Optimizing Oncology and Rare Disease Clinical Trials by Focusing on Science and Aligning with both Patients and Investigators

Optimizing Oncology and Rare Disease Clinical Trials by Focusing on Science and Aligning with both Patients and Investigators

May 30, 2024PAO-06-24-CL-09

Ergomed is a full-service clinical contract research organization (CRO) dedicated to providing specialized services to the pharmaceutical industry in support of drug development. With a legacy built on co-development and a commitment to scientific excellence, Ergomed leverages its expansive North American and European footprint to offer tailored, patient-centric solutions specialized in oncology and rare disease clinical research. Underpinned by a highly qualified team, the company thrives on a unique focus on patients, investigators, and sites while pursuing innovations that promise to reshape the future of clinical trials. In this Q&A, Dave Selkirk, President of Ergomed Clinical Research delves into the company’s specialized approach, the nuances of conducting global clinical research, and further clinical transformations on the horizon, in conversation with Pharma's Almanac Editor in Chief David Alvaro, Ph.D.

David Alvaro (DA): To begin, can you share a general overview of Ergomed?

Dave Selkirk (DS): At its core, Ergomed is a CRO partnering with pharmaceutical and biotech companies to manage the clinical development phase of their programs, from the initial stages through post-marketing research.

As a midsize, full-service CRO, we've carved out a niche primarily in oncology and rare disease research, working extensively across North America and Europe. We have a comprehensive grasp of these territories, including the full gamut of Western, Central, and Eastern European regions. We are also significantly expanding into East and South Asia.

What sets us apart is our scientifically driven approach — across the Ergomed Group we have 110 MDs, plus another 542 employees with graduate degrees, bringing a wealth of knowledge to the table. This scientific prowess underpins our advisory role to our clients, ensuring that their projects are poised for success. This advisory aspect is ingrained in our ethos, drawing on a rich co-development history where Ergomed had shared investments and, inherently, successes with our partners. While we've moved away from the economic side of the co-development model, that commitment to our clients' outcomes remains a guiding principle at Ergomed.

DA: Can you walk us through Ergomed's history, from its founding through any other significant milestones that have shaped the company's operations?

DS: Ergomed has a rich history stretching back more than 25 years. Our founder, Dr. Miroslav Reljanovic, is a neurologist and former principal investigator in clinical trials. He instilled a philosophy in Ergomed that remains central to our identity: a focus on the needs of the sites, investigators, and patients. This approach differentiates us within the CRO industry.

Dr. Reljanovic's expertise in neurology laid the foundation for our strength in rare neurological diseases. Over the years, strategic acquisitions have bolstered this expertise. Notably, the acquisition of PSR Group BV in the Netherlands solidified our position in rare disease research, and the purchase of MedSource in North America expanded our reach and presence in oncology. These acquisitions, and Dr. Reljanovic's vision, have been pivotal in enhancing our specialized services in these areas.

In addition to our CRO activities, we also launched PrimeVigilance in 2008, a division focusing on pharmacovigilance. This division has grown to be recognized for its expertise in a broad range of drug safety services including benefit-risk management, safety systems and reporting, medical information services, literature surveillance, pharmacoepidemiology, among others. It is another example of how Ergomed has evolved and adapted over time, continually improving and expanding its service offerings within the pharmaceutical and healthcare industry.

DA: How does that legacy of co-development continue to influence Ergomed’s approach to clinical trials?

DS: Our co-development heritage, where we once invested in and became part owners of the drugs we helped bring to market, has ingrained in us a deep understanding of the perspectives of our biotech partners. We understand the critical importance of adhering to timelines, designing protocols that align with care standards, and collecting outcomes data that will stand up to both regulatory and payor scrutiny, or be valuable in the case of a product sale or out-licensing.

We also appreciate the significance of major milestones within a study. For example, the “first-patient-in” milestone signals to the world that the trial conduct is underway, and it's vital for biotech companies seeking venture capital, as it provides opportunities for press releases, the dissemination of information, and conference presentations. This insight into the unique pressures and expectations of biotech firms is embedded in our culture.

Our approach is consultative — we're inquisitive by nature, and we make it our mission to thoroughly understand our clients' objectives. This enables us to customize our services to fit their specific needs. We're not here to just execute tasks; we strive to be strategic partners who add value by engaging actively and asking the right questions. It's this collaborative ethos that sets us apart and defines us as a CRO.

DA: Could you discuss the impact of digitalization and decentralization on the clinical trials that Ergomed supports?

DS: The pandemic accelerated the shift toward decentralized clinical trials, democratizing participation across the gamut of demographics by potentially allowing anyone with internet access to participate. This shift has broadened the epidemiologic diversity of trials, which historically skewed toward certain socio-economic groups. Now, we can reach a much wider population.

