial Impact of Patient Engagement in Clinical Trials

Posted on by Lorri Bingham

CTTI publishes framework for estimating the impact of patient engagement on key business drivers

To fully realize the impact of patient engagement in clinical trials, there is a need for robust and rigorous study of its quantitative effects on research and development. To begin to address this gap, CTTI recently published an approach for assessing the financial value of patient engagement that accounts for the business drivers of cost, risk, revenue, and time.

In a set of recommendations published along with the financial model, CTTI’s Patient Groups & Clinical Trials Project team outlines “rules of engagement” for successful collaborations between research sponsors and patient groups around clinical research. Together, the articles in Therapeutic Innovation & Regulatory Science (TIRS) provide a blueprint for establishing effective patient group engagement in research programs.

While research sponsors are increasingly partnering with patients in the design and conduct of clinical development programs, sponsors may be reluctant to go beyond pilot initiatives because of uncertainty in the return on investment. The new CTTI article describes a novel approach to quantifying the financial impact of patient-centric initiatives using risk-adjusted financial modeling techniques that integrate key business drivers into a summary metric (expected net present value). Expected net present value (ENPV) is a widely used metric by industry for project strategy and portfolio decisions.

Drawing on published data to develop a case study for a typical oncology development program, the authors also show how patient engagement activities that avoid a protocol amendment or improve the patient experience can contribute considerable financial value.

Ongoing work by CTTI’s Patient Groups & Clinical Trials Project is examining how sponsors and patient groups characterize the benefits of patient engagement and investments required for engagement. Along with the ENPV model, the project aims to provide resources to assist in determining which of the many engagement opportunities provide the greatest value.

Strengthening the Investigator Site Community

Posted on by Lorri Bingham

Gain new insights into challenges affecting investigator retention and potential solutions

Evidence shows that the pool of qualified clinical investigators in the United States is shrinking, while at the same time a high percentage of investigators are likely to stop leading trials after conducting just a single drug study—trends that threaten the quality and efficiency of U.S. regulatory trials.

On April 5, 2017, CTTI convened an expert meeting to explore this issue and others related to clinical trial investigator turnover and retention. A comprehensive meeting summary presents survey and interview data gathered from current and former trial investigators and explores approaches that can help investigators overcome the practical challenges of conducting clinical trials.

CTTI’s Investigator Community Project was created to better understand why some clinical investigators remain actively engaged in clinical research but others decide to leave it after a single experience. CTTI is applying these insights to develop strategies that can improve investigator retention and reduce turnover, ultimately leading to more efficient, higher-quality clinical trials. The multi-stakeholder project team will continue to review evidence and incorporate consensus findings from the expert meeting as it develops recommendations and other products that can be used to strengthen the investigator community.

CTTI Seeks to Speed Use of Mobile Technology in Clinical Trials through Novel Endpoint Recommendations

Posted on by Lorri Bingham

Novel Endpoints Recs Banner

New CTTI Recommendations and Tools Equip Stakeholders to Integrate Mobile Technology into Clinical Trials

Mobile technologies hold enormous promise for clinical research, but uncertainty about how to use the data captured by these devices has slowed progress. CTTI’s newly released recommendations and tools aim to change this by providing a pathway for using information gathered from mobile technologies to accelerate the development and evaluation of urgently needed therapies.

Mobile technologies such as remote sensors and wearables can be used to make trials faster, more efficient, and more inclusive. They can also spare patients from burdensome clinic visits while capturing new kinds of data that offer a better picture of how patients experience their disease or condition in their daily lives.

“Technology-derived endpoints offer the benefit of capturing information about patients’ experience in ‘real-world’ settings,” said Dr. Janet Woodcock, director of the FDA’s Center for Drug Evaluation and Research. “These tools have the potential to capture data that can be used to develop endpoints and evaluate therapies in the patient population.”

Responding to this potential, CTTI experts have crafted a set of recommendations and tools designed to help diverse stakeholders identify and develop novel endpoints based on data from mobile technologies for use in regulatory clinical trials.

“By engaging with experts who have been early champions of mobile technology in trials and combining that with patient insights, CTTI has created practical recommendations and action-oriented tools that have the potential to really accelerate the use of mobile technology in clinical trials,” noted Craig Lipset, Pfizer’s head of clinical innovation. “In particular, the use cases provide a realistic pathway for incorporating novel endpoints through technology into clinical development programs. CTTI’s recommendations show we may be closer than previously believed to realizing the benefits of these novel endpoints, creating a sense of urgency to act.”

