2021 is poised to be a year of epochal change and transformation in the clinical trial ecosystem. Consider the numerous significant events that have occurred in less than six months.
In January, the Clinical Trials Transformation Initiative (CTTI) unveiled its Transforming Trials 2030 vision stating that clinical trials should be patient-centered, easily accessible, designed with a quality approach, and fully integrated into routine healthcare delivery processes.
Source: Verana Health
As with so many aspects of our daily lives, the impact of the COVID-19 pandemic on clinical trials was both widespread and monumental. Thousands of trials—an estimated 80% of non-COVID-19 trials—came to a halt or were interrupted by the pandemic, which challenged the ability to conduct trials safely and effectively and was complicated by the reality that trials often deal with the populations most at risk from COVID-19 exposure.
Source: Hit Consultant
Over a year ago, as the WHO declared the spread of SARS-CoV-2 a pandemic and countries around the world began to feel the impact of COVID-19, hundreds of clinical trials found themselves slowed or temporarily closed as companies and clinics scrambled to reduce the risks to study participants and staff.
Source: Drug Discovery News
With two innovative vaccines beginning to provide protection against the coronavirus for Americans and people around the world, and more on the way, scientists and policy-makers are looking more sharply at the lag in identifying and producing medicines to moderate early infections and to treat seriously ill patients. And the demand for effective therapies has escalated with the emergence of new COVID-19 variants.
Source: Pharmaceutical Executive
If the U.S. hopes to generate substantial, meaningful data on therapies for COVID-19 and future pandemics, it will need to overhaul the clinical trial ecosystem to prioritize master protocols.
The coronavirus disease 2019 (COVID-19) pandemic has introduced changes to patient care, particularly during times of heightened COVID-19 caseloads in different geographic areas. For patients with cancer, the pandemic might have resulted in changes to treatment, a facilitation of disease assessments, and/or a shift of many in-person visits to telehealth platforms.
Source: Cancer Therapy Advisor
The Clinical Trials Transformation Initiative (CTTI) sponsors a Public Summit: The Fastest Path to Effective COVID-19 Treatments: Using Master Protocol Studies.
Source: Trial Site News
Patient behaviors and key biological metrics are significantly easier to track with the multitude of wearables at the disposal of clinical researchers. The most frequently used consumer wearables include ActiGraph, Apple Watch, and Fitbit. By February 2020, clinicaltrials.gov showed that ~460 wearables studies were underway, and, according to Kaiser Associates and Intel, 70% of clinical trials will incorporate some type of wearable sensors by 2025.
Source: Pharma's Almanac
The diversity of patients participating in clinical trials continues to be a problem in drug development. As sponsors and sites attempt to address the problem, regulators are also doing their best to address the issue.
Source: Clinical Leader
The Covid-19 pandemic has brought together regulators, life science companies, health care providers, academic health systems, patient groups, and others to develop safe and effective diagnostics, treatments, and vaccines to address the immediate public health crisis. These efforts have created unprecedented collaboration, flexibility, and speed in starting new, streamlined clinical trials and innovating on the clinical trial process overall.
Source: Advisory Board
In the clinical trials community, nearly every aspect of research is experiencing unprecedented disruptions due to COVID-19, especially to those trials that were mid-stream when the pandemic hit. The Clinical Trials Transformation Initiative (CTTI), a leading public-private partnership, recently held a webinar to help the clinical trials ecosystem adapt to one of the most common challenges - the swift switch to remote and virtual visits, which has left many sites in uncharted territory.
Source: SCRS InSite Journal
An 80,000-patient trial that used an FDA network of data from national health plans found that mailed education information did not increase the use of oral anticoagulants (OACS) among patients with atrial fibrillation (AF) who were not being treated.
The clinical trial enterprise has been due for a reckoning for a long time. The good news is that a fundamental re-thinking of how clinical trials are done – one that could bring lasting positive changes – has started to happen.
Source: Timmerman Report
Sumathi Sivapalasingam, senior medical director at Regeneron, the New York–based biotech, has spent recent weeks, like many Americans, working from home. But even hunkered down in her Brooklyn apartment, Sivapalasingam, an infectious disease specialist and physician who previously worked at Bellevue Hospital and battled outbreaks around the world for the CDC, has felt very much in the thick of the fight against COVID-19.
Scientists and drug companies searching for a coronavirus treatment have launched so many clinical trials that some now fear they will run out of patients to enroll, trial sites or personnel to carry out the tests.
