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Precision medicine has the potential to transform how we understand and treat autoimmune and inflammatory diseases, but only if we can align innovation with infrastructure.
Here, we outline a strategic framework designed to help pharma and biotech leaders navigate the evolution of autoimmune and inflammatory disease treatment. From integrated data ecosystems and cross-disciplinary partnerships to smarter clinical trial design and equitable access models, we explore the foundational steps needed to bring precision medicine into real-world care for these complex immune conditions.
Strategic framework for innovation
In this framework, we lay out key actions for pharma and biotech leaders to power precision medicine in an autoimmune and inflammatory disease context, as well as address strategic cornerstones which will enable teams to overcome the inherent challenges and complexities in these disease areas.
Integrated data ecosystems
Building platforms which enable comprehensive data integration will be key for enabling new insights into the biological and genetic mechanisms behind autoimmune and inflammatory conditions. Developing systems which consolidate clinical, genomic, and proteomic data to enable researchers and clinicians to address the heterogeneity of these conditions and garner greater understanding of these complex conditions.
Incorporating diverse data types, including environmental, occupational, ancestry, population, and behavioral data, as well as EHRs and omics data will be key to building comprehensive patient profiles and understanding how environmental and phenotypic variables interact with genetic factors.
To ensure safe, secure data sharing across industries and organizations, the establishment of universal data sharing guidelines, such as the FAIR Guiding Principles, will be key to continued, successful innovation. The FAIR Guiding Principles for scientific data management and stewardship were published in 2016 with the aim of improving the Findability, Accessibility, Interoperability, and Reuse of scientific data. These principles emphasize the need for computational systems to be able to find, access, interoperate, and reuse data with minimal human input. Collaboratively abiding by such principles will not only enable clinicians and researchers to make huge strides in seamless interoperability and data sharing, but empower them to fully harness the insights contained within complex autoimmune and inflammatory disease data. Adherence to unified stratification and cross-organizational sharing principles will therefore be pivotal to successful international collaboration and accelerated, cost-effective delivery of new precision medicine treatments and personalized patient care.
Cross-disciplinary collaborations
One of the most vital aspects of success in precision medicine for autoimmune and inflammatory diseases will be the implementation of cross-industry collaborations. In order for unified data sharing and stratification principles to be implemented effectively, multidisciplinary partnerships and collaboration across academia, industry and healthcare institutions will be necessary to accelerate development of new treatments, as well as ensure that patients are direct beneficiaries of research outcomes.
In 2021, a consortium of 11 cross-industry organizations including Sanofi, Ariana Pharmaceuticals, The Imagine Institute, and Sorbonne University launched the ATRACTion program. Funded with €9.9 million over 5 years, the project aims to accelerate the development of precision medicine in autoimmune and inflammatory diseases associated with primary immune deficiencies. One of the project’s primary goals is to develop AI-driven therapeutic decision support software to help healthcare providers make decisions around diagnostics and treatment options, as well as to develop new therapeutic strategies.
Additionally, the PARADISE project, launched in 2022, is a consortium formed of multidisciplinary experts from across seven European countries. The project aims to develop a personalized predictive tool that can accurately assess an individual’s immune system activation and predict risk of autoimmune disease relapse. In addition to harnessing new AI tools, the project is placing patient involvement at its heart and ensuring that the new tool aligns with needs and expectations by actively involving patients in its development process.
Projects such as these provide a blueprint for how cross-industry collaboration and expertise sharing has transformative potential for accelerating precision medicine development. Continuing to build innovation hubs and international consortia will therefore prove vital to the future success of precision medicine efforts in autoimmune and inflammatory diseases and the delivery of improved care to patients.
Alongside this, providing training to support the sharing of cross-disciplinary expertise will be key to the success of collaborative efforts, providing researchers, software developers and clinicians with the understanding they need to fit together the pieces of the precision medicine puzzle. For example, the University of Toronto runs a “Collaborative Specialization in Next-Generation Precision Medicine” training program which aims to support the development of novel therapeutic strategies through the integration of principles and methods from across life sciences and engineering.
