Saturday, June 22, 2024

Trust in Scientific Evidence for Rare Disease Treatments

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EURORDIS-Rare Diseases Europe is urging the European Medicines Agency (EMA) and the Committee for Medicinal Products for Human Use (CHMP) to hold a public stakeholder meeting following recent developments concerning the conditional marketing authorization for Translarna. This call comes in light of the CHMP’s recommendation in January 2024 to not renew the conditional marketing authorization for Translarna, a treatment for Duchenne muscular dystrophy.

The European Commission has requested a reassessment of this decision, prompting the CHMP to restart the evaluation of the application to renew Translarna’s conditional marketing authorization. EURORDIS shares the disappointment of patients and healthcare professionals following the results of three randomized clinical trials that did not demonstrate clinical benefits for Translarna. Furthermore, the data registries submitted by the marketing authorization holder were deemed methodologically inadequate by the EMA.

EURORDIS strongly advocates for the use of real-world data as complementary evidence to clinical trial data, particularly when no other type of evidence can be generated. Data from children treated with Translarna over the years could provide valuable insights for decision-making, provided that appropriate analytical methods are used. However, EURORDIS emphasizes that regulatory decisions must be based on robust scientific evidence to maintain public trust. Decisions made without a solid evidence base do not benefit patients.

Eurordis Calls for Transparent Review on Rare diseases

This situation has led EURORDIS to suggest a public stakeholder meeting to discuss the matter further and reflect on the potential influence of non-scientific factors on regulatory decisions. EURORDIS is calling on the EMA and CHMP to organize this meeting to facilitate exchanges with the patient community and support the CHMP’s decision-making process. Such a meeting would enable a transparent review of all data related to Translarna, allowing patients and citizens to understand the reasoning behind the decisions and to share their perspectives.

Since its inception, the EMA has been committed to evidence-based decision-making, free from political influence. Upholding this principle is crucial for maintaining patient trust and welfare. EURORDIS is, therefore, urging the EMA and CHMP to organize a public stakeholder meeting to facilitate exchanges with the patient community and support the CHMP’s decision-making process. This would ensure a transparent review of all data related to Translarna, allowing patients and citizens to understand the reasoning behind the decisions and share their perspectives.

Translarna has had conditional approval since 2014 for treating patients aged two years and older with Duchenne muscular dystrophy who are able to walk. The continuation of this authorization depends on thorough and transparent evaluation processes.

Rare Disease

Strengthening the Regulatory Process for Translarna

EURORDIS-Rare Diseases Europe is a non-profit alliance of over 1000 rare disease patient organizations from 74 countries, working to improve the lives of the 30 million people living with a rare disease in Europe. By connecting patients, families, and patient groups, as well as mobilizing all stakeholders, EURORDIS strengthens the patient voice and shapes research, policies, and patient services.

The organization’s call for a public stakeholder meeting is aimed at ensuring that the regulatory process remains transparent and accountable. This meeting would also help prevent the potential influence of non-scientific factors on regulatory decisions. By engaging with the patient community and other stakeholders, EMA and CHMP can ensure that their decisions are based on the best available evidence and are in the best interests of patients.

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The call for transparency and stakeholder engagement in the review process of Translarna reflects EURORDIS’s commitment to evidence-based decision-making. This initiative aims to uphold the integrity of the regulatory process and maintain public trust. The proposed public stakeholder meeting would provide a platform for open dialogue, enabling a thorough review of all relevant data and ensuring that the voices of patients and citizens are heard.

 

Resource: Rare Diseases Europe, May 31, 2024

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