Fri, 06/21/2024 - 12:00

Vaccines and Infectious Disease

In the wake of the COVID-19 pandemic, the world has witnessed both the devastating impact of infectious diseases and the remarkable speed at which vaccines can be developed and deployed. This unprecedented crisis has underscored the need for rapid vaccine clinical trials, driven by a combination of factors: maximizing public benefits, ensuring cost-effective clinical development, and securing a competitive edge in the pharmaceutical industry. The COVID-19 experience has demonstrated that accelerated vaccine development is not only possible but also essential for addressing current and future public health threats. Accelerating vaccine clinical trials is crucial for public health and economic stability. Rapidly spreading infectious diseases cause significant morbidity and mortality. Fast-tracking trials can help ensure a vaccine is available quickly, reducing population’s vulnerability and mitigating the spread and impact of the disease.

Beyond health, infectious disease outbreaks severely disrupt economic activities. Recent pandemics and epidemics have led to global economic downturns, with many people losing jobs and businesses closing. Rapid vaccine trials can hasten the end of such disruptions, facilitating a faster return to normalcy. The sooner a vaccine is available, the quicker public confidence is restored, allowing economies to recover and thrive.

Efficiency and cost-effectiveness are crucial in vaccine development to reduce the time and cost for trials. By streamlining clinical trial processes, more vaccines can be evaluated, benefiting more people and overall public health. Utilizing advanced technologies, such as digital health tools and data analytics, can enhance trial efficiency. Efficient trials not only save money but also reduce the time to market, enhancing the return on investment for pharmaceutical companies and facilitating quicker access to prophylactic vaccines for infectious diseases.

For pharmaceutical companies, the commercial benefits of initiating vaccine trials promptly are significant. In a competitive landscape, being the first to market can lead to substantial market share and brand recognition. Selecting and working with the right CRO partner is crucial to achieving accelerated trial timelines without compromising safety and efficacy. Partnering with CROs, particularly those with deep experience in vaccine trials and a rapid, nimble approach to trial operations, can ensure that new vaccines are evaluated and approved quickly, ensuring commercial goals are achieved.

The ability to rapidly develop and distribute a vaccine not only generates immediate revenue but also positions a company as a leader in innovation and responsiveness. This competitive edge can translate into long-term advantages, including increased trust from consumers, investors, and global health organizations. Early market entry allows companies to establish longer-term supply agreements and expand into global markets quickly. Moreover, the ability to provide a timely solution during a public health crisis enhances a company’s reputation, which can translate into increased trust and loyalty from both consumers and investors. This reputational boost can have long-lasting positive effects on the company's overall portfolio and market position.

To execute vaccine trials quickly, it’s essential to focus on startup activities. At VaxTRIALS, drawing from over 40 years of experience across many successful vaccine trials, we have developed a set of best practices to accelerate clinical trial startup timelines. Over the next 6 weeks, we’ll delve into 6 key factors that can accelerate vaccine trial startup timelines, sharing insights and strategies to contribute to a more rapid and effective response to emerging and re-emerging infectious diseases. Embracing rapid clinical trials not only addresses immediate threats but also builds a resilient and profitable framework for future health challenges. The experience of the COVID-19 pandemic has shown us that rapid, effective vaccine development is not only possible but imperative.

Thu, 05/16/2024 - 12:00

Vaccines and Infectious Disease

Alyssa King, Principal Data Manager, and Hannah Neill-Gubitz, Associate Project Leader, from the Emmes Group

Imagine there is a sudden outbreak of a novel disease with no known vaccine or treatment. Or, although a tested vaccine is available, there is not enough supply to effectively counteract the swift spread of the disease. 

Sadly, we can imagine those scenarios only too well as we have seen both in recent years with the COVID-19 pandemic and outbreaks of other infectious diseases, such as mpox (formerly known as monkeypox). 

When an outbreak occurs, urgent action and joint efforts with a trusted and experienced partner is imperative to act quickly and efficiently.

The Emmes Group has contributed to the response of major health crises since the 1980s. Through these experiences, our team has developed rapid response capabilities that allow us to take action urgently and decisively during emergent pandemic situations. 

When time is of the essence, our team implements innovative approaches to expedite study startup. Some recent examples include our involvement with vaccine studies led by government and biopharma companies. 

A recent open-label, Phase II study of a vaccine for mpox was initiated to investigate if a smaller dose from the limited supply of vaccine would be as effective as the traditional dose. For this study, we shortened the startup timeline without compromising quality or patient safety in any of the steps. The Emmes team utilized our established library of forms with a rolling approach to draft and complete as needed – rather than waiting to deliver any forms until all were complete. Taking this approach, we were able to review the protocol and Informed Consent Form (ICF) in one day and finalize the electronic Case Report Forms (eCRF) specifications in less than 10 days. Two days later, we deployed Advantage eClinical, the industry’s first fully unified eClinical platform, with the randomization module and critical safety eCRFs. We continued releasing the remaining eCRFs on a rolling basis throughout the startup period.

By streamlining and innovating processes while pursuing multiple workstreams in tandem, Emmes was able to prepare the study to go live just 12 days after protocol finalization. Following this expedited startup process, enrollment for the first stage of the two-phase study was completed in approximately one month.

When an outbreak of an infectious disease occurs, it may become prevalent in a new demographic group or geographic area. For example, instances of a disease that had primarily been seen in adult populations may suddenly increase among adolescents. These scenarios also require urgent responses. With our wealth of vaccine trial experience and biostatistics expertise, we provide statistical considerations and trial design recommendations to help sponsors select and reach the endpoints needed for these shifts in study populations. In a recent trial, we developed customized demographic reports that were updated three to four times daily to ensure the trial stayed on track with the demographic goals of the sponsor. 

