DIA 2023 – a “patients included” global event

The 2023 Drug Information Association global annual meeting was the ideal setting to put our collective heads together and pool our knowledge and experience, with the hopes of building ideas and innovation that elevate research for patients in need in an ever-changing landscape. 

This year’s meeting was designated as a “patients included” event, emphasizing DIA’s commitment to incorporating patients as experts living with their conditions. Patient perspectives were weaved into sessions throughout DIA’s 13 hot topic tracks, including data and technology, patient engagement, and value and access. Excited to dive deeper into the nuances of their experiences and collectively, as a community, we had an open dialogue about how to push the boundaries of innovative drug development and create actionable change and improved outcomes.

Conversations at DIA covered a variety of tangible solutions to advance patient-focused R&D, including those noted below. 

Critical role of patient-driven insights

Patient-reported insights gathered in a single trial can be robust and extremely valuable to help sponsors amplify their voice into ongoing research, better ensuring secured data points ultimately help make a difference in patient lives. For example, as the US Food and Drug Administration (FDA) and broader oncology community aim to address quality of life challenges, understanding how symptoms are improving or worsening with treatment is crucial. 

When establishing meaningful change thresholds, which are used for clinical outcome assessments to quantify a patient’s meaningful change in symptoms post-treatment, sponsors and study teams are leveraging both quantitative and qualitative approaches to better understand the patient experience. However, how to estimate MCTs and which MCTs are appropriate for the trial at hand can vary and the methodologies can be complex in nature. Once they are determined, sponsors and study teams must consider how to communicate MCT estimates with stakeholders, in order to translate the valued insights and related impact. 

Experts at DIA 2023 discussed combinations of quantitative strategies mixed with qualitative approaches that can help optimise MCT estimations. The primary quantitative strategy includes an anchor-based approach to identify patient thresholds, which requires estimating the change in the evaluated COA based on meaningful changes in another related measure or “anchor” (e.g., a Patient Global Impression of Severity). Anchor-based approaches may also be combined with estimates of the measurement variability on the evaluated COA (distribution-based method) and the results are then reviewed for convergence on a single value or range that represents meaningful within-patient change.

As the FDA recommends, qualitative methods can play a complementary role alongside anchor-based strategies to elicit patients’ perspectives of meaningful change. These approaches include patient-specific cognitive interviews or trial exit interviews that directly ask what scores are meaningful to them and are most effective, instead of simply providing a scale. An example question in an exit interview could be, “Do you feel like the change you have experienced from the start of the trial until now is a meaningful improvement (or worsening) in the nausea you experience?” Though these insights may prove invaluable, expert statisticians must account for how difficult it may be for patients to describe their experiences, especially over time, and results may contradict what the same patients have indicated via quantitative strategies. 

In some cases, patients independent of the trial may be interviewed to provide insights into the symptoms or impacts of interest to them and what level of change in these concepts or on the scale is important to them and could be considered meaningful. However, if we add to the complexities around gauging MCTs qualitatively, those not involved in the trial may find it hard to discuss hypothetical scenarios, and disease severity for these individuals would need to align with the target population. 

In-trial interviews 

Further exploring patient-based interviews in trials, whether through criteria screenings, onboarding, or even when leaving a trial early, can uncover qualitative insights that can contribute to informing clinical assessment strategies, protocol adjustments, and design for later trials. 

Experts at the DIA conference explained how in-trial interviews can be conducted at any time, depending on trial objectives and context, including: 

• At screening, baseline, or pre-treatment stages, insights regarding illness progression and symptom(s) impact, motivation for participation, treatment expectations, and more can help prevent recall bias about pre-treatment experience that could occur if study teams wait to secure the same insights further into the trial. 
• During the trial, understanding how anticipated and unanticipated changes associated with treatment or study participation (positive or negative), including discontinuation, close to the time of occurrence can provide greater depth and rationale for responses to trial measures and study regimens throughout the study duration.
• At trial completion or trial exit, allowing patients and stakeholders to reflect on the overall trial experience (e.g., convenience of study regimen or visits), the full impact of treatment and related symptoms on daily functioning, and other meaningful changes, whether trial and treatment expectations were met and more can supplement quantitative trial results and inform future COA strategies and trials in the same disease area.     

These interviews can be embedded into protocols and trial assessment schedules or conducted later, if deemed beneficial and when coordinated with good principles. Qualitative R&D experts will also share how early trial planning and involvement of key expertise (e.g., COA strategy, clinical operations, health economics and outcomes research, and regulatory affairs) can help to better address scientific and operational issues (e.g., site selection, data confidentiality, and adverse event reporting). 

Multifaceted approach to improving patient-centred R&D  

Whether aiming to provide individuals with digital solutions to participate in trials from home or integrating technologies and expertise that ultimately help accelerate research and much-needed therapies, today’s R&D is laser-focused on improving the patient experience. To successfully elevate patient centricity in current and future trials, a holistic approach to the burdens and growing complexities that various stakeholders face in the process is necessary. 

