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The role of the patient voice in regulatory decision-making in China and Europe: a comparison of approaches
Jul 02, 2025
While China's patient organisations (POs) emerged relatively late compared to those in Europe they have matured rapidly over the past decade across diverse disease areas and have started to drive meaningful changes in the way new health interventions are appraised, moving towards a more patient-centric approach. In this blog we compare the two approaches, and answer a few key questions, with a special focus on rare diseases.
How have patient organisations evolved differently in China and Europe?
91% of rare disease POs in China were established post-20101, driven by policy development and alliances such as CORD (Chinese Organisation for Rare Disorders), which supports POs by promoting communication exchanges, networking, and training. This momentum has increased the number of rare disease POs from 18 to over 150 in 12 years2, with alliances playing pivotal roles in capacity-building and resource integration.
In contrast, POs in Europe have a longer history, having evolved into influential institutions over the past 80 years with a pan-European dynamic, creating significant impact and enabling change for patient communities. Their maturity is reinforced not only by their longer history but also by robust institutional and political support from entities like the European Union (EU) and the European Medicines Agency (EMA). Today, European national and umbrella patient organisations play a significant role in healthcare decision making, although their influence varies across different countries, institutions, and stakeholder groups3.
Where did the focus on Patient Voice originate?
Under China’s governing principle of “serving the people”, China's healthcare reforms since 2009 have prioritized patient-centric institutional policies and increased the impact of the patient voice. One of the objectives of the Health China 2030 Plan is to improve health service capabilities with an efficient healthcare system, and both the 2016 Drug Review and Approval Reforms and the 2018 National Reimbursement Updates highlight China's focus on patient accessibility, by accelerating the innovative drugs approval process and expanding reimbursement coverage. This dual-engine strategy combines regulatory efficiency with reimbursement expansion, integrating Western models with China's unique volume-based procurement system to achieve cost economies of scale.
In Europe, policies to systematically incorporate patient perspectives into regulatory activities have been evolving since the early 2000s. This shift began with patients joining committees within the European Medicines Agency (EMA) and the establishment of the Engagement Framework in 2005, recently updated in 2022, with key patient working groups organisations. Policies at the HTA level are also evolving, particularly with the new EU Joint Clinical Assessment (JCA) framework under the HTA Regulation (EU 2021/2282), which is focused on the systematic inclusion of the patient voice. Prominent umbrella and pan-European patient organisations have driven much of this policy evolution, advocating for enhanced patient involvement in both regulatory and HTA decision-making.
What are the key patient-centric Policies in Drug Development?
China's drug regulatory system has recently undergone a paradigm shift toward patient-centric drug development; systematically integrating patient perspectives through evolving policy frameworks and technological innovation. The December 2021 Guidelines for Patient-Reported Outcomes (PROs) in Drug Clinical Trials issued by the Center for Drug Evaluation (CDE) established foundational standards for PRO application in drug development. It provides standards for using PRO as primary or secondary endpoints.
By 2023, China further solidified its commitment through three pivotal pillars:
- Encourage early-stage patient consultations to ensure trials reflect patient experiences
- Integrate patient experience data as a key consideration in clinical trial design
- Enhance patient accessibility through formalized decentralized clinical trials (DCTs): allowing hybrid models that integrate telemedicine and local sample collection sites, significantly improving accessibility for rural and mobility-challenged populations
The regulatory evolution has yielded measurable outcomes: PRO adoption surged from fewer than 20 trials (<5% of total trials) in 2010 to over 2,000 (~30% of total trials) by 20204, reflecting both compliance pressure and industry recognition of PRO's dual role in enhancing trial efficiency and patient relevance.