In partnership with a group of investigators, regulators, patient representatives, technology developers, and research sponsors, four use cases were written to better guide the development and use of novel endpoints as part of clinical trials for Parkinson’s disease, heart failure, diabetes, and Duchenne muscular dystrophy.

“The Michael J. Fox Foundation was pleased to participate in CTTI’s efforts to develop novel mobile endpoints for use in clinical trials,” said Lauren Bataille, senior associate director of research partnerships at the Foundation. “We look forward to leveraging these assets to support Parkinson’s research collaborations and speed the developments of measures that matter to patients.”

CTTI’s Novel Endpoints recommendations are the first to be released as part of a larger body of work to address multiple challenges to using mobile technology in clinical trials. CTTI’s Mobile Clinical Trials Program includes additional projects on legal and regulatory considerations, stakeholder perceptions, and scientific and technical issues related to the use of mobile devices. Recommendations from these projects will follow over the next year.

Click here to view more information on CTTI’s Novel Endpoints Project.

To view a recording of the webinar that unveiled these recommendations, click here.

Accelerating the Use of Mobile Technology In Clinical Trials: Webinar Recording Now Available

Posted on by Lorri Bingham

EXPERTS UNVEIL NEW RECOMMENDATIONS AND TOOLS FOR INTEGRATING NOVEL ENDPOINTS DERIVED FROM MOBILE TECHNOLOGY INTO CLINICAL TRIALS

Mobile technologies are reshaping the landscape of clinical trials and making it possible to capture new kinds of patient data. Learn how novel endpoints derived from these technologies can be leveraged for more efficient, less burdensome clinical trials that generate the high-quality data needed to address the outcomes that matter most to patients.

In this webinar, a group of expert presenters and panelists describe CTTI’s new recommendations and tools for identifying and developing technology-based novel endpoints and incorporating them into clinical trials. Drawn from backgrounds spanning academia, industry, technology development, patient advocacy, and regulatory agencies, CTTI’s expert stakeholders provide a “guided tour” of these resources. They also discuss key strategies—including early engagement and discussion with regulators—for successfully integrating novel endpoints into clinical trials that enable a better understanding of the effects of diseases and their treatments on patients’ daily lives. CTTI’s MCT Novel Endpoints recommendations, which were developed from a multi-stakeholder expert meeting that applied four “real-world” use cases (heart failure, diabetes, Parkinson’s disease, and Duchenne muscular dystrophy), address the following key areas:

  • Identifying and developing high-quality novel endpoints that best reflect patients’ priorities and needs
  • Selecting the best outcome measures and matching those measures with appropriate technologies
  • Developing products that address unmet needs and adapting solutions that meet the demands of the clinical research and patient care environments
  • Supporting trial sponsors and researchers in working effectively with regulators to develop evidence-based, high-quality novel endpoints that can be used to support regulatory review and approval

FOR MORE INFORMATION ON THIS WEBINAR, INCLUDING POWERPOINT SLIDES, PRESENTER INFORMATION, AND MORE, CLICK HERE.

The MCT Novel Endpoints Project is part of CTTI’s larger Mobile Clinical Trials Program.

CTTI Advances Mobile Clinical Trials & Patient Engagement at DIA 2017

Posted on by Lorri Bingham

Driving insights to action with our latest findings

CTTI Schedule at DIA 2017At this year’s DIA Annual Meeting, CTTI will present new approaches that can help bring the use of mobile technology in clinical trials from promise to reality. Advancements in data capture with mobile technology can translate to improved clinical trial quality and efficiency. CTTI will share case examples and methods for addressing specific challenges of integrating mobile devices into clinical trials in four presentations. These are preliminary results from CTTI’s Mobile Clinical Trials Program.

In addition, we will share our latest findings on measuring the financial impact of patient engagement from the Patient Groups & Clinical Trials Project. If you’ve been searching for a way to help sponsors and patient groups speak the same language in terms of perceived value of collaboration, this is the presentation for you. It is widely accepted that patient engagement can add value to the research process, but when it comes to business decisions, sponsors may be reluctant to allocate resources to patient engagement without a measure of financial impact. We present financial modeling for the impact of patient engagement to help inform these decisions.

