Empowered By Us
Empowered By Us is an independent, patient-led initiative advancing inclusion, equity, and health system reform for people with rare diseases and conditions, including dwarfism.
ID: 246166594802-94
Lobbying Activity
Response to EU’s next long-term budget (MFF) – EU funding for competitiveness
3 Nov 2025
Empowered By Us (EBU) is a patient-led strategic initiative driving inclusion, innovation, and equity in rare diseases. EBU operates at the intersection of policy, industry, and lived experience to co-create frameworks that align access, evidence, and competitiveness across Europe. Its work includes the development of the Fast Track access model (Netherlands) and the Rare Disease Innovation and Equity Strand proposal under the European Competitiveness Fund, reflecting a practical, system-level approach to inclusion as infrastructure for growth.
Read full responseResponse to Critical Medicines Act
1 Jul 2025
Empowered By Us welcomes the opportunity to contribute to the implementation of the Critical Medicines Act. This submission focuses on the specific vulnerabilities faced by rare disease patients and low-volume therapies within European medicine supply systems. It highlights structural equity gaps in the current proposal and offers three targeted recommendations: Explicitly include rare and off-patent therapies in strategic prioritisation Embed structural participation of civil society in CMA governance Require disaggregated access monitoring and equity-based impact analysis True resilience means protecting those most at risk. Our submission outlines how the CMA can become a blueprint for fairness, sustainability, and inclusive access. Please find our 1-page submission attached.
Read full response24 Jun 2025
Empowered By Us welcomes the opportunity to contribute to the implementation of Regulation (EU) 2021/2282 through this draft Implementing Regulation. We support the goals of timely, high-quality joint clinical assessments (JCAs) for medical devices and in vitro diagnostics, and respectfully submit the following implementation-focused recommendations to ensure proportionality, relevance, and transparency across diverse patient populations and health system contexts. 1. Strengthen expert identification procedures (Article 8): The Regulation outlines criteria for individual expert selection, but does not require the inclusion of lived or practice-based user expertise for rare, complex, or functionally limited populations. We recommend mandating such inclusion when the device indication justifies it, to ensure real-world relevance and equity in assessments. 2. Enable flexible evidence pathways (Annexes IV): Current dossier templates prioritise RCTs and comparative data. We propose the addition of a Justification for Alternative Evidence section to allow for real-world, observational, or mixed-method data where conventional trials are infeasible. This is especially relevant for small populations and early access pathways. 3. Integrate usability and experience reporting: The templates do not include any field for reporting usability, accessibility, or patient-reported experience measures (PREMs). Including these data points would ensure that JCAs reflect the practical impact of a device on patients autonomy, safety, and burden of use. 4. Balance commercial confidentiality with public interest (Article 21): While protecting sensitive information is essential, the current provisions allow for redaction of content that may affect patient understanding or use. We recommend a mechanism for review by independent patient representatives under confidentiality, or at minimum, a justification for redacting end-user-relevant data. These targeted additions would strengthen the Regulations implementation by improving transparency, procedural consistency, and stakeholder inclusion. Empowered By Us remains available to contribute to further practical guidance or tool development to support inclusive rollout of the EU HTA system. These recommendations are particularly relevant for rare diseases, where evidence generation is often constrained, patient populations are small and heterogeneous, and real-world usability is critical to long-term impact. Without procedural flexibility and inclusive expert engagement, JCAs risk unintentionally excluding innovations that serve rare, paediatric, or complex conditions. Implementing safeguards at this stage is essential to ensure the EU HTA framework delivers on its promise of equitable access for all.
