Blog
November 1, 2021

Integrated patient data – the future of advanced therapies

The cell and gene therapy (CGT) sector is poised for exponential growth — which will come in novel ways that reflect the unique nature of this class of transformative therapeutics. In this last installment of this blog post series, we’ll look at some of the ways that patient-related data can drive growth in CGT, and how a modern approach to data integrations will make that possible.

In CGT, “growth” does not mean variations on a tablet, or standard mixtures of chemotherapy in a bag, where batch sizes and quantities simply increase, along with profit margins. Because CGTs are based on living cells, expansion is coming in the form of different therapeutic technologies and manufacturing processes, additional sources of raw materials based on live human cells, and a variety of therapy administration methods required by starting cell type, the patient’s underlying illness, or both. All of this expansion generates more data from patients and/or donors, and more need to capture it and truly understand it. 

Title: The number of unique advanced therapy products is growing rapidly
Subtitle: Growth in the advanced therapies sector is multi-dimensional. The technology types are evolving, new cell types are being explored, and the gene therapy sector is expanding — generating more patient/donor data and creating an imperative for a new data management paradigm.
[Reference 1]
Title: Record-setting investment levels fuel growth
Subtitle: The potential offered by advanced therapies is fueling excitement and unprecedented levels of funding and growth, putting pressure on the sector to enable patient access at scale.
[Reference 2]

Adoption and uptake of advanced therapies have faced hurdles thus far, with Healthcare Providers (HCPs), patients, and payers each facing points of resistance. Breaking down the barriers to adoption will rely in large part on effectively capturing and utilizing patient/donor data in the short and long term.

  • Patients and HCPs would like more help understanding CGT treatments to make better, more informed decisions. Treatments have not yet been widely accessible outside of select specialized treatment centers, but as developers use data to better understand the technologies and safety profiles, CGTs that can be more broadly delivered will become available. 
  • Patient stakeholder groups play a different role in the advanced therapy value chain as well, often providing  direct feedback, education, and engagement. These groups can be important links between patients and other stakeholders by understanding and educating patients and stakeholders and helping to ensure access to treatments for patients. Their role may also include organizing the collection of patient data and providing ready-to-use data sets. 
  • Payers have a keen interest in the treatment and efficacy of CGTs on a per patient basis because they are concerned with understanding the value of these high-priced therapies and developing reimbursement strategies.
Title: All the journeys are interconnected — the patient/donor + product + reimbursement
Subtitle: The three journeys must be well understood and established to work together to ensure timely access for treatment to patients.

Integrations are essential for  success

Scaling and industrialization are paramount for reducing risk and increasing the success of patient and product journeys and expanding patient access to these life-changing therapies as they become available. Today, capturing, consolidating, and utilizing the hundreds of patient-related data points spread out across the value chain is expensive and time consuming, and not always handled effectively. An extensive amount of knowledge and efficiency is left untapped. CGT requires a modern approach to data management and integrations as advanced as its patient-centric paradigm — automated workflows, API-based connectivity, and secure, plug-and-play system integrations.

Read our recent whitepaper, “CMC obstacles in CGT: Four solutions to solve six challenges” to learn more.

As discussed in prior parts of this blog series, in CGT, patient/donor data is different. It’s mission-critical, at multiple steps in the manufacturing and delivery process. Patient/donor data is an essential workflow enabler for workflows that happen across multiple locations and users, meaning patient data is spread out across systems — extending far beyond the traditional GMP environment.

High-quality patient/donor data and information is much more difficult to capture with the complexity, interactive, and variable nature of the value chain. Success in CGT requires integrating multiple external partners and stakeholders throughout the value chain — and passing patient-and/or donor-related data back and forth along the way. The comprehensive and transparent view provided by an end-to-end integrated value chain gives stakeholders the accurate information needed to focus on the patient and the patient’s safety.