Advanced regulatory engagement
In such a newly developing field, it’s important to foster a culture of proactive dialogue with regulatory agencies. Engaging early with regulatory bodies in the development and trial design process will help to align on innovative study experiences which leverage the latest technologies, as well as ensure that studies are utilizing approval pathways which help to support and accelerate development of precision therapies.
Leveraging regulatory frameworks that support expedited approvals for therapies targeting autoimmune and inflammatory diseases is key to accelerating precision medicine efforts in this area and ensuring that breakthrough treatments are reaching patients faster - without compromising on safety. Utilizing pathways such as the US Food and Drug Administration’s Regenerative Medicine Advanced Therapy (RMAT) and Fast Track designations, or Europe’s Priority Medicines (PRIME) Scheme, can help to accelerate review and approval processes, reducing the time and cost needed to deliver breakthrough treatments to patients currently living with high unmet need.
Health economics and outcomes research (HEOR)
With the cost of developing and bringing a precision medicine therapy to market estimated to be approximately $4.4 billion (in the more advanced oncology context), it’s vital that cost-effective strategies are integrated into treatment development at the earliest possible stage. Not only do cost savings serve to benefit the organizations and research teams developing the therapies, but ultimately make breakthrough treatments more accessible to healthcare services and individual patients. An economical approach is therefore vital to securing equity of access and ensuring that treatment is accessible to those most in need.
Early integration of cost-effectiveness
While highly relevant to all condition areas, cost-effectiveness in developing precision therapies for autoimmune and inflammatory diseases is particularly important due to the diversity and heterogeneity of these conditions. Integrating a Precision Health Economics and Outcomes Research (P-HEOR) model helps to combine economic and clinical value by identifying distinct patient subgroups based on how individuals interact with and utilize healthcare services. Using this type of machine learning (ML) model to understand patient behaviors and needs can help researchers and teams assess the economic value of treatment regimens and identify key areas of unmet patient need. This ensures that a proposed study is addressing a treatment pathway which is both economically sound and actively responding to patient needs.
By using ML and incorporating big data and predictive analytics, researchers can create more nuanced value propositions for new interventions, tailoring them to specific patient clusters and providing clear evidence of cost-effectiveness.
Utilizing real-world evidence
Real-world evidence (RWE) plays a crucial role in supporting market access and fair reimbursement for precision medicine approaches. By fully utilizing RWE, patient data, and the latest emerging technologies, teams can enable a “precision reimbursement” model which extends beyond more traditional contracts. For example, applying ML models to real world data enables teams to identify clinical features that define patients who are most likely to benefit from a given treatment.
Using ML models to make powerful predictions about patient outcomes, pharmaceutical companies can collaborate with healthcare providers to identify data-driven reimbursement models which reflect the true value of a treatment, ultimately powering better allocation of funding and directing the right treatments to the right patients.
Flexible clinical trial design
Adaptive clinical trial designs are key to accelerating the speed and cost-effectiveness of precision medicine studies for autoimmune and inflammatory diseases. Responding to unfolding data and participant feedback enables a flexible, more efficient approach with a greater chance of meeting recruitment targets and overall trial success. For example, a study by The Economist Intelligence Unit found that therapies developed using a patient-centric design had a 20% higher chance of going to market compared to those developed using a more traditional approach.
Platform trial design
Platform trials differ from traditional clinical trial design in that they are open ended. In this model, new interventions can be added, assessed, and removed as the trial goes on, without having to specify what these interventions will be at the outset.
Additionally, platform trials have the flexibility to update the control or “usual care” group as the study progresses. For example, if the platform trial shows a therapy to be much more effective than the current gold standard, that new drug can be rolled out to patients immediately, becoming the new “usual care” against which all new drugs in the study are tested.