COVID-19 should come as a prompt to the clinical research sector to rapidly reinvent itself as it works to design and launch clinical trials for vaccine and therapeutic candidates “in the teeth of the epidemic,” says Janet Woodcock, director of FDA’s Center for Drug Evaluation and Research.
The urgent need to have at least a few proven COVID-19 therapies approved in the U.S. before the pandemic’s expected resurgence in the fall has become biopharma’s Manhattan Project – a coming together of industry, researchers and government agencies to take on a single global enemy in an effort that could fundamentally change future drug development by introducing more efficient ways of demonstrating safety and efficacy.
Spencer Health Solutions has invited Dr. Pamela Tenaerts to join us on the “People Always, Patients Sometimes” podcast to learn more about an important initiative to improve clinical trials. Dr. Tenaerts is the Executive Director of the Clinical Trial Transformation Initiative known as CTTI.
Source: Spencer Health Solutions
Traditional clinical trials are often expensive, inefficient, include highly selective populations, and can create significant participant burden via travel and other logistical demands. Remote or decentralized clinical trials (DCTs)—those executed through telemedicine, mobile/local healthcare providers (HCPs) and/or mobile technologies that are not bound by geographic limitations—are gaining traction, but adoption has been slow and variable.
Source: Applied Clinical Trials
Sponsors should ‘continually’ think about providing tools to help healthcare professionals discuss clinical trials with women and older people.
Outsourcing-Pharma’s webinar on special patient populations happened this week but you still have the chance to catch-up on the discussions on demand.
At a recent conference, a lawyer explaining blockchain stated: “Technology is advancing faster than it has at any point in history—and it will never move this slowly again.”
Source: Applied Clinical Trials
Getting study teams to consider using readily available data to help design and enroll clinical trials can be more difficult than it should be.
The Clinical Trials Transformation Initiative (CTTI) has unveiled new recommendations for the use of real-world data (RWD) sources to improve the quality and efficiency of clinical trials.
Source: Clinical Trials Arena
The European Medicines Agency and the US Food and Drug Administration have committed to engaging patients in their regulatory processes to promote patient-focused medicinal product development, as well as improve transparency and trust in the regulatory system. Here, we highlight exchanges of experience between the agencies and some impacts on patient engagement.
Source: Nature Reviews Drug Discovery
With technology companies constantly rolling out new mobile apps and wearable devices claiming to monitor, treat and cure various health conditions, strict standards are necessary to make it clear to consumers which of these tools are actually medically valid.
Source: Becker's Hospital Review
“We’re all in the process of learning what [decentralized clinical trials] are about,” Isaac Rodriguez-Chavez, PhD, MHSc, an Officer for the U.S. Food and Drug Administration (FDA) Center for Drug Evaluation and Research in the Clinical Research Methodology, Regulatory Compliance, and Medical Policy Development area, told attendees of the Direct-to-Patient Clinical Trials conference sponsored by CBI in Philadelphia today (August 9).
Source: ACRP Blog
Risk-based monitoring should be described as a “best practice,” says ACRO, which recently released a survey that found the majority of new trial starts in 2018 employed some form of RBM.
The Massachusetts-based Digital Medicine Society (DiMe) launched this week with a strategic advisory board that includes Amazon, as well as top drugmakers like Novartis, and a scientific leadership board including US Food and Drug Administration (FDA) officials.
Through projects such as ADAPTABLE CTTI, and the NIH Collaboratory, the DCRI is defining how to use RWE in clinical research.
From drones delivering blood in Rwanda, to blockchain health records in Estonia it is clear to see that digital health tools have spread worldwide. But how these systems are being implemented varies greatly depending on the government, region and resources.
Source: Mobi Health News
Modernizing clinical trials is an agency wide priority. As more diseases are being redefined based on genomic subtype, researchers have more novel targets and more opportunities to precisely modulate or even repair the basic biological drivers of illness.
It is critical to understand the patient and site perspective to “maximize opportunities and minimize challenges” associated with the use of mobile technology in clinical trials, says CTTI executive director.
The digital transformation of healthcare will see significant growth in the next 12 months fueled by institutional interest in driving down costs and improving patient engagement. Expect increased pharma investment, improving regulatory status, payer engagement with digital tools and better telemedicine connectivity through consolidation.
We believe patients are experts when it comes to their health conditions. Ultimately, they’re the ones who can share aspects of their diseases that even our most qualified doctors and scientists couldn’t possibly know. This valuable patient input is a critical part of our understanding of diseases and conditions that may help us to advance medical product development.