DIA 2017 will be held in Chicago from June 18-22 and is themed “Driving Insights to Action.” Please join us for CTTI’s presentations:

Presentation: At the cornerstone of wearable technology and patient-focused clinical trial endpoints (#227)
Date & Time: Tuesday, June 20, 2017 from 10:30-11:45 a.m.
Presenters: Leonard Sacks, Nikunj Patel, Robert DiCicco, Ray Dorsey
Related CTTI Projects: MCT Novel Endpoints & Mobile Devices

Presentation: Generating novel endpoints through mobile technology: A promising and realistic new approach to trial design (#244)
Date & Time: Tuesday, June 20, 2017 from 2:00-3:15 p.m.
Presenter: Robert DiCicco
Related CTTI Project: MCT Novel Endpoints

Presentation: Getting down to the nitty-gritty: Addressing the specific challenges of electronic data and mobile devices in clinical trials (#308)
Date & Time: Wednesday, June 21, 2017 from 10:30-11:30 a.m.
Moderator: Jonathan Helfgott; Presenter: Jennifer Goldsack
Related CTTI Project: MCT Mobile Devices

Presentation: Getting down to the bottom line: Measuring the financial impact of patient engagement (#316)
Date & Time: Wednesday June 21, 2017 from 10:30-11:45 a.m.
Presenter: Bennett Levitan
Related CTTI Project: Patient Groups & Clinical Trials

Presentation: Straight from the horse’s mouth: Lessons learned from legal & regulatory challenges of real remote trials (#366)
Date & Time: Wednesday, June 21, 2017 from 4:00-5:15 p.m.
Presenter: Gerrit Hamre
Related CTTI Project: MCT Legal & Regulatory

Webinar June 26: Launching New CTTI Recommendations to Accelerate the Appropriate Use of Mobile Technology in Clinical Trials

Posted on by Lorri Bingham

CTTI Webinar

Mobile technologies offer unique opportunities for making clinical trials faster, more efficient, and more representative of actual patient experiences. By addressing unmet needs for quality outcome measures, enabling more efficient alternatives to traditional clinical research models, and reducing dependence on data that represent only a tiny snapshot of patients’ experience of disease, mobile technologies allow us to capture new kinds of information while reducing the burdens of trial participation. However, realizing these benefits requires a clear understanding of how to successfully select, develop and incorporate technology-derived novel endpoints and incorporate them into clinical trials—a need that CTTI is addressing through its MCT Novel Endpoints Project.

Join us for a special webinar in which CTTI will present new recommendations and tools to support the selection, development, and inclusion of technology-derived novel endpoints in clinical trials.

ADD TO CALENDAR

Topic: Developing novel endpoints generated using mobile technology for use in clinical trials
Date: Monday, June 26, 2017 12:00 – 1:30 PM EDT (GMT-04:00)
Webinar Link: http://bit.ly/2rAJ9K7
Presenters:

  • Martin Landray (University of Oxford)
  • Marc Walton (Janssen)

Panel members sharing their perspectives on adoption of the recommendations:

  • Leonard Sacks (US Food and Drug Administration)
  • Lauren Bataille (Michael J. Fox Foundation)
  • Wendy Snyder (Amgen)
  • Rob Wilson (ActiGraph)

The webinar will include a first look at evidence-based approaches and tools that can be applied by research sponsors, investigators, technology developers, and patient groups to facilitate the use of novel endpoints derived from mobile technology in clinical trials. You will learn about a set of tools developed by CTTI’s expert stakeholders to guide the identification, development and inclusion of appropriate novel endpoints.

Our presenters and panel members will show how:

  • The clinical research enterprise can benefit from the appropriate inclusion of technology-derived novel endpoints in clinical trials
  • Sponsors and investigators can work effectively with regulators to develop high-quality novel endpoints to support regulatory submission and approval
  • Patient groups can both drive the identification of novel endpoints that measure symptoms that matter and participate in their development
  • Tech developers can better match product development to unmet needs and adapt solutions for the demands of clinical trials and the patient care environment
  • Stakeholders can collaborate to avoid common pitfalls in the development process

You will be introduced to four “real-world” use cases that shaped the development of these tools and recommendations, with key lessons to be carried forward to other therapeutic areas. You will also learn how these tools and recommendations can be applied in practice from our panel of early adopters.

This webinar is open to the public. Please feel free to share this invitation with your colleagues.

CTTI Launches New Project to Improve Process for Qualifying Investigators to Conduct Clinical Trials

Posted on by Lorri Bingham

Ineffective or redundant training for investigators wastes time, resources, and does not prepare investigators for the quality conduct of clinical trials. Rather than accepting GCP training as the default solution for qualifying investigators to conduct clinical trials, CTTI is collecting evidence to determine effective and efficient approaches.

While good clinical practice (GCP) training has become the standard for qualifying investigators to conduct clinical trials, little evidence has been collected to determine whether this training is providing the necessary knowledge and skills. CTTI’s new Investigator Qualification Project will evaluate current approaches to investigator qualification, including GCP training, and issue recommendations on effective and efficient methods for investigators to become qualifiehttps://ctti-clinicaltrials.org/projects/investigator-qualification-gcp-trainingd to conduct clinical trials. Investigators, research sponsors, and trial participants are all expected to benefit from an improved investigator qualification process.