Patient centricity
Patient centricity in clinical trials is already changing the study design and delivery landscape, and offers numerous benefits to both patients and researchers. This type of trial design is focused on patient-reported outcomes and has been shown to lead to increased recruitment and retention rates, a higher chance of overall trial success, and greater trial efficiency. Such approaches are also key to supporting diversity and inclusivity efforts and ensuring that all populations are represented in, and benefit from, research and clinical trials. By using a patient-centric approach to trials, researchers can also ensure that they are addressing logistical barriers and cultural sensitivities.
Digital tools and real-time monitoring
Using wearable devices and smartphone applications to enable real-time data monitoring is offering an unprecedented opportunity to improve the patient-centricity, adaptability, and efficiency of clinical trials.
Trial designs can leverage these new tools effectively by:
- Integrating wearable devices and mobile applications to collect continuous patient data, enabling more precise assessment of treatment efficacy and safety.
- Implementing the use of ML algorithms to analyze real-time data and identify early signals of treatment response or adverse events.
- Developing adaptive trial protocols that allow for dynamic adjustment of treatment regimens based on individual patient responses.
By implementing these strategies, stakeholders can aid the development and adoption of precision medicine approaches, ultimately leading to improved patient outcomes and greater healthcare and research efficiency.
Stakeholder engagement
The future of precision medicine for autoimmune and inflammatory diseases hinges on robust collaboration and engagement across a diverse ecosystem of stakeholders. By fostering multidisciplinary partnerships, listening to patient voices, and aligning efforts across the public and private sectors, organizations can help to accelerate the development of breakthrough precision therapies in these underserved condition areas.
Patient advocacy groups, payers, and policymakers
Patient advocacy groups play a crucial role in advancing precision medicine for autoimmune and inflammatory diseases. These organizations, such as the Autoimmune Association, are at the forefront of raising awareness, promoting education, and supporting research initiatives. By actively involving patient advocates in the development of precision therapies, researchers can ensure that patient needs and lived experience are central to the study design process.
Engagement with payers and policymakers is also key to the successful adoption of precision medicine approaches, with collaboration playing a fundamental role in overcoming siloed approaches and fragmentation across the autoimmune and inflammatory disease landscape. Coordinated efforts to improve genomic literacy and training, data sharing, and cohesive best practices for trial design and reimbursement models will ultimately determine the long-term success of precision medicine efforts.
Public-private partnerships
Public-private partnerships have emerged as a powerful vehicle for driving innovation in precision medicine. Bringing together expertise from academia, government, industry, and patient advocacy groups, these partnerships make it possible to overcome the complexities of autoimmune and inflammatory diseases and accelerate treatment development.
Launched in 2021, the Accelerating Medicines Partnership® Autoimmune and Immune-Mediated Diseases (AMP® AIM) program is a great demonstration of the power of public-private partnerships. Bringing together 15 cross-organizational partners, the partnership aims to deepen scientific understanding of the cellular and molecular interactions that lead to disease.
Innovation culture and talent development
A culture of continuous learning that recognizes the rapidly evolving nature of new technologies and data analysis capabilities is another key element of success. Implementing strategic frameworks which champion cross functional expertise, integrate continuous training, and encourage a culture of informed risk-taking will play a central role in meaningfully addressing the challenges and complexities of autoimmune and inflammatory conditions
Growth mindset
Establishing a dynamic, highly-trained workforce which embraces ongoing education in cutting-edge fields like genomics, bioinformatics, and AI sits at the heart of innovation culture. Organizations should provide structured opportunities to upskill through workshops, certifications, and collaborative research initiatives, to not only continuously train employees but embed a foundational culture which recognizes cross functional expertise-sharing as a cornerstone of improved understanding and success.
In order for teams to deliver on the promise of precision medicine, they will need to be able to respond and adapt to new technologies including AI, ML, and gene editing innovations. Encouraging a culture of knowledge sharing, mentorship, and proactive education around data analysis, software development, and genetics will be key to accelerating and developing precision medicine solutions.
