Building Inclusive Science: The Vital Role of Diversity, Equity and Inclusion in Clinical Research

Diversity, equity and inclusion (DEI) have become important elements in the landscape of clinical research. It is important for the principles of DEI to be understood for their goals, intended outcomes and applications; otherwise, they may be regarded as mere buzzwords or just another box to tick on organizational mandates.

DEI are critical components of building a thriving, fair, all-encompassing, respectful and innovative society. By understanding and actively promoting DEI, individuals and organizations can help create environments where everyone has the opportunity to succeed regardless of their background or identity by overcoming and dismantling systemic discriminatory barriers.

Implementing DEI principles is not without challenges. It requires ongoing commitment, education and sometimes difficult conversations. There can be resistance to change, and it may take time to dismantle long-standing biases and systemic barriers.

Building DEI within life science organizations, particularly in the area of clinical research, is not just a social imperative but also a scientific one, ensuring that drugs developed are both safe and effective for the populations they are intended to serve.

To shed greater light on incorporating DEI practices and plans in clinical research, Xtalks spoke with Stacy Weil, DHI, Senior Vice President, Business Operations and Patient Centricity.

Patient Recruitment and Engagement in Neuroscience Studies

Clinical research into neurological diseases is often hindered by a myriad of patient related problems. What can be done to overcome these issues and improve the efficacy of neuroscience trials?

In the wake of the pandemic, new opportunities and challenges for neuroscience clinical trials are emerging. After years of slow progress, mental health is now the focus of major fundraising and legislative efforts. In the US, for instance, National Institutes of Health (NIH) support rose from $6,460 million in 2016 to $10,967 million in 2022 and Congress recently proposed an FDA neuroscience Centre of Excellence.

A literature data analysis of global trends in neuroscience research through 2019 demonstrates an accelerating trend inneuroscience progress leading up to the pandemic. Now, Alzheimer’s and major depressive disorder studies are seeing a resurgence, while new types of medications are under clinical investigation for disorders such as schizophrenia.

However, myriad factors are making patient recruitment and engagement more complicated for these studies. Geopolitical disruptions and pandemic outbreaks are rendering previously fruitful geographies inaccessible. Outreach for enrolment and engagement of younger people requires a different approach than traditionally successful methods. High inflation and gas prices discourage patients from travelling to distant investigative sites. Meanwhile, increased expectations for inclusion, as in the FDA guidance on enhancing diversity in clinical trial populations, requires inventiveapproaches for finding and retaining study patients.

We all want a silver bullet: unfortunately, the varied and complex issues preclude a simple solution. Nevertheless, with patience and the right approach, these studies can be completed. This paper discusses the roadblocks and considerations for tackling patient recruitment and engagement for neuroscience and other difficult-to-enroll studies in today’s research climate.

Pharmaceutical Outsourcing | Registries and Natural History Studies in Rare Disease Drug Development

Increasing emphasis on rare diseases has led to a greater need to accurately define the profile, characteristics, and outcomes of patients living with those conditions. The development of safe and effective treatments of rare diseases requires a deep understanding of the disease of interest, including its frequency, clinical features, risk factors, outcomes, burden, and evolution. Unfortunately, for most rare diseases, this information may not be available. Observational studies — including both patient registries and natural history studies — help address this challenge by providing insight into not just the disease but also the endpoints and outcome measures that will be relevant and meaningful to patients and other key stakeholders in clinical trials.

Issues Faced in Rare Disease Development

In rare diseases, patient populations are inherently small and geographically dispersed. Due to the low incidence and prevalence of these conditions, diagnosis may be delayed, and data collection may be scarce, incomplete, or inconsistent. Understanding the disease is also complicated by:

Disease heterogeneity, including multiple and atypical phenotypes
Lack of clinical experience and expert guidelines
Absence of validated methods for assessing disease-specific conditions
Lack of natural history data

Observational studies play a critical role in filling these gaps in disease knowledge and informing the design of interventional studies to support successful drug development.

Observational Study Types

In clinical trials, patients receive interventions according to a pre-defined protocol with the objective of improving outcomes. In contrast, the overarching goal in observational studies is to observe natural relationships between disease characteristics and outcomes. Patients may receive but are not assigned to specific interventions.

International Clinical Trials | Maximizing Strategic Development for Digital Medicines and Therapeutics

The realm of digital medicine is one that is always moving. As regulatory agencies try to keep up with the changing landscape, how can companies get the best out of new digital medicines and therapeutics?

Premier Research Named Partner of Choice

Premier Research has been cited in a Clinical Leader article as the CRO partner of choice for Daré Bioscience. The article, “How To Build A Strong CRO Partnership And Find Drug Development Success,” explores criteria to keep in mind when selecting the ideal CRO partner and how a positive sponsor-CRO partnership led to success in a recent women’s health clinical trial.

Read the full Clinical Leader article here

PM360 | New Developments in Gynecological Cancer Research

Substantial unmet needs exist in the diagnosis and treatment of gynecologic cancers. Often, these tumors are detected in later stages when treatment options are limited, and prognosis is poor. However, the gynecologic cancer space is on the cusp of change. The recent introduction of novel medicines, namely targeted therapies, has improved patient care and outcome. This progress—along with increased focus on women’s health, clinical trial diversity, and health equity—has renewed interest in developing gynecologic cancer treatments.

Brief Background on Gynecologic Cancers

Gynecologic cancers comprise five major types: ovarian, uterine and endometrial, cervical, vaginal, and vulvar. All biological females are at risk for gynecologic cancers, and this risk increases with age. Other risk factors include:

Family history
History of human papillomavirus (HPV) infection, for cervical cancer
Genetic mutations such as BRCA1 and BRCA2, for ovarian cancer
Hormone replacement therapy

Awareness, education, and screening, if available, play important roles in prevention, but gynecologic cancers continue to be a major cause of morbidity and mortality worldwide.