The extent and value of decentralization varies across therapeutic areas. There is not a binary choice between traditional in-person and virtual trial models.  Instead, the key is to find the right balance between the two in a hybrid model. Certain aspects of clinical trials, particularly in oncology and rare diseases, still often require some components of in-person interactions, due to technological and diagnostic necessities, like in-depth imaging or treatments that require specialized training, expertise, or complex administration protocols.

Conversely, many patient-reported outcomes and simpler assessments can now be captured digitally. Patients can self-administer tests like blood pinpricks or provide urine and fecal samples from home.  More complex tests might only need a home visit from a healthcare professional rather than requiring the patient to travel to the clinic.

At Ergomed, given our focus on complex areas like oncology and rare diseases, we advocate for a balanced hybrid approach. We work closely with sponsors to design trials that blend essential in-person assessments at clinical sites or hubs with more straightforward, remote follow-ups. This model maintains efficiency, regulatory compliance, and a focus on both the patient and investigator experiences. With our background in co-development, we fully understand the importance of designing trials that meet regulatory requirements while also being efficient and considerate of the patients’ needs.

DA: Has the hybrid model of clinical trials been particularly beneficial in the context of oncology and rare diseases with geographically dispersed patients?

DS: The extremely low prevalence of certain rare diseases presents unique challenges for clinical trials. Patients afflicted with certain orphan indications may be clustered in specific ethnic groups or regions, while others are broadly dispersed around the globe.  These divergent scenarios make site selection and patient travel logistically challenging. This is especially true for patients with genetic diseases prevalent within isolated populations.

In these scenarios, applying a hybrid model to clinical trials is essential. We must carefully design trials to minimize patient travel — ideally, the patient should only need to travel to a site for the most complex assessments requiring advanced technologies and/or clinician skill. The rest of the trial activities, wherever possible, are conducted remotely through digital platforms such as real-time data exchange tools, wearables, videos calls, text messaging, etc. Wherever possible, it is also vital to incorporate off-site or in-home data collection by qualified healthcare professionals.  This approach not only makes participation feasible for these patients but also respects their time and unique circumstances, making the study design as inclusive and accessible as possible.

The hybrid (decentralized/in-person) model of clinical trials is also highly applicable and beneficial to oncology patients. In this large and broad therapeutic area, treatment therapies can be invasive, cytotoxic, and/or have side effect profiles that are challenging for patients and their caregivers.  Incorporating at least some components of the trial into a decentralized structure can eliminate significant burden from the patients’ care journey.  Improving their quality of life is an extremely powerful way to improve their clinical trial experience, and therefore to increase their level of engagement/participation.

DA: Could you elaborate on the three focus areas of patient-centricity, investigator centricity, and data-driven trial intelligence within Ergomed?

DS: At Ergomed, we've structured a dedicated Strategic Solutions and Patient Centricity group that concentrates on three key axes: patients, investigators, and data. Each axis is spearheaded by a director: we have a Patient Engagement Director, a Clinician Engagement Director, and a Data Intelligence Director, each an expert in their domain.

The Patient Engagement Director works closely with patient advocacy groups, which are especially crucial for successful rare disease studies. This person’s job is to understand the patients' experiences and their journey through the healthcare system, which can be very complex in both oncology and rare disease.  This knowledge is used to inform protocol design and logistics and raise trial awareness in the patient community through the channels established by these groups. This role is pivotal in ensuring that the trials we design are patient-friendly and that participating in the trial doesn't diminish the patients' quality of life. It’s about empathy and logistics; understanding that participation hinges on minimizing the burden posed by the trial, empathizing with the patient and their support system/caregivers, and respecting the patient's perspective.

The Clinician Engagement Director's role mirrors that of the Patient Engagement Director but focusing instead on the clinicians' viewpoints. It’s about grasping the clinicians' perspective when treating these complex disease states, their interactions with other healthcare professionals, and their treatment challenges. This insight ensures the clinical trials are appealing to investigators and aligns as much as possible with their standard of care practices, making it more likely that they will want to participate and recommend the trial to their patients as a viable treatment option. These peer-to-peer interactions can also help identify barriers to care access for patients that need to be considered in designing a strategy for successful study execution.

Lastly, the Data Intelligence Director taps into various data sources, both public resources like GlobalData, Citeline, CT.gov, etc. and more specialized ones like PubMed, peer-reviewed journals, etc. to understand the epidemiology and logistics involved in trial planning. This individual analyzes the distribution of disease and the prevalence of diagnostic tests, offering crucial insights into the number of trial sites needed to execute the study and the aggressive yet achievable time required to recruit patients. It's a comprehensive approach that ensures we’re not only initiating trials but also adapting them as they progress, staying informed through real-time feedback from both patients and clinicians.

