Best Practices for Clinical Registries While Leveraging Real World Evidence

Authors:

Sarah W. Lawrence, MS, Senior Clinical Project Manager, Navitas Clinical Research

Yun Lu, PhD, Chief Science and Innovation Officer, Navitas Clinical Research

Andra Matthews, Sr.Clinical Project Manager, Navitas Clinical Research

The Real World Data (RWD) and Real World Evidence (RWE) market is expected to expand consistently at a Compound Annual Growth Rate (CAGR) of 7.4% from 2022 to 2029, with a predicted worth of $1.66 billion by the conclusion of the projected period. RWD and RWE has become a valuable asset for life sciences organizations, including those in the government sectors, disease foundations, pharmaceutical, biotechnology, and medical device sectors. Clinical registries are one approach to amassing RWD and RWE while also promoting healthcare and research collaboration and dissemination. These registries allow for the ongoing collection of clinical data among a specific population, which could potentially provide crucial insights to influence development of new interventions and support clinical decision-making in both research and patient care.

Here we will discuss the key differences between clinical trials and registries and important considerations when creating a registry program.

Difference between Clinical Trials and Registry Programs

The United States Food and Drug Administration (FDA) defines clinical trials as a method to test potential treatments in human volunteers to see whether they should be approved for wider use in the general population. A treatment could be a drug, medical device, or biologic, such as a vaccine, blood product, or gene therapy. Clinical trials are investigational studies that test potential treatments in human volunteers. These studies are interventional and focus on protecting the safety of participants while evaluating the efficacy of drugs, devices, or behavioral interventions. Clinical trials are designed to find new treatments, diagnoses, and preventions for diseases and gain approval for new drugs or devices.

On the other hand, the Agency for Healthcare Research and Quality defines a patient registry as an organized system that uses observational study methods to collect uniform data (clinical and other) to evaluate specified outcomes for a population defined by a particular disease, condition, or exposure, and that serves one or more stated scientific, clinical, or policy purposes. A registry database is a file (or files) derived from the registry. Patient registries can comprise data from clinical standard care practices, patient reported outcomes, or information from electronic medical records. Registries can be used to access richer data that characterize diseases, define a patient population, or provide information on the effectiveness of drugs or devices, among other uses. Therefore, registries are powerful tools for many organizations and functions to collect RWD and RWE and include it along the biopharma value chain.

Understanding the underlying differences between registries and clinical trials helps sharpen focus on value for registry planning. As organizations consider implementation of a new registry program, it is important to consider the unique characteristics of registries, and how these characteristics inform protocol design and strategy, regulatory and ethics considerations, and data collection, data management and eSolution platform. An important first step in securing a registry’s ability to produce meaningful data and results is to gain from lessons from across the industry.

Pitfalls to Avoid When Planning a Registry Program

In an effort to take advantage of RWD and RWE generated by registries, many organizations rush to implement programs without careful consideration of the necessary steps that are required to ensure its success. Given the observational nature of the registry, investigators may underestimate the planning and effort that is required to establish a registry. Here, we outline common pitfalls that organizations should avoid when planning a registry program.

1. Lack of purpose

Organizations that fail to identify the main purpose of their registry program run the risk of losing focus as enrollment increases and more data is captured. This loss of focus may result in collection of poor quality data for the desired outcomes. From an operations perspective, lack of focus can result in cost overages, create greater protocol inconsistencies between sites and patients, and pose an overall threat to the project’s timeline. Without clear purpose or well-defined goals, registries have a higher likelihood of failure and may fall short of expectations.

Organizations should have clear answers to the following questions:

• Why do you want to run a disease clinical outcome registry? 
• What is the need for a patient quality outcome registry?
• What are your primary objectives for a registry?
• What is your study population for a registry?

These and other questions, depending on the area or field of focus, will help to bring increased clarity about the type of data to be collected.

2. Limited Stakeholder Engagement

Registries are more likely to be unsuccessful if they don’t seek engagement with relevant stakeholders from the planning stage. Registries benefit from involved, committed  stakeholders who possess a significant degree of interest in the research or data included in it. These stakeholders can include scientific advisors, patients, their advocates, clinicians, and other groups. Engaging these stakeholders is crucial to provide guidance and direction in developing protocols and planning studies, especially during the initial stages of registry development. Clinical advisors, for instance, can offer valuable input on the tools and assessments used for data collection. During the enrollment and data collection phases, stakeholders can ensure accountability by ensuring the registry achieves its goals and offer feedback on the best practices to follow through the registry life cycle.

3. Poor Operational Sufficiency for Data Collection

Failing to account for the operational and technical skillset necessary to operate a registry program is another common issue. As was outlined, registry programs are very different from clinical trials, and it’s important to consider which operational areas require more or less attention and resources. Deciding how to organize ethics and IRB submissions and what level of clinical monitoring will be assigned are some major considerations. Since quality data is the most important piece of a successful registry program, special attention should be paid to data sources. Data standardization and common data elements are vital to harmonize and integrate multiple data sources from research and EHR/EMR systems. Organizations should carefully examine approaches to data quality control, management approach, and software choice during the planning stages to ensure data integrity.