Read full responseResponse to Biotech Act
5 Jun 2025
Empowered By Us is an independent, patient-led initiative focused on equitable health innovation, rare disease policy, and strategic patient involvement. We welcome the opportunity to contribute to the development of the EU Biotech Act. The Biotech Act must not only support scientific excellence. It must also foster innovation that is accessible, societally meaningful, and system-ready. For underrepresented populations, including those living with rare diseases, the promise of biotechnology remains structurally out of reach. These groups often face fragmented development pathways, limited investment incentives, and outcome frameworks that fail to reflect lived realities. We urge the Commission to ensure that equity, relevance, and co-creation are embedded from the start. Specifically, we recommend three priority actions: 1. Require early-stage patient and civil society involvement: Patients and public stakeholders should be structurally included in early development processes. This includes helping define innovation priorities and shaping success metrics. Early co-creation improves system fit, reduces downstream friction, and builds public trust. 2. Incentivise innovation for unmet and non-lucrative needs: Rare, ultra-orphan, and underserved areas must be explicitly eligible for EU biotech support. This includes access to targeted funding, infrastructure, and regulatory guidance. Public investment should reflect public value, not only market potential. 3. Align success metrics with public health and system priorities: Developers should be supported to adopt outcome frameworks that reflect quality of life, functional status, caregiver burden, and long-term system impact. These should be aligned with HTA frameworks across Member States to accelerate real-world uptake. This approach ensures that biotech growth translates into meaningful outcomes, not just more products. The Biotech Act presents an opportunity to shape not only how fast we innovate, but who benefits from that innovation. Empowered By Us remains available for dialogue and co-design as this legislation evolves. Contact for Dialogue: Sophie H. Turner Strategic Patient Advocate Empowered By Us | contact@empoweredbyus.com
Read full responseResponse to Establishment of an expert panel on orphan and paediatric devices
20 May 2025
Empowered By Us welcomes the European Commissions proposal to establish an expert panel on medical devices for orphan and paediatric populations. This is a timely and necessary initiative that recognises the distinct clinical and social challenges faced by those living with rare conditions or requiring age-specific solutions. As a strategic patient-led initiative working at both national and EU levels, we support the inclusion of expert panels that provide specialised scientific and clinical advice. However, we strongly advocate that this structure must go beyond technical evaluation. It must also embed real, lived experience to ensure relevance and equity in outcomes. We encourage the Commission and the European Medicines Agency to consider the following in the operationalisation of this panel: 1. Patient involvement as a core design principle Patients and families affected by rare and paediatric conditions are experts in their own right. Their lived experience should not only inform decision-making, but shape it. We recommend the formal inclusion of at least one lived-experience expert (for example, a patient advocate or caregiver) in each panel opinion process. This can be through roles such as reviewer, co-rapporteur or assigned advisor. 2. Recognition of lived experience as data Evaluations of devices, particularly in rare conditions, often rely on limited or fragmented clinical evidence. In such cases, real-world experience can fill critical knowledge gaps, especially around long-term quality of life and usability. The perspectives of patients and carers provide crucial insight into functionality, acceptability and real-world benefit. These inputs should be explicitly recognised as a form of contextual evidence alongside clinical data. 3. Transparent handling of stakeholder input It is encouraging that the annex categories for panel work refer to information from stakeholders including patient organisations. To ensure this input is genuinely valued, the methodology for integrating stakeholder contributions should be made transparent. This will enhance legitimacy, trust and clarity for all contributors. 4. Dedicated resources for co-creation Patient groups should be supported not just for consultation but for meaningful co-creation. This includes financial recognition for time, preparation and follow-up involved in panel participation or submission review. Resourcing smaller or underrepresented groups is especially important to ensure inclusive participation. 5. Ongoing dialogue rather than one-off input We recommend establishing a standing liaison mechanism, such as a Patient Advisory Circle, linked to this expert panel. This would ensure the work remains continuously informed by lived realities, not only by occasional calls for comment. This panel has the potential to set a new standard for patient-inclusive regulatory science. Empowered By Us is ready to contribute through structured consultation, active participation and strategic input from the communities we represent. These include individuals with skeletal dysplasias such as achondroplasia and others affected by underrepresented rare conditions.
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