Pharmaceutical Commerce: Empowering Patients in Oncology Trials

Patient engagement is critical to the success of oncology clinical trials, many of which have endpoints that require study participants to be followed for many years to evaluate side effects and long-term survival data. And yet, sponsors often postpone careful consideration of strategies for patient engagement and retention until after the treatment phase of their studies. Consequently, patients may be lost to follow-up, or protocol amendments may be required to increase retention, adding cost, delaying timelines, or even adversely impacting data quality. Another risk frequently overlooked is the potential for patients to perceive study participation as a negative experience, which can make recruitment for future clinical trials even more challenging.

Integrating patient perspectives

Patient engagement is valuable not only for gathering protocol-specific insights, but also for informing the overall recruitment and retention strategy. Combining data with a deep understanding of the patient perspective is crucial in oncology studies, since each patient has a unique experience with their disease and their own approach to coping with their condition, both physically and psychologically. Understanding these nuances makes it easier for sponsors to design clinical trials that will be accepted by potential patients as a viable treatment option.

Sponsors should consider engaging with not just patients, but also family members, caregivers, patient advocates, and advocacy groups. Useful insight to gather include disease impact, current treatment, unmet needs, barriers to adherence, and points in the care journey where patients are most likely to consider investigative treatments.

PharmaLive | Advancing Gene Therapy Development: Key Regulatory and Clinical Trial Considerations

Insights into the human genome have created myriad therapeutic opportunities for previously untreatable diseases. Gene therapy offers promise for addressing unmet medical needs across therapeutic areas and even the potential for curing certain genetically based conditions. As research tools and technology become more sophisticated, gene therapy development is accelerating at an unprecedented pace. According to the American Society of Gene & Cell Therapy, there are currently 19 gene therapies, including genetically modified cell therapies, approved for clinical use globally. Nearly 1,900 gene therapies are in development, of which 34 are in Phase 3 or pre-registration trials.

Gene therapy development is a complex endeavor, with evolving regulations and complicated study logistics. In this article, we explore the regulatory and clinical trial landscape for gene therapy trials and offer strategies for successfully negotiating the challenges of executing these studies.

Pharmaceutical Outsourcing: Decentralized Clinical Trials

Patient centricity—also known as patient-centered care—has been a growing force across healthcare. Like the consumer-driven change in other industries, patient centricity upends established models. Whereas traditionally, healthcare professionals dictate care decisions with little consideration for the patient’s desires, patient-centric care puts the patient first. Its tenets include:

• Empowering patients through technology

• Reducing inconvenience

• Designing protocols with patient associations

• Always making patients feel valued

In 2020, when Covid-related patient-safety concerns accelerated demand for decentralized clinical trials (DCTs), investigators found a surprising side benefit: DCTs are inherently patient-centered.

Now patients themselves are driving the demand for DCTs, and DCTs are being shaped to reinforce patient centricity further.

DCTs: The Shape of the Modern Clinical Trial

For over a decade, growing reliance on technology to manage disparate aspects of clinical trials has created an analogous growth in freedom from trial sites. Various e-tools have been incorporated for the convenience of sponsors and investigators. Electronic health records and eConsent forms can be accessed by staff anywhere. Centralized systems simplify data aggregation, monitoring, and analytics. Whether from ePro devices, wearables, or home testing, real-world information has opened rich new data streams while reducing overall trial costs. Still, uptake in the historically risk-averse healthcare industry was slow until COVID-19 forced broad-based changes in clinical trial deployment.

Clinical Researcher: Preparing for the Future

In July 2021, the Industry Standard Research (ISR) Report on Hybrid/Virtual/Decentralized Clinical Trials Market Outlook surveyed 109 industry leaders worldwide who had been involved in DCTs over the past year.{1} Respondents anticipated a 12% increase in hybrid trials over the next two years—and predicted that DCTs would outstrip traditional trial models within three years. They praised the increased ease of patient recruitment and improved patient compliance that DCTs generate. They were also impressed with the access to rich data—often sampled multiple times a day—representing a trove not possible with traditional trials.

Yet that does not mean all is perfect. DCTs—including for our purposes the range of hybrid onsite/offsite, siteless, remote, and virtual trials, depending on your favorite terminology—rely heavily on technology for data capture, and immature technology can pose problems. Therefore it is critical that sponsors choose a contract research organization (CRO) with the specialized experience to foresee and forestall this new breed of potential issues.

From a CRO’s standpoint, decentralization is not revolutionary. Technologies used for electronic patient-reported outcome (ePRO) collection and electronic informed consent (eConsent), just to name a few, are longstanding facets of trial management, and other technologies have steadily gained broad-based acceptance and popularity. Further, as wearable technologies and home monitoring devices become standard accessories for the health-conscious, their data gathering in clinical trials seems increasingly natural.

These devices are also producing better results. The rising popularity of DCTs is based primarily on their ability to better support patients—saving them time and out-of-pocket costs while minimizing their exposure to outside pathogens. That increased support has led to improved compliance and better data, which are, after all, the holy grail of any trial. Yet the sheer volume of data produced is one of the key challenges created by the surge in wearables. Data arrive day by day—sometimes minute by minute—often from multiple devices. Accurately collecting, managing, and analyzing all these data can be overwhelming. Yet, those processes are also critical to trial success—adding pressure to the task of choosing a CRO wisely.

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