Four Best Practices for a Successful Registry Program

Organizations with focused, deliberate intent on setting achievable goals and objectives for their registry are well-positioned to satisfy their stakeholders. Establishing regular communication with relevant advisory boards and partnering with teams whose skillsets match up with the registry also ensure that stated deliverables are met. Here are some best practices for a successful registry program.

1. Perform Feasibility and Landscape Analysis

Organizations should have clear objectives and goals to ensure relevant data is collected to meet registry objectives. In addition to assessing feasibility, conducting a landscape analysis will also set the foundation for a successful registry. Identifying existing gaps in the field will reveal strategic areas where an organization should allocate resources and will help identify areas of focus. 

2. Select an Appropriate Registry Platform

Registries utilize data from a wide variety of sources including electronic medical record systems, patient reported outcomes from surveys or clinical assessments, or raw data from imaging studies or genetic testing. Selecting an appropriate platform that can sufficiently support the Registry data is a crucial decision in the planning process. A powerful registry e-Solution platform needs to provide immediate access to all of the data assets, support metadata driven data harmonization and integration, facilitate risk-based monitoring, quality management, medical data review, data reporting and visualization, and submission in one convenient package. Selecting a platform that meets the needs of the registry is one of the most important decisions in the program planning process.

3. Consider The Role of a Registry Coordinating Center

Allocating appropriate personnel to support a registry program will ensure milestones are met and deliverables are fulfilled for all stakeholders. Three key areas of support make up a Registry Coordinating Center, who will maintain all aspects of registry functions and implement any necessary modifications over the course of a program. These areas include registry operation, regulatory support, and data coordination. Registry operations should be led by an experienced project management team that can track spending and monitor project deadlines and deliverables. Proper regulatory support will ensure that the registry is in compliance with appropriate domestic and/or international policies. Partnering with a data team that has the skillset to produce, manage, and maintain rigorous datasets is essential to a Registry’s success. Effective data coordination teams allow organizations to deliver on promises made to Board leaders and stakeholders and are able to pivot appropriately when needed to address unexpected changes or challenges to a project without compromising existing data. 

4. Plan For Sustainability

Finally, all organizations that are planning a registry should consider the program’s sustainability in the long term. This is best achieved by engaging with relevant stakeholders and developing public-private partnerships to finance ongoing activities. Sustainability planning will also include consideration of funding sources in accordance with guidance from The Patient-Centered Outcomes Research Institute (PCORI), the Agency for Healthcare Research and Quality (AHRQ), U.S FDA, and other entities guiding the standards for effective sustainability and  appropriate use and sharing of data. Successful registries with well-planned sustainability measures generate follow-up projects and fruitful partnerships, ensuring the production of long term, meaningful data.

Registries are a powerful tool to use RWD and RWE, provide crucial guidance to impact clinical decision-making in research and patient care. Organizations should be deliberate in setting achievable goals and objectives for their registry program to satisfy stakeholders. Establishing regular communication with relevant stakeholders, including patients, is vital to the success of the registry program.  To make the most of out of developing clinical registries, it is important to have a strategic global capability to enhance benefits, longevity, and relevance to build a key pillar in the pharma value chain. 

References:

1. https://www.meticulousresearch.com/product/real-world-data-market-5297
2. https://effectivehealthcare.ahrq.gov/products/registries-guide-4th-edition/users-guide
3. https://www.fda.gov/drugs/development-approval-process-drugs/conducting-clinical-trials

Author details:

Sarah Lawrence, MS is a research professional with vast experience in research programs and clinical trials. With a background in genetics and public health, her areas of expertise include chronic disease, oncology, and Neurological disorders including Alzheimer’s disease and dementia. She brings her operational and regulatory expertise and support to several non-profit foundations in the execution of registry studies.

Sarah W. Lawrence, MS

Senior Clinical Project Manager

Navitas Clinical Research

11300 Rockville Pike, Suite 500, Rockville, Maryland 20852 USA

Phone: 301-338-0912

Fax: 301-770-4183

Email: slawrence@navitaslifesciences.com

ORCID ID number is: 0009-0009-0677-4887

Yun Lu, PhD has extensive years of experience leading efforts with clinical data, data standard and therapeutic and disease specific common data element (CDE) development and implementation, meta-data driven harmonization, and information technology integration, process development and validation life cycle support. She has supported a wide variety of Phase I- IV clinical trials and disease registries in various therapeutic areas.

Yun Lu, PhD

Chief Science and Innovation Officer

Navitas Clinical Research

11300 Rockville Pike, Suite 500, Rockville, Maryland 20852 USA

Phone: 301-338-0941

Fax: 301-770-4183

Email: ylu@navitaslifesciences.com

Andra Matthews has over numerous years of experience managing clinical project lifecycles from inception to monitoring, through to closing. Her expertise includes disorders of the central nervous system including Alzheimer’s disease and dementia, and vaccine research. She has worked with non-profit foundations to provide operational support to registry studies.

Andra Matthews

Sr.Clinical Project Manager

Navitas Clinical Research

11300 Rockville Pike, Suite 500, Rockville, Maryland 20852 USA

Phone: 301-770-2730

Fax: 301-770-4183

Email: amatthews@navitaslifesciences.com