The Chinese government has consistently paid attention to rare disease drug development. In 2021, the CDE issued the Technical Guidelines for Clinical Development of Rare Disease Drugs to encourage PRO application and to consider PRO as primary endpoints in rare disease clinical trials. Building on this foundation, 2024 marked a new phase of rare disease policies with the launch of the 2024 New Pilot Regulatory Program (the “CARE”) by the CDE. This program establishes a five-stage implementation framework to systematically embed patient engagement across the entire drug lifecycle:
- Stage A, R&D Initiation: collect patient experience data (PED)
- Stage B, Pre-IND (Investigational New Drug): integrate PED into the target product profile, clinical development plan and design of early study protocols and develop clinical outcome assessments (COAs)
- Stage C, Pre-pivotal study: optimize patient experience and define COA instruments
- Stage D, Pre-NDA/NDA (New Drug Application): incorporate patient perspectives into related documents to support NDA applications and benefit-risk assessments
- Stage E, Post-approval: investigate real-world patient experiences and integrate patient experience data (PED) into benefit-risk reassessment
Applicants may apply to join the CARE Program voluntarily and then the CDE selects 1–2 candidates per stage. Candidates are required to complete specific tasks in related stages.
Further advancing this trajectory, the CDE released the 2025 PRO Guidance for Rheumatic Diseases Drug Clinical Trials, which standardizes PRO application in clinical trials to enhance scientific rigor and accelerate the development of innovative drugs in rheumatic diseases.
In Europe, the European Medicines Agency (EMA) drives patient involvement in drug development through a comprehensive framework that includes guidelines on Patient Experience Data (PED), scientific advice, and protocol assistance. By engaging patients early in the pre-submission phase and incorporating their input throughout the medicine's lifecycle, the EMA ensures that patient perspectives are integral to regulatory decision-making. The Patients' and Consumers' Working Party (PCWP) within the EMA involves patients and consumers in discussions on various regulatory topics, ensuring their perspectives are considered in decision-making. Pilot programs, such as the pilot on early dialogue with patient organisations for orphan marketing authorisation applications, have led to the adoption of the CHMP early contact with patient organisations as an established methodology, further reinforcing the importance of patient-centric approaches, while regulatory oversight and feedback mechanisms ensure pharmaceutical companies adhere to these guidelines5-6.
There is also a growing trend of partnerships between industry and the public sector, driving initiatives to systematically embed the patient voice into health research and innovation projects. Notable among these is the Innovative Health Initiative, an EU public-private partnership dedicated to funding health research and fostering innovation. A recent example is the READI project, launched in January 2025, which focuses on research and diversity inclusion. This pioneering initiative, co-led by patients, aims to promote a more cohesive and democratic ecosystem for clinical studies by engaging key stakeholders and ensuring the inclusion of underserved and underrepresented populations.
How are Policies integrating the Patient Voice into drug approval and reimbursement in practice?
For the NDA (New Drug Application) planning stage, the National Medical Products Administration (NMPA) has established four pathways to accelerate approvals for clinically urgent and breakthrough therapies, including conditional approval, priority review, breakthrough therapy designation, and special approval procedures. These frameworks, together with the “CARE” program, aim to accelerate therapies for severe diseases while integrating patient voices into regulatory decision-making. For example, Sanofi’s QFITLIA® (fitusiran) (a drug for haemophilia A or B with or without factor inhibitors) was granted priority review in China in 2018 but faced prolonged approval delays. This drug was then accepted into the “CARE” program in November 2024 to accelerate its approval. The drug’s clinical trial design incorporated patient qualitative interviews, with plans to collect patient experience data (PED) during the post-approval stage. 7
For NRDL (National Reimbursement Drug List) reimbursement, the pricing decision in China is mainly based on CEA (Cost Effectiveness Analysis) and BIA (Budget Impact Analysis). The disease burden and drug benefits will be assessed in the CEA model, including PRO. Since 2019, the PO called CORD (Chinese Organisation for Rare Disorders) and IQVIA have jointly released a series of reports on rare diseases in China. Among these, the report titled “China Rare Disease Drug Accessibility Report 2019” has drawn widespread attention from policymakers and the public. By presenting case studies of POs, this report underscores their critical role in addressing patient accessibility, advocating for strengthening participation of patients and patient organisations in rare disease governance8. As the first comprehensive analysis of drug accessibility and reimbursement landscape in China, its patient-centered recommendations likely contributed to subsequent policy discussions. The report coincided with China’s NRDL updates since 2019, aligning with one of its principles to prioritize disease and patients’ unmet needs, indirectly influencing rare disease policy considerations at both national and local levels. Moreover, POs may indirectly impact the NRDL listing. A PO called Lymphoma Home9 published six white papers documenting patients’ survival challenges in 2021 and submitted them to pharmacoeconomic experts as evidence to support the NRDL inclusion of Gazyva. Gazyva has been successfully listed in NRDL in 2021.
