Blog: Data Ownership of Patient Registries

Data ownership as leverage in accelerating rare disease drug development? Finally, there’s a cloud software that empowers patient groups!

Feb 3, 2021

By Harsha Rajasimha, Ph.D. & Sharlene Brown, Ph.D.

Patient groups can optimize patient registries, cross-sectional studies, and/or longitudinal natural history studies to maximize collaborations with sponsors of orphan drugs and gene therapies

A disease is considered rare if it affects fewer than 200,000 people in the United States. There are over 8,000 named rare diseases and each year, about 250 new diseases are discovered. Since the enactment of the USA Orphan Drug Act of 1983, there have only been about 600 orphan drugs approved. According to Dr. Christopher Austin, Director of the National Center for Advancing Translational Sciences (NCATS) at the National Institutes of Health(NIH), at the current rate of therapy development, it will be 2,000+ years before every rare disease has an FDA approved treatment–unacceptable. A radical change is needed to increase this timeline by 100X.

Patient advocates spotlight rare diseases and accelerate precision drug development

The 21st Century Cures Act (Cures Act) of 2016 was designed to accelerate the development of curative medical interventions and bring modern innovations to the patients who need them faster and more efficiently. While a majority of rare diseases tend to have a simple genetic basis (e.g. 1 gene mutation), affected patients can present with complex and heterogeneous pathophysiologies (e.g. severity of symptoms, involvement of multiple organ systems, multiple biomarkers, genetic or physiological bases of disease subcategories, etc.). The FDA and NIH emphasize the need for real world evidence from observational studies of patients with rare diseases as important repositories of information to aid therapy development.

Patient registries, cross-sectional studies, and longitudinal natural history studies involve collection of multiple types of clinical outcomes that can be grouped as follows:

  • Outcomes as assessed by a clinician
  • Patient-reported outcomes
  • Caregiver-reported outcomes
  • Performance-based outcomes measured 

These observational studies are typically initiated or sponsored by the NIH, FDA, biopharmaceutical companies, and patient groups. Patient groups like Global Genes, NORD, IndoUSrare, and other disease specific groups have been the driving force of drug development in the rare disease space by advocating for life saving therapies that affect their respective patient populations. Non-profit patient groups are ideal homes for patient registries and natural history databases because they have strong patient relationships that can carry rare disease drug developers through clinical milestones ultimately leading to regulatory approvals (e.g. cystic fibrosis foundation, cureduchenne, etc.).  Although patient foundations protect patient interests at their core, they can lack the expertise and resources necessary to leverage disease understanding and patient relationships when collaborating with sponsors. By creating patient registries or natural history databases, patient groups aggregate valuable data that sponsors can potentially use to: 

  • Identify patient-desired outcomes that influence drug design
  • Inform study protocols and logistics planning
  • Serve as historical control arms for drug trials in patient populations that are too small or ill to support multiple trial arms
  • Identify primary and secondary endpoints for drug trials that are relevant to patient lives and disease modification

Valuable patient data goes uncollected by patient groups and unseen by clinical researchers and drug developers 

Patient advocates may need assistance in one or more of these areas in order to to start and complete such sophisticated patient studies:

  • Defining the vision for all possible uses of the data collected for future analysis towards driving drug development programs.
  • Collaborating with clinical research investigators at academic medical centers or biotechnology companies to design clinically and scientifically sound study protocols, data collection methods, and statistical data analysis plans.
  • Complying with the US federal regulatory guidelines such as 21 CFR part 50 (informed consent) and 21 CFR part 56 (institutional review board IRB review). Depending on the particular use case (e.g., FDA-regulated drug trial), additional requirements such as 21 CFR part 11 may apply.
  • Selecting the right kind of software for such rare disease studies is critical but complex.

Some patient advocacy groups (e.g. in the case of muscular dystrophies) are well funded through philanthropic donations and fundraising programs that allocate the resources needed to hire chief medical officers, researchers, regulatory experts, and technical personnel to drive the data programs. However, most patient advocacy groups have to rely on external advisers and collaborators to move the needle for their rare disease. 

These small population rare disease studies often have tighter budgets and require a more human-centric, empathetic approach to reduce the burden for setup and data collection. Yet, the regulatory compliance requirements still apply and high quality data must still be collected in a secure, scientific manner over an extended period of time (e.g. several months or  years) despite limited resources. Few flexible, scalable, and compliant technology platforms meet these stringent requirements and have been a barrier to more widespread adoption of decentralized or hybrid clinical study protocol designs for small population studies, particularly observational and real world data studies or interventional trials involving single-infusion therapies (e.g. antisense oligonucleotide therapies (ASOs), gene therapies, or biologics) or drug repurposing. 

Compliant, scientifically sound patient data for any group anywhere

The compassionate team at Jeeva listened to 1000+ clinical research stakeholders, most of whom cover the rare disease space,and has now developed a single, easy-to-use web accessible tool that eliminates data collection bottlenecks and simplifies and accelerates the study process. Jeeva’s user-friendly, integrated cloud-based software platform combines best-in-class solutions together into a secure, touchless research station.

To learn more about how Jeeva addresses these and many other costly challenges in clinical research or to take the software for a test drive, schedule your free consultation or demo today. Or request your no-obligation 30-day free trial of the Jeeva software. Check our linktree for the latest deals for your clinical team.

Harsha Rajasimha, MS, Ph.D., is a rare disease social entrepreneur globally recognized for his work in genomics data science, persistent advocacy, and technology innovations addressing grand challenges facing the estimated 350 Million people suffering from rare diseases worldwide. Harsha earned his bachelor’s degree in computer science and engineering from Bangalore University, a master’s degree in computer science, and a doctorate in the interdisciplinary program genetics, bioinformatics, and computational biology from Virginia Tech. He can be reached on LinkedIn.

Sharlene Brown, Ph.D., is director of business development at Jeeva. Brown has over 8 years of experience as a Scientific Consultant and founder of Dr. Bird Consulting, LLC, a boutique firm specializing in the life sciences and ikigai. She is also the Vice-Chair of the Women In Bio – Capital Region. She can be reached on LinkedIn.

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Posted in Blog, Cell and gene therapy, Clinical Research Software, Clinical Trials, Cohort studies, Informational content, Natural history study, Observational study, Patient registry, Precision Medicine, Rare disease, Thought Leadership.

One Comment

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