DA: Can you expand on the support Ergomed offers for site management and how this role has evolved, especially considering the main concerns and needs of sites?

DS: Ergomed recognizes the increasingly complex demands placed on clinical trial sites, especially with the documentation and administration tasks that have grown since the COVID-19 pandemic. Distinct from our Clinical Research Associates (CRAs) who focus on ensuring protocol compliance, patient safety, and patient data integrity, our Site Management group focuses on supporting the Study Coordinator role at the investigator site. The Site Management team can assist from a distance, or also physically go on-site, managing logistics, mapping procedures, and handling the administrative and planning aspects of trials.

Our Site Management team stands out by offering bespoke support, not by offering only pre-packaged solutions but by listening to Study Coordinators, assessing their needs, and customizing a plan to support them efficiently. This might include ensuring that they have the necessary supplies, managing patient calendars, assisting with data entry, recommending protocol/industry best practices, or aiding with documentation and invoicing processes.

The Study Physician team is an additional level of site support offered by Ergomed, with this service being primarily directed to the Principal and/or Sub-Investigator.  Just as the Site Management group is distinct from the CRA role, so too the Study Physician team is distinct from the Medical Monitor role.  As has been established throughout the CRO industry, Medical Monitors focus on patient safety data ensuring the validity of medical assessments and searching for trends/signals. By contrast, Ergomed’s unique Study Physicians provide a peer-level medical and scientific consultation layer for the site’s medical doctors who treat the trial patients with the investigational therapy. This unique structure allows us to address scientific and medical challenges beyond safety data, underpinning profound protocol understanding, and fostering discussions that enhance patient recruitment and trial success.

This comprehensive, responsive support system of both Site Management and Study Physicians targets the specific needs of both Coordinators and Investigators and differentiates Ergomed from traditional CROs.

DA: How important is Ergomed’s large geographic footprint in helping sponsors to understand the regulatory and cultural nuances across Europe and the United States for their global trials?

DS: Our expansive reach across Europe and North America is a considerable strength. Our presence across these regions' diverse ethnic landscapes mean we have cultivated a deep understanding of a broad array of regulatory environments, along with cultural norms. Our Patient and Clinician Engagement Directors conduct outreach in numerous languages and across different regions, ensuring that we gather region-specific insights.

This extensive network allows us to not just assume a universal response from doctors or patients based on data from one country, but to actively seek and incorporate feedback from multiple regions to tailor our trial plans. This comprehensive approach is invaluable, particularly for global trials where such nuances can significantly impact the success and relevance of the research. Having this large footprint enables us to operate efficiently and sensitively on a global scale, leveraging the best countries and regions based upon therapeutic standards of care recruitment potential and start-up timelines.

DA: What do you see as the primary differences companies face in implementing clinical trials in the United States versus Europe?

DS: While the European Medicines Agency (EMA) governs most European countries and the Food and Drug Administration (FDA) oversees the USA, their regulatory principles and good clinical practices are generally well aligned, despite some differences in wording and nuances.

However, the timelines for clinical trial approvals can vary significantly. In the United States, the use of central ethics committees allows for a quicker IND (Investigational New Drug) approval process, generally enabling faster trial initiation compared with the European Union.

Culturally, North America has a more receptive attitude toward decentralized clinical trials and home healthcare, a sentiment that's gradually being embraced in Western Europe and also more commonly under consideration in Central and Eastern Europe. Technological advancements and increasing exposure from progressive sponsors and CROs are fostering greater acceptance of hybrid and decentralized trials in these regions.

This process is dynamic, and as technology becomes more integrated into everyday life, even regions that have historically been less receptive are beginning to adapt.  We’re seeing a trend toward a more unified global approach to clinical trials, influenced by both regulatory and cultural shifts.

DA: Can you tell us more about the Ergomed team, particularly some of the key individuals and their contributions to the organization?

DS: Ergomed’s strength is significantly derived from its people. Key figures in our team include Dr. Juliet Moritz, our Senior Vice President of Strategic Solutions and Patient Centricity, who, along with her team, embodies the co-development mindset, offering crucial insights to our sponsor partners in oncology and rare disease.

Natalia Grassis, our Executive Vice President of Global Operations, oversees the entire global operational spectrum of our clinical trials. She ensures that each trial is well-staffed, resourced, and equipped, playing a critical role in Ergomed's successful partnerships and trial outcomes.

Dr. Colin Hayward, our Head of Medical Science and a seasoned professional in oncology as well as rare disease, advises clients on protocol design and regulatory matters, contributing to the depth of our understanding of biotech sponsors' priorities.