In the European systems, where patient centricity is more developed, patient organisations and the patient voice are integrated into EU Health Technology Assessment (HTA), individual countries frameworks, and into reimbursement decisions through structured involvement, guidance frameworks, and national processes. However, approaches vary significantly across countries. For example, Germany's IQWiG, France's HAS, and the UK's NICE have established processes for integrating patient perspectives, while other countries may have less structured approaches. However, a more established process does not always equate to higher impact on decision outcomes and the patient perspective may not influence the access and reimbursement decision making to the degree it should or is expected by patients.
The EU Joint Clinical Assessment (JCA) implemented from the 12th of January 2025, aims to standardize patient involvement, accelerate access to medicines, and enhance transparency by centralizing clinical evidence assessments across member states. This initiative seeks to address existing disparities and ensure a more inclusive, transparent, and patient-centred approach to healthcare assessments and reimbursement decisions in Europe.
How should manufacturers react to these changes?
China’s emphasis on integrating the patient voice into policies represents a critical inflection point for pharmaceutical companies. To align with this shift and respond to the call to action, companies need to adopt a proactive, collaborative approach, grounded in strategic imperatives such as transitioning from viewing POs as passive recipients of information to partners in co-creating drug development and commercialization. Chinese POs are moving from "peripheral supporters" to "stakeholders in their own right”, and their future development hinges on policy implementation, resource integration, and their own professional capacity building.
Meanwhile, the EU ecosystem is promoting the enhancement of patients’ role as active stakeholders and partners along the entire product lifecycle, with current key legislations implementation and revision providing a unique opportunity for changes.
Pharma companies should support a paradigm shift by building patient centricity into their internal framework as well as support the development of POs through awareness, technical upskilling, and resource provision by:
- Embedding patient perspectives from the earliest stages of drug development—including preclinical planning—to ensure that patient needs and experiences shape the entire product lifecycle. Early and meaningful engagement with POs not only enhances relevance of outcomes but also fosters authentic advocacy that grows organically over time.
- Supporting patient empowerment in standardized regulatory and access decision making processes (e.g. the product value assessment/HTA Landscape) As regulatory and health technology assessment processes and patient involvement become more structured and formalized globally, pharma must proactively collaborate with POs to build capacity. This includes co-developing training, resources, and platforms that equip patients to effectively engage and advocate within evolving decision-making frameworks
References:
1. Xiao, Lei. Roles of patient organisations in rare disease research and orphan drug development. International Journal of Pharmaceutical Research, vol. 44, no. 2, 2017, pp. 209-14. doi:10.13220/j.cnki.jipr.2017.02.022
2. Liu, Minjie. Interview with Huang Rufang: A Decade of Trailblazing China's Rare Disease Philanthropy. 2023. Culture and History Panorama: Figures, no. 9.
3. https://www.eu-patient.eu/members/what-is-a-patient-organisation/historical-context/. Last accessed on 28th of May 2025
4. Zhou H, Yao M, Gu X, et al. Application of Patient-Reported Outcome Measurements in Clinical Trials in China. JAMA Network Open. 2022;5(5):e2211644. doi:10.1001/jamanetworkopen.2022.11644
5. https://www.ema.europa.eu/en/documents/report/pilot-early-dialogue-patient-organisations-orphan-marketing-authorisation-applications-outcome-report_en.pdf. Last accessed on 28th of May 2025
6. https://www.ema.europa.eu/en/documents/report/stakeholder-engagement-report-2022-2023_en.pdf. Last accessed on 28th of May 2025
7. Mao, N. Y., & Huang, R. F. (2024). Study on Pathways and Models of Patient Participation in Drug Regulation. [Research report]. China Pharmaceutical University, CORD Rare Disease Center. https://www.raredisease.cn/
8. National Medical Products Administration, Center for Drug Evaluation. (2024). [Guidelines for the application of the "CARE Program" (Patient-Centered Rare Disease Drug Development Pilot Project)]. Retrieved November 20, 2024
9. Huang, R., & Shao, W. (2019). China Rare Disease Drug Accessibility Report 2019. Hangzhou, China: Cord Rare Disease Center (CORD) & IQVIA.