Guest Column | November 17, 2020

New Approaches In Patient Tracking For The Rare Disease Sector

By Bhavesh Patel and Connor Bitter, Life Sciences Practice, CRA

Finding Person

While we have seen exponential growth in the effort to develop drugs to treat rare diseases in the 30 years since the Orphan Drug Act was introduced, unique challenges remain. Awareness of rare diseases is often low among stakeholders including physicians, healthcare providers, payers, and even patients themselves; trial protocols and regulatory pathways may be unclear, especially for investigational rare disease drugs that are first-in-class; and data on disease incidence, onset, progression, and burden are often limited.

Given the small patient populations, strategies to identify and track patients can play a central role in the success of the development and commercialization of rare disease therapeutics. Our Life Sciences Practice team at CRA recently reviewed strategies currently used by life sciences companies to support patient identification, recruitment, and tracking and assessed how new technologies such as cloud-based platforms could be leveraged to improve tracking and to better support clinical development and market access efforts.  

Patient Identification: Assessing The Current Landscape

In rare diseases, patient identification efforts are typically led by local team members, including sales representatives, access managers, and country managers, who use several tools and strategies to find and record newly diagnosed or at-risk patients. In this “high touch” approach they leverage relationships with physicians, work to access the limited number of available patient registries, and connect directly with treatment centers to learn about patients who are being diagnosed and treated. These efforts lead to ad-hoc databases that highlight patient dynamics to better understand the target market and lead to refined patient identification strategies; in rare genetic diseases, these databases also are used to explore family histories and founder mutations.

In a loosely organized network of people in the field working to support patient identification, efforts to maintain up-to-date and easily accessible records are often inadequate. Most companies have limited, if any, turnkey patient tracking tools in place. Information about patients is often not updated regularly or readily available in a centralized location. Data across different countries, regions, or platforms may be incompatible with other tracking tools. These issues are compounded by the fact that information about patients may change quickly and unexpectedly. Patients may move or stop treatment; participants in clinical research may drop out or be unenrolled; and patients on treatment may switch to other treatment options or different insurance plans. Such inconsistencies increase the risk that data are inaccurate and that patients are lost or double counted. Further, data entry in standard spreadsheet formats can be cumbersome and time-consuming, presenting potentially significant organizational or validation issues.

The inefficiencies and risks in this process can frustrate stakeholders at all levels, from sales representatives seeking to spend less time on data reporting to company leadership depending on status reports to make often pivotal decisions related to development and commercialization programs. Conversely, many of the most successful and efficient patient tracking technologies and protocols can involve extensive and often large-scale infrastructures and approaches for data collection that require a significant investment of time and resources to implement.

The Need For Enhanced Patient Tracking

Our analysis at CRA indicated that there is a clear need for a more holistic approach to identifying patients and more robust processes in patient tracking to ensure that companies can connect with patients, monitor and maintain relevant information, and then leverage that information as needed to support targeted business strategies. By simplifying the data collection process, streamlining data entry, using a centralized approach, and assigning ownership to specific parts of the patient identification and tracking process, stakeholders across different functions would be well positioned to make better and more informed business decisions. Improved patient tracking would also allow rare disease companies to accomplish several additional mission-critical goals, including:

  • Strengthen efforts in patient recruitment by assessing medical facilities based on tracked patient data to reduce time to treatment initiation and better identify rare disease patients who are eligible to receive investigational therapies or commercially available products.
  • Better understand patient motivations by leveraging closer and more methodical monitoring of rare disease patients to gain insights into the factors that lead to initiation, retention, and discontinuation of treatment.
  • Support other internal and external business objectives by being readily able to share data across company functions to inform initiatives such as incentive programs for sales teams, revenue forecasting, and marketing efforts.

Benefits Of A Cloud-based Patient Tracking Platform

Advances in cloud-based technology can support efforts to consider tailored and more holistic approaches in patient tracking that can efficiently and cost-effectively address many established and emerging challenges in patient identification. There are now cloud-based patient tracking platforms that can be customized for companies and product indications to allow for streamlined data entry and reporting with access across multiple business units or organizations. These platforms can remove the burden of mapping patients across and within markets and provide rare disease companies with a single source of data to inform business decision-making and support internal processes including revenue forecasting, opportunity assessments, and commercial optimization. Cloud-based tools can also significantly reduce the time required for data entry and organization, allowing teams and resources to stay focused on identifying patients and enhancing their access to care.

Streamlined Data Entry

Many rare disease companies agree that one of the major challenges in creating a functional patient tracking system is collecting de-identified patient data from field team members and consolidating that data in a systematic way. Cloud-based platforms can be designed so that data is instantly customizable at entry using a highly intuitive protocol that can result in fewer data entry errors, less time spent on quality control, and the ability to quickly access compliant data. Some of these platforms are also code-free, minimizing costs of training and education for company teams.

Automated Reporting

Another challenge rare disease companies often face is the significant time spent reporting on collected patient data. Cloud-based tracking platforms can automate this process by allowing effective integration of data input from field users through multiple systems and browsers and enabling preselected self-service analytics, both of which are essential to identifying actionable insights. Based on case study examples from different rare disease companies, patient identification yields optimal results only when end users are well positioned and encouraged to generate patient tracking reports on their own with minimal need for IT support. To further support effective patient identification initiatives, cloud-based platforms also help identify exceptions and report anomalies in data that could be easily missed in manual review of unsynchronized spreadsheets.

Data Accessibility

Real-time patient tracking with cloud-based platforms also supports entirely new standards in data accessibility. Platforms can be designed so that all data are available on a single database to streamline access. The flexibility of cloud-based platforms also allows patient data reports to be made available across different functions and data to be securely integrated among aligned systems in a process that is in compliance with General Data Protection Regulation (GDPR) guidelines for countries outside the U.S. and the Health Insurance Portability and Accountability Act (HIPAA) for patient tracking in the U.S.


As more companies initiate and advance clinical development programs targeting treatments for rare diseases, many will need to consider and implement new and previously untried strategies in patient identification and tracking. The adoption and implementation of advanced technologies and tools including cloud-based platforms will be essential to their success. Effective patient tracking tools can help rare disease companies better understand patient needs and motivations, assess disease burden, make accurate projections related to incidence and treatment populations, and enhance data analyses to support strategic business decisions. Cloud-based platforms can help ensure that users in markets around the world are operating with the same foundational knowledge and using similar modes of data entry. More efficient and precise collection of data that is readily accessible can also play a role in both the advancement of new rare disease therapies and relevant services in patient and clinician education and result in optimal levels of care.

About The Authors:

BBhavesh Patel, Ph.D., is a principal in CRA’s Life Sciences Practice. He has worked on numerous global pricing, market access, and commercial strategy engagements and currently leads CRA’s Rare Diseases issues leadership platform.



CConnor Bitter is a consulting associate in CRA’s Life Sciences Practice. He focuses on commercial and medical strategy engagements with a quantitative or analytics-based approach across disease areas, particularly in neuroscience, nephrology, immunology, and rare diseases.

The views expressed herein are the authors’ and not those of CRA or any organizations with which the authors are affiliated.