Lastly, Dr. Nikola Strumberger, our Senior Vice President of Business Development (a Medical Doctor himself) has an extensive global network throughout the pharma and biotech industry.  His medical background brings invaluable expertise when engaging with active and prospective sponsors alike.

Together, these individuals and the entire Ergomed team ensure that our operations are executed with expertise, innovation, and a deep commitment to advancing clinical research to address unmet medical need.

DS: I see several transformative trends on the horizon for clinical research. First, there has been an increasing acknowledgment of breakthrough therapies in North America and Europe, with regulatory systems in place to fast-track their approval. This acceleration allows vital treatments to reach patients sooner, often with the requirement for post-marketing commitments to gather long-term safety data. As a result, we might see a rise in post-marketing research demands, shifting these studies from voluntary branding exercises to rigorous, regulatory-mandated programs.

Decentralized clinical trials are another trend set to continue evolving as technology advances. With innovations such as mobile health applications and wearables, more assessments that previously required in-person clinic visits may shift to digital and point-of-care scenarios. Even medical assessments currently requiring skilled technique, invasive and/or large volume sample collection, or even large and complex equipment, are likely to become increasingly self-administered via advancements in technology and mobility.  These evolutions broaden the epidemiologic diversity of clinical trial populations and augment the relevance of the findings to the full global population.

Additionally, the concept of synthetic control arms is emerging as an exciting development. Instead of traditional control arms with active or placebo comparators, synthetic control arms utilize real-world data from patients receiving standard care. This method, facilitated by electronic medical records, claims data, disease registries and historical clinical trial data, could revolutionize control groups in clinical trials by reducing costs and logistical burdens. While this approach is still in its infancy, the potential for this methodology is significant, and we anticipate that it will play a larger role in years to come. Ergomed is poised to embrace these changes, ensuring that our clinical research methodologies remain at the forefront of innovation.

DA: Are there other ways that you envision technology enabling clinical research to evolve beyond current approaches?

DS: Technology is poised to revolutionize clinical research in ways we've only begun to explore. One intriguing prospect is the ever-advancing technology that allows increasing comprehensive results from decreasing small samples of blood.  If self-phlebotomy (i.e., pin prick for blood spot collection) can yield an adequate blood volume for a broad array of assessments, new possibilities for frequent, event-triggered data collection will become feasible like never before.

Wearables are already a part of this transformation, moving us away from the traditional, discrete timepoint model of data collection. Many of us already wear devices that monitor a wide range of physiological data in real time.  Imagine a future with a wearable device collecting data to a degree of scientific rigor that would permit the data to be included in a clinical trial, and ultimately a regulatory or payor submission. This could offer us a detailed picture of what's happening before, during, and after a clinical event, giving us insights into potential triggers and the effects of different activities or states, such as exercise or sleep, on the patient's health.

While the scope of data currently collected by wearables is somewhat limited, the potential for growth is immense. As these devices become capable of capturing more in-depth and varied health metrics, we can use artificial intelligence (AI) to sift through this vast amount of data to identify trends and signals that were previously undetectable.

Both sponsors and patients are beginning to embrace the potential of wearables, but we're still at the tip of the iceberg in terms of what's possible. With AI's evolving capacity to process vast amounts of data, the future of clinical research could see a significant shift toward a more dynamic, real-time understanding of patient health and disease progression. This approach not only enhances our understanding but could also lead to more personalized and timely interventions.

DA: How do you foresee Ergomed growing and changing over the next few years as the sector evolves?

DS: Ergomed’s commitment to specialization remains unwavering. We’re dedicated to sustaining our high level of medical science expertise, particularly in the fields of oncology and rare disease. These areas are not only extensive but also highly complex, often intertwining with neurological and genetic factors. We consciously choose not to dilute our focus by trying to be a generalist CRO that covers all therapeutic areas; instead, our strength lies in our deep understanding of our specialized fields. We are seeing increasing numbers of sponsors in the USA, Asia, and Europe interacting with us because of our scientific expertise.  It is the ‘rocket fuel’ that drives our strong growth in both the near and long-term.

In line with this commitment to specialization, we aim to grow further as a significant midsize player in the CRO industry, including geographical expansion as part of our growth strategy.  While we have a robust presence in North America and Europe, we’re growing our direct operational presence in both East and South Asian countries. We’ve laid the groundwork with existing offices in Japan and India and intend to further our local Asian presence in the vital country of China.

Our goal is to build upon our existing strong foundations — our knowledge of investigators, care standards, and patient populations — to cement our position as a therapeutically specialized yet geographically expansive CRO. This expansion is aligned with our overall mission to enhance our global impact while staying true to our areas of expertise in oncology and rare disease.

chat button