Source: FDA Voice
Hospital-acquired bacterial pneumonia (HABP) is the most common hospital-acquired infection, the most common cause of hospital-onset sepsis, and a highly morbid complication for patients. The estimated mortality rate is between 15% and 30% for both ventilated and nonventilated patients. There is consequently a pressing need for better strategies to prevent and treat this condition.
Source: JAMA Network Open
We entered 2018 with a solid foundation in place for building better, more efficient clinical trials. Throughout the year, progress – particularly in the areas of mobile technologies, real-world evidence, and patient engagement – resulted in new, tangible building blocks for constructing the future of clinical trials.
The Clinical Trials Transformation Initiative (CTTI) recently released recommendations and resources proposing a new approach to investigator qualification - and approach that CTTI Executive Director Pamela Tenaerts said "goes beyond repetitive, identical training, and includes individual experience and protocol-specific preparation."
In a nearly 3,700 word missive by Scott Gottlieb, posted on August 29, 2018, the FDA commissioner attempts to bring some clarity to efforts by the agency to bring more innovation into the drug development process. Noting FDA’s goal of ensuring the right drug or device is delivered to the right patient at the right time, Gottlieb states the vision is moving closer to reality with innovative new products becoming available to pharma companies and its researchers.
Source: Clinical Leader
Our longstanding goal for medical care is to ensure that the right drug or device is delivered to the right patient at the right time. This vision is increasingly possible with the innovative products that are becoming available. Many of these opportunities are enabled by new technology platforms such as digital health, targeted medicines, and regenerative medicine, including cell and gene therapies. These new technologies offer transformative opportunities. But they also challenge the U.S. Food and Drug Administration (FDA) to modernize its approach to evaluating new innovations. In many cases, we’ve had to refashion our regulatory approach to create more modern platforms that are better suited to the efficient evaluation of these advances.
Source: FDA Voice
The clinical research community has long shared an exciting vision of using mobile technologies to collect objective, reliable data in clinical trials. Mobile technologies have the potential to capture more informative real-world data from patients, reduce barriers to trial participation, and lower the costs associated with conducting clinical trials. However, technical, clinical, operational and regulatory challenges have been barriers to widespread adoption of mobile technologies in regulated clinical trials – until now.
CTTI’s latest recommendations will help researchers deploy mobile technologies in clinical trials and “pave the way for improving how we develop medicines,” says Pfizer exec.
A dazzling new array of wearable and other mobile technologies can provide a more complete clinical trial picture, increase efficiency, and reduce the burden on patients, says Ken Skodacek, part of the U.S. Food and Drug Administration’s (FDA) Clinical Trials Program and Payer Communication Task Force at the Center for Devices and Radiological Health (CDRH). However, he also cautioned collecting too much data or flawed data will undermine all those potential gains.
Source: ACRP Blog
The advocates will work to enhance the US FDA’s understanding of how to best engage across patient communities, according to CTTI.
Much of the credit for improving the quality and efficiency of clinical trials in recent years goes to joint reform initiatives and greater disclosure of regulatory decisions and study results. Bioresearch sponsors are listing more studies on the ClinicalTrials.gov website, although the record is weaker for timely disclosure of research results for newly approved medical products. Under pressure to share more research data to avoid repeated errors and waste, biopharma companies also are providing qualified experts with access to confidential studies. And some sponsors are pledging to publish new research reports only in open access journals.
Source: Applied Clinical Trials
Last week CTTI and industry professionals gathered to celebrate the initiative's 10-year anniversary. To learn more about some of the key projects completed and challenges faced over the last decade - and what to expect from the next 10 years - we caught up with CTTI Executive Director Pamela Tenaerts.
Leaders of the biomedical research community recently celebrated progress over the last decade in devising strategies to improve the quality and efficiency of clinical trials. The 10th anniversary of the Clinical Trials Transformative Initiative (CTTI) last week provided an opportunity for FDA officials to join with study sponsors and research experts to examine the policy achievements and plans for future efforts of this multi-faceted initiative.
Source: Applied Clinical Trials
At a symposium marking the Clinical Trials Transformation Initiative’s 10th anniversary, industry and regulatory discussed the progress made by its Quality by Design initiative and the additional work needed to encourage the research industry to be more focused on trial quality and efficiency.
Nearly half of all so-called “one and done” principal investigators who retreat from clinical research after conducting a single study want back in if they can find resources and advice to help on their second attempt, according to findings from a new Clinical Trials Transformation Initiative (CTTI) survey and recommendations project.