Considering the site’s perspective 

As patients’ desire to be valuable partners in the trial process grows, their insights are helping to guide trial design and components. By leveraging patient insights via surveys, focus groups, previous trials, and more, sponsors and study teams can weave support services, expertise, and technologies into trial design, in order to ensure sites are better supported from end-to-end as all stakeholders aim to give patients what they need to participate. 

Recruitment and engagement strategies will continue to evolve, and site teams will have to adapt to new or updated technologies and operational processes, while always keeping focus on optimal patient care. It can be a hefty lift for site teams to manage without support. 

A key session at DIA 2023 focused on tangible strategies to help reduce burdens placed on trial sites. Experts shared key solutions to improving recruitment efforts, such as addressing staffing concerns, as well as patient-centric technology integration, training, and ongoing support. Sites may also need support with patient referral, engagement, and onboarding processes. For example, clinical trial educators can educate patients and caregivers about the trial and related therapy, and study coordinators can assist with scheduling visits and other administrative tasks to take time-consuming activities away from overloaded site staff.

Artificial intelligence and process automation  

With more information, technology, and expert stakeholders involved in today’s landscape, sponsors can heighten trial efficiency and quality like never before. Companies are recognising the potential that artificial intelligence and machine learning capabilities and automation may bring to many aspects of R&D. Throughout DIA 2023, AI/ML was the talk of the meeting, especially given the somewhat overnight mainstream awareness of ChatGPT. As it relates to speeding up trial activities that can be time-consuming, tedious, and. in some cases, not manually feasible, R&D technology experts onsite provided an overview of AI/ML-powered solutions and related case study findings for:   

• Fine-tuned enrolment strategies, including benchmarking similar or competitive protocols, recommended country and site inclusion, predictive enrolment and study activation rates, and risk profiles.
• Intelligent data review, where ML models trained on historical data can improve detection of data discrepancies to formulate queries that expert data managers overseeing the process can handle accordingly. Using AI-driven automation with high data volumes, data managers can now shift their focus from highly redundant tasks to more analytical tasks on critical data.

• Optimised risk management, surfacing insights, and signal detection across several critical trial aspects, including:
  • Site risk analytics, helping to de-risk studies by surfacing risk attributes at the site level for trial performance and quality measures.
  • Outlier analysis on labs and vitals data for high accuracy detection of potential data issues and trends.
  • Investigational product compliance insights for accurate detection and categorisation of investigational product deviations, such as loss, overdose/underdose, storage issues, etc.

DIA 2023 was a jam-packed event, focused on collaboration, community, and illumination, aiming to help drive clinical development transformation with patients front and centre in our collective efforts. 

About the authors

Stephanie Rhoten, PhD, associate director, statistics and psychometrics, patient centred solutions, IQVIA
Dr Rhoten is an associate director with patient centred analytics at IQVIA and has more than seven years of experience in quantitative methods and data analytics. Her expertise converges upon statistically assessing clinical trial endpoints and validating clinical outcome assessments for drug labelling. 

Cynthia Verst, president, design and delivery innovation, R&D solutions, IQVIA, and president-elect, DIA Board of Directors 
Dr Verst is responsible for driving innovation and transformation throughout the trial lifecycle. Having previously served as president of both clinical operations and real world and late phase research for IQVIA, Dr Verst brings a holistic and comprehensive view of clinical research, and champions the advanced use of real-world data, analytics, and technology to advance therapies for patients. Recognised by Fierce Biotech as a top woman in biotech, and as a PharmaVoice 100 leader, she currently sits on the Board of Directors for the Association of Clinical Research Organizations and is the Chair of the DIA Board of Directors.

Robert Krupnick, PhD, senior principal, Qualitative Research Center of Excellence lead, patient centred solutions, IQVIA 
Dr Krupnick leads the Qualitative Research Center of Excellence for the patient centred solutions team at IQVIA. As a psychologist with more than 25 years of experience as an insights research and consulting professional, largely focused on strategic pharmaceutical and biotech assignments, he specialises in the application of qualitative research to listening to and amplifying the patient voice, especially as this relates to patient-reported outcomes evaluation, market opportunity assessment, and brand communications development. 

Wendy Morahan, senior director, Clinical Data Analytics Suite, IQVIA Technologies 
Wendy has more than 25 years of diverse life sciences experience in academic research, preclinical drug discovery, and clinical trials, culminating in a focus and passion for delivering technology solutions that help bring treatments to patients faster. She is currently part of the product strategy leadership team for IQVIA Clinical Data Analytics Suite, providing both SaaS solutions for the market and IQVIA’s internal needs. As part of the CDAS team, Wendy is responsible for strategy, product management leadership, and go-to-market activities.