Title: The FDA and EMA encourage modern, real-time data management for patient safety
Subtitle: Real-time access to patient data during clinical trials is important for all drug products, and even more so for CGTs. The patient and donor data remain critical many years after products are approved and patients are treated.
[Reference 3]
Title: The FDA and EMA encourage modern, real-time data management for patient safety
Subtitle: Real-time access to patient data during clinical trials is important for all drug products, and even more so for CGTs. The patient and donor data remain critical many years after products are approved and patients are treated.
[Reference 4]

In addition, privacy and security must be maintained at every step. The Life Sciences industry is at high risk for breaches and operates under strict regulatory guidelines. Protecting patient-related data and securing how it is stored and transmitted is critical for the CGT industry’s success. Secure systems, and system integrations, for patient/donor data and compliance with regulations such as HIPAA, GDPR, and Title 21 CFR Part 11 are mandatory aspects of any integration methodology.

Title: Patient data in CGT is protected by multiple regulations and standards
Subtitle: A variety of regulatory agencies play a role in the protection of patient/donor data. How it is stored, who has access, and the security with which it is transmitted are all important considerations for CGTs.

A modern, secure approach to integrations will greatly simplify workflows, security, updates, and compliance and improve speed, patient safety, and patient data privacy. In order to industrialize and support the scaling that is coming, integration cannot depend on custom, one-off approaches or on-premise systems. Instead, purpose-built, API-based, flexible, cloud-based systems are needed to link all aspects of the ecosystem around its center – the patient. A plug-and-play, API-based approach to integrations will facilitate access to patient/donor data in the many places the data resides and ensure that it can be utilized to deliver and improve safety, efficacy, and adoption.

Title: Patient data drives the cell and gene therapy value chain
Subtitle: Patient and donor data flows through every part of the interdependent CGT value chain and security and privacy are critical.
Title: Data security and privacy require constant vigilance in Life Sciences
Subtitle: Patient data is high value and makes the Life Sciences industry a desirable target for cybercriminals. Secure, modern integrations at all points in the value chain are needed to protect patient data.
 [Reference 5]
Title: Data security and privacy require constant vigilance in Life Sciences
Subtitle: Patient data is high value and makes the Life Sciences industry a desirable target for cybercriminals. Secure, modern integrations at all points in the value chain are needed to protect patient data.
[Reference 6]

Truly harnessing the power of data in the CGT sector relies on modern, secure data management and connectivity practices across an integrated ecosystem. High-quality, accessible patient-related data will provide the information needed to build the advanced therapies value story, increasing understanding and patient access to CGTs. An integrated ecosystem will provide the ability for the sector to build on its successes, enabling scaling and industrialization, ultimately bringing life-changing therapies to more and more patients in need.

Blog #1 – Patient data – the unique role in cell and gene therapies (CGTs)
Blog #2 – Patient data – why it is critical for cell and gene therapy stakeholders
Blog #3 –  Patient data – unique challenges, critical uses in the cell therapy value chain

References

  1. Evaluate Pharma, Vineti proprietary database. 2021.
  2. Alliance for Regenerative Medicine. Regenerative Medicine in 2021: A Year of Firsts and Records. H1 2021.
  3. U.S. Department of Health and Human Services, Food and Drug Administration, Center for Drug Evaluation and Research (CDER) and Center for Biologics Evaluation and Research (CBER) and Oncology Center of Excellence (OCE). Expansion Cohorts: Use in First-in-Human Clinical Trials to Expedite Development of Oncology Drugs and Biologics: Draft Guidance for Industry. Silver Spring, MD. The FDA. August 2018.: https://www.fda.gov/media/115172/download
  4. U.S. Department of Health and Human Services, Food and Drug Administration, Center for Biologics Evaluation and Research. Chemistry, Manufacturing, and Control (CMC) Information for Human Gene Therapy Investigational New Drug Applications (INDs): Guidance for Industry. Silver Spring, MD. The FDA. January 2020.: https://www.fda.gov/media/113760/download
  5. Evans, Melanie and McMillan, Robert. Cyberattacks Cost Hospitals Millions During Covid-19. Wall Street Journal 2021 February 26: https://www.wsj.com/articles/cyberattacks-cost-hospitals-millions-during-covid-19-11614346713
  6. Mallempati, Raj. How the Pharmaceutical Industry Can Secure Networks to Avoid Cyberattacks. Forbes 2021 March 18: https://www.forbes.com/sites/forbesbusinesscouncil/2021/03/18/how-the-pharmaceutical-industry-can-secure-networks-to-avoid-cyberattacks/?sh=1d93015f1eb3