Source: ACRP Blog
It can be seriously disruptive when the site investigator for your trial leaves or decides to step down, but new recommendations from the Clinical Trials Transformation Initiative (CTTI) aim to mitigate that risk.
CTTI has released new recommendations that can be implemented by sponsors, CROs, and others, in order to decrease the number of ‘one and done’ clinical trial site investigators.
CTTI's new evidence-based recommendations provide a standard way to plan for and make decisions about pregnancy testing in clinical trials, and also improve communications and transparency with trial participants," Calvert explained.
"Clinical drug development is generally an inefficient process. The cost of conducting clinical trials drives R&D spending, and much of the elaborate superstructure involved needs to be reassessed and could be pared down without harming participants. The EMA actively promotes better design and more efficient trial conduct and supports the efforts of the Clinical Trials Transformation Initiative, created by the Food and Drug Administration and Duke University, and other efforts to streamline trials."
"Some of the first formal efforts to outline the science of patient input borrow, from software development, the use of frameworks to provide a logical structure for organizing information, identifying sources of the information, and suggesting ways it might be used and viewed by distinct parties… The Clinical Trials Transformation Initiative (CTTI) created perhaps the most recognizable tool, and its work has become a guidepost.”
For more information of CTTI's Patient Groups & Clinical Trials Project, click here.
Source: Science Translational Medicine
CTTI is featured as a driver of action-oriented solutions in this Wall Street Journal article that explores the critical need for improved patient recruitment rates. “We want to create trials that everyone involved can champion, and that doctors can feel good about engaging their patients in,” says Jamie Roberts, senior clinical project manager for the CTTI Recruitment Project.
Source: Wall Street Journal
CTTI participated in MediaplanetUSA’s Clinical Trials campaign, where associations and industry experts came together to spotlight the need for more clinical trial participants, to change the public perception and showcase why clinical trials are so important in helping those in need. The campaign was distributed within the centerfold of USA Today on December 18, 2015.
"In recent years there has been an increasing focus on patient engagement, centering on new opportunities for FDA to incorporate the patient perspective into its regulatory decision making. These discussions have resulted in Congress and FDA establishing new policies and programs... However, the public dialogue has largely left out the opportunities for meaningful engagement between patients and research sponsors (academia and the medical product developers). That changed in 2014, when Clinical Trials Transformation Initiative (CTTI) initiated the Patient Groups & Clinical Trials (PGCT) Project. "
Source: FDA Law Blog
On CTTI's Quality by Design work: “Those principles were tested and refined through a series of workshops,” says Glessner. “I can’t speak highly enough about the power of those workshops. They actually involved putting materials into individual’s hands and testing them. Then after a period of time CTTI wanted to know whether the workshops were effective, and to determine that approximately 20 individuals were interviewed. These were deep, detailed interviews asking participants what they were able to take away from the workshops, what they were able to apply in their own businesses, and what problems continued to be a struggle for them.”
Source: Clinical Leader
"In the words of Doctor Hans-Georg Eichler, Senior Medical Officer at the European Medicines Agency (EMA): 'We understand that trials as we do them today are not sustainable, they're expensive, they're inefficient, they take too long-patients complain why they have to wait so long for new medicines-and they're very bureaucratic.' EMA is part of the Clinical Trials Transformation Initiative (CTTI), established by the FDA and Duke University (Durham, USA), that aims to improve the quality and efficiency of trials."
Source: Mobile Health Global
“We've been working [for] the past many years on trying to reform the clinical trial system in both the United States and worldwide because clinical trials are very resource wasteful in a sense… We're also working with the… Clinical Trials Transformation Initiative, to bite off different pieces of the current trial conduct and design and re-design those, and I think we've made significant progress there.”
“We think that there is a lot of merit in supporting improvements in the clinical trial infrastructure itself. The Clinical Trials Transformation Initiative has contributed so much in terms of innovative trial design, as it is working on establishing centralized IRBs for multicentered trials, which is an important advancement.”
Source: Applied Clinical Trials
“FDA’s active participation in partnerships like the Biomarkers Consortium, the Critical Path Initiative, and the Clinical Trials Transformation Initiative is critically important. What has the agency learned from these partnerships, and have officials taken necessary steps to implement new data as broadly and consistently as possible? Much progress remains until efficient trials with flexible designs aided by innovative technologies are no longer the exception to the rule.”
Source: 21st Century Cures