Risk tolerance
A central element of the progressive mindset required to successfully deliver new innovations is cultivating an environment where calculated risk-taking is encouraged. Innovative breakthroughs often emerge from high-risk, high-reward projects and can accelerate advancements in areas such as biomarker discovery and cell and gene therapy development. Without innovative concepts and balanced risk-taking approaches, the potential of new technologies like CAR-T cell therapies and CRISPR will likely remain underutilized.
Intellectual property and data sharing
The success of precision medicine initiatives in the context of complex autoimmune and inflammatory diseases will rely heavily on effective data sharing and intellectual property (IP) strategies. Balancing IP protection with open science initiatives and developing frameworks for secure, interoperable data sharing are crucial for advancing research and improving patient outcomes.
One of the biggest challenges facing precision medicine development is navigating IP protection while recognizing that many initiatives make use of independent registries and open data resources – especially in the context of heterogeneous conditions where diversity of disease presentation leads to limited validation. Striking the balance between publicly available and privately protected data is essential for driving innovation and powering the future of therapeutic development.
For example, as well as carrying out research and development (R&D) in-house, pharmaceutical organizations have the capacity to accelerate R&D efforts by sharing select IP assets with third party researchers. Not only does this support increased speed of innovation and delivery of benefit to patients, it also helps to address and improve equity of access with the potential to support new and adapted therapies in low and middle income countries.
Strategies for addressing the conflict between IP and open data include:
- Selective IP protection: Focusing on patenting core innovations while openly sharing non-critical data.
- Open access publishing: Encouraging researchers to publish findings in open-access journals to accelerate knowledge dissemination.
- Tiered access models: Implementing systems that allow different levels of data access based on user credentials and research needs.
- Collaborative licensing agreements: Developing flexible licensing terms that protect IP while enabling broader research use.
Secure and collaborative data sharing
In addition to frameworks such as the FAIR Guiding Principles, implementing robust security protocols that enable data integrity and patient privacy is key to guaranteeing ethical use of data while fostering a culture of collaboration.
Key components of secure data sharing frameworks include:
- Encryption, anonymization and consent: Strong encryption methods, thorough anonymization techniques, clear patient consent and up-to-date staff training are required to ensure effective protection and sharing of patient data.
- Blockchain technology: Leveraging blockchain can enable secure, transparent, and auditable data sharing among multiple parties, as well as continuous monitoring of access.
- Federated learning: Federated learning is a decentralized ML technique that trains AI models across multiple devices, but without sharing raw data. In a health data context it enables data privacy, security, efficiency, and scalability – all while keeping data local.
- Standardized data formats: Adoption of common data standards as outlined in the FAIR Guiding Principles facilitates seamless integration and analysis across different geographies and institutions.
- Ethical governance: Ensuring compliance with regulations such as the Health Insurance Portability and Accountability Act (HIPAA) in the US and the General Data Protection Regulation (GDPR) in the European Union is fundamental to secure and ethical data use and storage.
As the field of precision medicine evolves, continuous reevaluation and adaptation of these strategies will be crucial to maintain patient data security and the delicate balance between open data innovation and IP protection.
Key takeaway
Delivering on the promise of precision medicine in autoimmune and inflammatory disease will require more than technological progress. It demands a shared commitment to integration, collaboration, and continuous learning, from regulators and researchers to technologists, payers, and patient advocates.
By building strategic infrastructure, investing in talent and tools, and working on a culture of transparency and risk-tolerant innovation, the healthcare ecosystem can move beyond fragmented efforts and toward coordinated, sustainable change. This framework is a starting point; it's a roadmap to help ensure that breakthrough therapies don’t remain siloed in research labs, but reach the people who need them most.
To find out more, read our full whitepaper: "The future of precision medicine in autoimmune and inflammatory disease: A strategic framework."