Using a combination of established tools and processes and a proactive approach to customization and innovation, the Emmes team works with speed and flexibility to meet sponsor needs and timelines for vaccine trials. 

Through our individual roles in data and project management for the Emmes Group, we are proud to stand shoulder-to-shoulder with sponsors, clinicians, patients, and healthy volunteers in these critical situations – all working urgently with a common purpose: to make the world a healthier place for us all. 

To learn more about the Emmes Group’s vaccines and infectious disease experience and capabilities, please visit us online at https://www.emmes.com/vaccines-and-infectious-diseases.

Wed, 05/01/2024 - 12:00

Neuroscience

The New Psychedelic Era: Evolving Trials to Unlock their Potential for Patients

Steffanie Wilson, Ph.D., Vice President and Neuroscience Therapeutic Area Lead for the Emmes Group

From ancient times, people have made connections between mind-altering substances and healing. In the 1950s and 1960s, research began to demonstrate that psychedelic drugs (psychoactive substances that cause changes in mood, perception, and some cognitive processes) showed promise in treating addiction and mental health disorders. However, they were included as Schedule 1 drugs in the Controlled Substances Act of 1970, dramatically reducing research into their medical value for many years.  

As our understanding and research processes have evolved, it’s been exciting to see more support and funding for clinical research into the use of psychedelics. To name just a few examples, in 2021, Johns Hopkins University received a multimillion-dollar grant to investigate the use of psilocybin on tobacco addiction. The 2024 National Defense Authorization Act (NDDA) includes funding for psychedelics research involving military service members with post-traumatic stress disorder or traumatic brain injuries. And just this month, the European Union announced PsyPal, the first EU-funded clinical project focused on using psychedelics to assist with mental health treatments. 

Recently, I served as a statistician and corporate oversight for a Phase II clinical trial using psilocybin as an intervention for major depressive disorder (MDD) that yielded encouraging results. As the team shared in the paper “Single-Dose Psilocybin Treatment for Major Depressive Disorder: A Randomized Clinical Trial,” published in the Journal of the American Medical Association in 2023, “Psilocybin treatment was associated with a clinically significant sustained reduction in depressive symptoms and functional disability, without serious adverse events. These findings add to increasing evidence that psilocybin—when administered with psychological support—may hold promise as a novel intervention for MDD.”

Results such as these make me optimistic about the potential of psychedelics to improve the human experience and inspire me to help overcome the distinctive challenges that come with this type of research. After all, trials involving psychedelic substances aren’t the same as traditional clinical trials. For instance, you don’t simply hand someone a pill and send them on their way. It’s difficult to maintain blinding – for the patients and the clinicians – when the intervention being tested is a psychedelic substance. Then there’s the role psychotherapy should play in conjunction with the substance administered. Special care needs to be taken in designing the trial, ensuring the right separation of roles, and providing site staff with the specific training and understanding needed to execute the study.

From just these few examples, it’s clear that trials involving psychedelic substances require specialized expertise, and those interested in this research will want to partner with a CRO that truly understands both the challenges and the solutions. 

With 7+ years of experience supporting larger multi-site clinical trials in the psychedelic space, the Emmes Group understands the big picture and the nuances. We want to use our rare and valuable experience to help sponsors unlock exciting new treatments for patients. 

We've developed relationships with many of the sites who are at the forefront of this research. We understand their infrastructure, capabilities and working styles and have unique insight into site performance specifically in psychedelic clinical trials. We also know that the number of sites with Schedule 1 licenses is limited. Developing and maintaining strong relationships with these in-demand sites is vital, and we make these partnerships a priority. 

Carefully selecting and clearly defining endpoints is critical to the success of all clinical trials and is absolutely key in unlocking the potential of psychedelic treatments. Through the Emmes Group’s experience in psychedelic research as well as our full neuroscience portfolio, which includes work with the National Institute on Drug Abuse (NIDA) and National Institute of Neurological Disorders and Stroke (NINDS), we have an unmatched perspective on endpoint selection. We participate in the generation of consensus-driven recommendations for endpoint selection in neurology and psychiatry applications as part of the National Institutes of Health (NIH) common data element initiative. As a result, we are able to guide sponsors in defining endpoints that are right for their psychedelic clinical trials.

As noted, the patient experience in a psychedelic trial is much different than in a trial testing a more traditional treatment. The Emmes team takes that into account as well as the FDA's guidance series on patient-focused drug development. This guidance places a significant focus on incorporating patient perspective in clinical development programs. We have in-house capabilities to provide full-scope support for qualitative interviews that can be invaluable in psychedelic trials because of the unique and varied experiences people have during their dosing sessions. 

If you attended the Multidisciplinary Association for Psychedelic Studies (MAPS) conference in 2023, you may have experienced firsthand enlightening presentations that paired quantitative evidence with representative quotes and videos telling the stories of individual patient stories, both of their disease experience as well as with the psychedelic intervention itself. The combination of those two sources of data was powerful. Emmes can amplify patient voices in psychedelic research. 

With our unique experience, capabilities and relationships, we can truly help our research partners develop the data to expand our understanding and unlock the future of psychedelic interventions. 

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Additional resources on this topic you may find valuable:

Identifying and Selecting Sites for Psychedelic Clinical Trials

Unlocking the complexities of psychedelic clinical trials and FDA's approach to guidance