STATEMENT: New treatments and regional data policies, the most debated in the event of rare diseases

(Information sent by the signatory company).

STATEMENT: New treatments and regional data policies, the most debated in the event of rare diseases

(Information sent by the signatory company)

Madrid, February 17

New treatments and regional data policies, the most debated in the event of rare diseases

Decision makers at the regional level met at the Europe Rare Disease Summit, organized by Bamberg Health, to discuss the challenges and lessons learned around a topic that affects between 3.5% and 5.9% of the population at a regional level. world

Within the framework of a new Rare Disease Day, focused on raising awareness about the necessary improvements in treatments, diagnoses and public policies, the Europe Rare Disease Summit was held, organized by Bamberg Health. Actors from different countries in the region, both from the public and private sectors and third sector organizations, met to discuss the need to work together to improve the quality of life of those affected by this type of disease. "By working together with biopharmaceutical companies, regulatory agencies, payers, HTAs and political actors, we have more collective strength and a better chance to break new ground so that patients, caregivers and the healthcare system can fully benefit from medical innovation," says Thomas Bols, one of the speakers at the event and Head of Government Affairs and Patient Advocacy, EMEA and APAC at PTC Therapeutics. The various panels discussed topics such as government initiatives, national and regional plans against rare diseases; the challenges and opportunities in the development of and access to orphan drugs; the role of artificial intelligence and big data related to registration and diagnosis; advances in treatment and diagnosis focused on gene therapies; the implementation and development challenges of viral vector and mRNA technologies against rare diseases; the importance of the patient's voice in the approval and access to new drugs. Regarding the challenges around data, Gianni d'Errico, Vice President at ICPer-Med - International Consortium for Personalized Medicine, said: "The European Health Data Space is a great opportunity to finally break down the barriers around the exchange of data. This can help national organizations around Europe to improve their collection and analysis capacity that will help to better understand the real situation of rare diseases from a broader perspective." The event was held with the collaboration of important Spanish organizations and at a European level such as FEDER, Rare Disease Croatia, Rare Beacon, which work to give more visibility to the issue, achieve improvements in access and treatment, and accompany affected families. In turn, it had the support of large companies in the industry such as Pfizer, Moderna, PTC therapeutics, Recordati Rare Disease, UCB, Takeda, Clinerion, Idonia and Kyowa Kirin. About Bamberg Health Website " Twitter " LinkedInBamberg Health is a platform that connects more than 20,000 health professionals, and aims to offer new perspectives and solutions in health management and innovation through virtual and live events. It provides a place where professionals can meet to exchange knowledge and do business without barriers, contributing to the advancement of the pharmaceutical and healthcare industries. Bamberg Health organizes international health summits in Europe and Latin America.

ContactContact name: Camila DemarcoContact telephone: 34649252693


Pie de foto: Executives from different companies and associationsAutor: Bamberg Health