Prioritising Patient-Driven Outcomes for Rare Disease Biotechs
The Joint Clinical Assessment (JCA) framework, established under the EU Health Technology Assessment (HTA) Regulation (Regulation (EU) 2021/2282), introduces a unified approach to evaluating health technologies across EU member states. This framework aims to streamline assessments and enhance patient access to innovative therapies. For rare disease biotechs, navigating the JCA effectively necessitates a patient-centric strategy that includes upskilling patient advocates, leveraging advanced tools, and fostering cross-border collaborations.
1. Upskilling Patient Advocates: A Foundation for JCA Success
Patient advocates play a crucial role in representing patient perspectives during the HTA process. Providing them with comprehensive HTA training ensures they can effectively contribute to evidence generation, advocacy efforts, and regulatory engagements.
- Understanding the JCA Process: Training programs should elucidate the JCA's procedures, timelines, and the significance of patient input, enabling advocates to engage meaningfully.
- Contributing to Evidence Generation: Educating advocates on methodologies for developing patient-reported outcomes (PROs) and real-world evidence (RWE) ensures that patient experiences are accurately captured and presented.
- Effective Communication Skills: Training enhances advocates' abilities to articulate disease burdens and unmet needs compellingly to HTA bodies, ensuring that patient voices are heard and considered.
- Cross-Border Collaboration: Equipping advocates with skills to collaborate across EU member states fosters a unified patient voice, essential for the JCA's collaborative framework.
2. Leveraging Advanced Tools to Meet JCA Requirements
Utilizing advanced technological tools is vital for rare disease biotechs to generate robust evidence and facilitate patient engagement.
- Real-World Evidence Platforms: Implementing digital platforms to collect and analyze RWE provides insights into treatment outcomes and patient quality of life, aligning with JCA's evidence requirements.
- AI-Powered Analytics: Employing artificial intelligence to process complex datasets aids in identifying patient populations and predicting treatment responses, enhancing the quality of evidence submitted.
- Decentralized Data Collection: Utilizing mobile health technologies enables data gathering from diverse patient groups across regions, ensuring comprehensive evidence that reflects real-world scenarios.
- Digital Engagement Tools: Leveraging digital platforms facilitates continuous patient engagement, ensuring that patient perspectives are integrated throughout the product lifecycle.
3. Cross-Border Collaboration: Amplifying Impact
Given the dispersed nature of rare disease populations, cross-border collaboration is essential.
- Unified Advocacy Efforts: Coordinating with patient advocacy groups across EU countries ensures consistent messaging and strengthens the collective patient voice in the JCA process.
- Shared Data Resources: Collaborating on data collection and sharing initiatives enhances the robustness of evidence, addressing the challenges of small patient populations inherent in rare diseases.
- Multilingual Campaigns: Developing educational materials in multiple languages ensures inclusivity and effective communication with diverse patient communities across Europe.
Empowering Patient-Centric Strategies
The JCA framework represents a pivotal moment for rare disease biotechs to enhance patient-centricity in their strategies. By upskilling patient advocates, leveraging advanced tools, and fostering collaboration, biotechs can navigate the complexities of the JCA while amplifying patient voices and driving equitable access.
Let’s Work Together: Contact us today to design tailored HTA training programmes, develop advanced patient engagement strategies, and build cross-border collaborations. Together, we can position your rare disease product for success under the JCA framework, ensuring that the patients who need it most are at the heart of every decision.
Author
Rob Wyer, Managing Partner, swii.ch health
Rob is Managing Partner of swii.ch health, a rare disease patient engagement consultancy specialising in customer engagement and digital transformation in Life Sciences. Swii.ch provides specialised expertise in customer engagement insight, strategy and patient led design, digital transformation and excellence, programme management, and impact measurement.
References:
- Regulation (EU) 2021/2282 on Health Technology Assessment https://eur-lex.europa.eu/legal-content/EN/ALL/?uri=CELEX%3A32021R2282
- Implementation of the Regulation on Health Technology Assessment https://health.ec.europa.eu/health-technology-assessment/implementation-regulation-health-technology-assessment_en
- NICE Real-World Evidence Framework https://www.nice.org.uk/corporate/ecd9
- Use of Real-World Evidence in Regulatory Decision-Making: EMA Publishes Review https://www.ema.europa.eu/en/news/use-real-world-evidence-regulatory-decision-making-ema-publishes-review-its-studies
- Joint Clinical Assessments - European Commission - Public Health https://health.ec.europa.eu/health-technology-assessment/implementation-regulation-health-technology-assessment/joint-clinical-assessments_en
Header image by Ankush Minda on Unsplash
