Validated Solutions to Optimize the Future of Clinical Trials

Tangible benefits of decentralized trials, improving inequities in trials, and data-driven drug repurposing.
Several years prior to the pandemic, a limited number of life sciences companies and clinical research organizations dedicated resources and expertise to better address longstanding challenges in trial design and execution. These sponsors and CROs looked at age-old problems in a new way to integrate and gauge how purpose-driven innovations might help resolve specific challenges.

After several years of applying these new solutions, especially as the pandemic brought the targeted challenges to the forefront, we now have the opportunity to reflect on and review the tangible insights we’ve collected as an industry. We can now determine what specific solutions are proving to truly optimize future trials in terms of increasing patient centricity, improving outcomes with quality in mind and reducing inefficiencies.

Decentralized trials: tangible benefits 
Some sponsors and CROs were focused on developing and integrating decentralized trial solutions into trial design and operations years before the pandemic. There is no doubt that during and after the pandemic, more of the industry, including regulatory agencies, has been open to integrating decentralized trial solutions into trial design and operations to increase patient engagement. With increased usage, we now have more insight into how these solutions can actually help sponsors meet trial program goals.

2022 analysis by IQVIA that examined 12 DCT studies across six major regions, several therapeutic areas and indications and in phases I, II and III/IIIb found multiple ways DCT solutions help address challenges in specific trial activities that typically are key factors in timeline delays and added costs, including:

  • Faster study start-up initiation with nearly 50% reduction in time between final protocol and first patient enrolled.
  • Enhanced patient engagement and compliance that can be seen by reductions in recruitment time (-78%), dropout rates (-15%) and protocol deviation rates (-54%).

Thinking beyond timelines and cost, integration of DCT solutions is effectively helping sponsors remove critical barriers that can hinder participation from target patient populations, such as location, level of responsibilities and lack of awareness.

Improving inequities in trials 
As with DCT solutions, the key to enhancing diversity in trials is to tackle this old issue with new ways of thinking and related actions. In April, the U.S. Food and Drug Administration issued draft guidance to help improve representation, calling for sponsors to submit diversity plans earlier (before phase II) and to focus on building a clinical development program in which evidence of clinically meaningful differences, safety and efficacy among subpopulations is closely examined over the trial’s full lifecycle.

In turn, sponsors are taking noticeable strides in creating the change we collectively want to see:

  • As with other trial goals, sponsors are intentionally and proactively setting diversity goals and embedding them into every step, from protocol design to site selection, start-up, recruitment and execution.
  • To help sponsors set evidence-based diversity goals, CROs like IQVIA are focusing on understanding the share of disease burden across various sub-patient populations and the intended treatment population. Leveraging combinations of varying datasets (e.g., published literature, government datasets and electronic health records) with advanced analytics tools and related expertise, sponsors and CROs can better understand diversity and disease burden. However, securing data is not as cut and dry as it may seem because it’s difficult, for example, to correlate patient population to protocol or to how it’s clinically coded in real-world settings. It’s a granular approach, requiring deep expertise to ensure sponsors and CROs are acting on accurate insights.
  • Using COVID-19 vaccine research as an example, foregoing competition and further breaking down information silos among industry stakeholders is helping to make a broader and more long-term impact on bettering trial representation. For examples, sponsors and CRO partners are collaborating with digital social media companies and tech solutions companies to push for innovative ways to reach the right patients and are building relationships with diverse investigators who can be instrumental in helping study teams reach patients where they are.

Data-driven drug repurposing 
The industry is deriving a variety of benefits in drug development by continuously fine-tuning artificial intelligence and machine learning capabilities. Specifically, COVID-19 was a prime example of how rapid exploration of existing anti-viral molecules leveraging AI-based models and related data-driven insights provided a timely and cost-effective solution to successfully repurpose some compounds for virus treatment.

Unlike traditional repurposing approaches that are primarily driven by therapy area and not strength of hypothesis, AI/ML allows sponsors to open up asset potential beyond the boundaries of a particular therapeutic area and related business objectives. AI-based repurposing techniques provide sponsors the opportunity to automate quick and accurate scanning of a vast chemical space to better identify data-backed patterns for potential drug usage in a manner that simply is not feasible through manual processing and analysis. This allows clinicians and scientists to drive assets with increased potential to the top of the list for development and fine tune strategies to determine where investments will best help patients in need.

Automating extraction of insights and evidence from tremendously large collections of data (e.g., electronic medical records, claims data, scientific literature and genomics data) may allow sponsors to:

  • Revive a failed molecule for a different disease target or indication, including course correcting within a live trial.
  • Optimize dosage, formulations or drug administration.
  • Broaden focus to patients in other therapy areas, especially for approved or late-stage drugs with safety profiles available.
  • Bypass early drug discovery stages to potentially allow patients living with rare diseases and difficult-to-treat diseases earlier access to new treatments.

In considering AI-based repurposing, approaches need to be scientifically accurate, which requires guidance by clinical and medical experts. Also, data science experts need to be integrated into the larger development team to help make sure these techniques are applied appropriately to the overall strategy and process.

Turning lessons into best practices 
Thanks to those with foresight to focus on different or new solutions and approaches to optimize trials even prior to the pandemic, we have collected a good amount of key learnings to leverage for future trials as we look toward 2023.

We are seeing the positive impact of relying on innovative thinking, agile strategies and purposeful tech-enabled solutions to address longstanding challenges in successful drug development, which is helping sponsors better meet their goal to enhance patient care.

With more than three decades at IQVIA, Mark Brown, head of Patient Centric Solutions and Decentralized Trials, has touched on several areas of expertise in clinical research, including clinical documentation, data management, system compliance and validation, site selection and management, protocol and patient feasibility and decentralized trial solutions. In addition to leading the Decentralized Clinical Trials team, Mark helps oversee the Patient Recruitment Operations team and Avacare, IQVIA’s U.S.-based site management organization. In 2020, Mark led the creation and launch of a service to support clinical research sites by addressing flexible staffing needs due to dynamic workloads related to large COVID-19 vaccine research trials and related staffing shortages. This led to placement of more than 600 research staff in less than six months.  

Posted in News.

Leave a Reply

Your email address will not be published. Required fields are marked *