Accelerating the path to regulatory submission with RWD

The use of real-world data (RWD) in pharmaceutical development is becoming increasingly prevalent and the FDA is encouraging its usage, having released guidance “intended to accelerate medical product development and bring innovations faster and more efficiently to the patients who need them.”¹ 

With the proper tools, RWD can be leveraged throughout the pharmaceutical product life cycle to accelerate regulatory approval, enable commercial success and ensure the right patient receives the right treatment at the right time. In this first of a four-part series, we will delve into the benefits RWD can offer at each stage of the pharmaceutical product life cycle, starting with clinical trials. 

More trial, less error

Drugs developed with the support of patient-centric RWD have shown an 89% likelihood of launch, compared to only a 68% likelihood for drugs developed without leveraging RWD.² Therefore, incorporating RWD from the beginning can lead to increased success. Before a trial even starts, there are several ways that RWD can address and overcome key challenges and expedite the path to regulatory approval:

Inform study design - Inappropriate study design often leads to clinical trial failures. RWD provides researchers with a robust understanding of the representative population to better inform study design, including determining realistic endpoints, sample sizes and inclusion/exclusion criteria for participants. 

Aid in site location - RWD can help pinpoint geographies where a particular condition may be prevalent to increase efficiencies and accelerate trial planning. For example, HealthVerity worked with two top 20 pharmaceutical companies studying RSV, helping them identify ideal clinical trial sites using RWD to determine disease spread. As another example, the Alzheimer’s Association reported that clinical trial sites for memory impairment drugs do not cohesively align with areas that have larger populations of individuals 60 years of age and older, causing strain on sites, delays and performance issues.³ RWD can address this concern, offering insight into the demographics of certain geographies. Additionally, RWD can be leveraged to discover physicians treating patients meeting your specific criteria that could serve as investigators or assist in recruiting efforts.

Discover ideal participants and improve patient recruitment - Clinical trial recruitment is a major challenge and a failure to meet enrollment thresholds can result in delays, cancellations and increased costs. This can be a particular issue when studying rare diseases that often go undiagnosed or under diagnosed. RWD can help you discover patients who have undergone certain procedures or tests indicative of the condition. Additionally, some treatments being studied have very specific requirements, such as cancers with certain gene mutations or biomarkers. RWD can improve the effectiveness and efficiency of trial recruitment strategies by helping you discover cohorts that fit your inclusion and exclusion criteria, as well as locate untapped pools of participants and increase the diversity of subjects.

Enhance enrollment screening and qualification - For participating patients who have provided consent, RWD can facilitate patient screening, validate patient-reported data and provide a deeper view into participants’ health. With RWD, researchers can gain a more comprehensive understanding of the patients’ medical history, including comorbid conditions, their baseline, and whether they meet inclusion or exclusion criteria. RWD can also supplement information about the condition, such as disease onset, trajectory, past healthcare utilization, diagnoses and treatments, and provide insight into the patient journey leading up to randomization within the trial. 

Six fundamental factors for leveraging real-world data

RWD can provide insights to increase clinical trial efficiencies, expediting the trial and increasing the likelihood of success. To continue that trajectory throughout the product life cycle, it is critical to consider these key factors:

1. Accurate patient identity resolution - To follow a clinical trial participant’s real-world healthcare journey, you need to be able to accurately resolve their identity across data sources. High error rates can lead to fragmented views of the patient journey or inaccurate correlations.
   
   
2. Governance of patient consent preferences - Interacting with patients, particularly in clinical trials, requires tracking and adhering to a wide variety of permissions. If not properly managed, violations of these permissions can have significant repercussions at the federal and state level. You need to be able to manage that consent throughout the product life cycle and respond accordingly if the patient’s consent preferences change.
   
   
3. Strict patient privacy and HIPAA compliance - It is imperative that you ensure patient privacy and comply with HIPAA regulations when utilizing RWD. This should be done by securing a third-party Expert Determination that certifies HIPAA compliance.
   
   
4. Data provenance for greater transparency - You want to know where your data comes from, who’s providing it and how it’s generated; not just for your own peace of mind, but to meet FDA guidance.
   
   
5. A broad ecosystem of both identifiable and de-identified data - In order to unlock more of the patient journey and see what you couldn’t see before about a patient, you need a broad ecosystem with a variety of data sources and types, including open and closed medical claims, pharmacy data, lab results, biomarkers, electronic medical records, hospital chargemaster, and consumer data. This can even include identifiable data in certain instances and in this case, ensuring proper HIPAA compliance and data rights management is paramount.
   
   
6. Timely and simple data sourcing and contract administration - To accelerate regulatory approval and speed to market, you don’t want to be bogged down sourcing data, negotiating and managing contracts, securing HIPAA certifications, and managing consent preferences and data usage rights. You want a solution that handles this for you, providing a single contract for multiple sources to get research-ready data faster so you can gather insights sooner.

HealthVerity FLOW offers a fully governed, HIPAA-compliant and 21 CFR Part 11-certified platform to manage all of this for you. This end-to-end solution manages patient identity, governs permissions, and enables on-demand retrieval of HIPAA-compliant data, both de-identified and identifiable, to contextualize patient journeys and drive success throughout the product life cycle.

In the next installment of our four-part series, we will discuss how real-world data can be utilized during a trial to draw insights faster, contextualize findings and expedite regulatory submissions.

Get more trial, less error when HealthVerity FLOW enhances your clinical trials and accelerates the path to regulatory approval:

¹ FDA. Considerations for the Use of Real-World Data and Real-World Evidence To Support Regulatory Decision-Making for Drug and Biological Products. August 2023. https://www.fda.gov/regulatory-information/search-fda-guidance-documents/considerations-use-real-world-data-and-real-world-evidence-support-regulatory-decision-making-drug.

² The Economist. Real-World Data Trials - Summary. https://druginnovation.eiu.com/real-world-data-trials/..

³ Stanton, S.; Dwyer, J.; Ward, N.; Vradenburg, G.; Taylor, J. (2020). Are U.S. clinical trials sites in the right places to meet the demand for the population density of those over 60? Alzheimer's & Dementia, vol. 16, issue 10, December 7, 2020. https://alz-journals.onlinelibrary.wiley.com/doi/10.1002/alz.044244.