CTTI previously released recommendations to address the issue of staff being required to take GCP training before each clinical trial, often multiple times each year. The Investigator Qualification Project is an extension of this work and will focus on the quality of training. CTTI will gather evidence to identify gaps and redundancies in current training, as well as suggest other ways of ensuring that investigators are qualified for the quality conduct of clinical trials.

New CTTI Recommendations Provide Path for More Efficient Clinical Trials Using Clinical Registries

Posted on by Lorri Bingham

Registry Trials

CTTI’s newly released recommendations have the potential to streamline clinical trials by using registry information. Registries are data collection tools typically used to better understand long-term trends in a specific population, such as patients with a particular disease or patients exposed to a certain treatment.

“High-quality registries are an increasingly important source of evidence for regulatory decisions and surveillance, conveying important information, for example, regarding real-world medical product use and outcomes throughout the total product lifecycle,” said John Laschinger, MD, of the U.S. Food and Drug Administration, who helped lead the CTTI work. “Use of high-quality registries as a vehicle for efficient conduct of randomized clinical trials is a proven concept that can be considered for future pre-market trials,” he added.

If registries are designed appropriately, the “real-world” data within them should often be able to meet the expectations of regulatory agencies, and support decisions about medical products. In this regard, CTTI addresses how to assess data quality, ensure patient protections, and link with other data sources. The recommendations apply to both existing and new registries. Following these best practices can assist in evaluating suitability of registry data for regulatory purposes.

“Conducting randomized clinical trials within registries can increase efficiencies in data collection and decrease site workload, potentially leading to significant cost savings,” said Sunil Rao, MD, principal investigator of the successful randomized registry-based clinical trial, SAFE-PCI for Women.

While the scope of this project was limited to registries, many of the principles and tools in these recommendations have the potential to be applied to using health care systems or other existing data sources, such as those available within claims databases, to facilitate more efficient clinical trials.

More information about these recommendations on conducting clinical trials using registries is available both on the Registry Trials Project page and in a webinar recording.

 

*To view the press release of this announcement, click here.

CTTI Publishes Recommendations to Enhance Data Monitoring Committees

Posted on by Lorri Bingham

Improve trial oversight by applying best practices for DMC setup and operation

CTTI has published recommendations to enhance the functioning of data monitoring committees (DMCs) for clinical trials. Sponsors, DMC members, and all those involved in clinical trial design and conduct can apply CTTI’s recommendations to improve DMC operation and the quality of trial oversight, which ultimately benefits patients.

The monitoring of accumulating data in a clinical trial helps to ensure participant safety, as well as the validity and integrity of the trial. For an increasing number of trials, this is accomplished through an independent DMC that can advise on whether to continue, modify, or terminate a trial based on a benefit-risk assessment. Though DMCs play an important role in the oversight of clinical trials, there is considerable variability in how they are composed and operate.

CTTI’s recommendations, which appear in the journal Clinical Trials, address the following areas:

  • Clarifying the role of DMCs
  • Best practices for DMC conduct
  • Effective communication practices
  • Strategies for preparing the next generation of DMC members

The recommendations were developed through a consensus of experts from multiple stakeholder groups after collecting and analyzing data on DMC practices.

Learn more about CTTI’s Data Monitoring Committees Project.

CTTI Points to Stakeholder Engagement, Data-Driven Insights as Keys to Transforming Clinical Trials

Posted on by Lorri Bingham

An editorial appearing in the May 12th edition of Media Planet, a supplement of USA Today, describes the unique methods that CTTI pioneered, and continues to rely on, to transform clinical research.

In Improving Clinical Trials Through Thoughtful Collaboration, Annemarie Forrest, CTTI’s Associate Director of Projects, makes the case that all voices must be included in the conversation if we are going to transform the U.S. clinical trials system. Key to CTTI’s ongoing efforts is the combination of data-driven approaches with the active engagement of all stakeholders, including healthcare providers, regulators, academia, industry, and patients and their families. By fostering dialogue and learning among people who represent a diverse array of backgrounds, knowledge, and experience, CTTI works to enable novel insights about how to accelerate the development of new therapies for the patients and families who need them most.

The tools and recommendations that grow out of CTTI programs and initiatives offer workable, evidence-based solutions. From companies adopting CTTI recommendations to improve the design and conduct of clinical trials to major patient advocacy groups using our tools to streamline trials through their interactions with trial sponsors, CTTI is helping to lead the way toward a better, more efficient clinical trials enterprise.

Find out more about CTTI’s projects and programs here.