Rare Disease News & FDA Approvals

PolicyRSS3 days ago

CMS and FDA Announce RAPID Coverage Pathway to Accelerate Patient Access to Life-Changing Medical Devices

The FDA and Medicare (CMS) created a new fast-track pathway called RAPID that helps patients get access to breakthrough medical devices more quickly. This pathway applies to certain advanced devices that treat serious conditions. The goal is to reduce the time between when a device is approved and when Medicare patients can actually use it.

WHY IT MATTERSPatients with rare diseases who rely on breakthrough medical devices may now access life-changing treatments months or years faster than through traditional Medicare coverage processes.

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PolicyPUBMEDMar 26

Genomic pathway managers: a novel role in the genomic medicine care pathway in France-overview and perspectives.

France is creating a new type of healthcare worker called a Genomic Pathway Manager to help doctors order genetic tests for patients with rare diseases and cancer. These managers will make it easier for patients to get genetic testing by organizing the process and helping doctors understand when and how to use these tests. The goal is to make genetic testing available to more people across France starting in 2025.

WHY IT MATTERSIf you have a rare disease in France, this new system could help your doctor identify your condition faster through genetic testing, potentially leading to earlier diagnosis and treatment options.

ℹ Good to knowrare diseases (general)cancer genetic predisposition Read →

PolicyPUBMEDMar 26

Global Access to IMD Drugs: Bridging the Gap Between High-Income Countries and Latin America.

People with inherited metabolic diseases (IMDs) in Latin America struggle to get life-saving medicines that are more available in wealthy countries. This article looks at why these medicines are hard to find in places like Chile and suggests ways to make them more accessible to everyone who needs them, no matter where they live.

WHY IT MATTERSIf you or a family member has an inherited metabolic disease in Latin America, this research directly addresses the barriers preventing you from accessing treatments that patients in high-income countries can obtain.

💬 Ask your doctorInborn Errors of Metabolism (IMDs)Inherited Metabolic Diseases Rare Diseases Read →

Rare disease news

CMS and FDA Announce RAPID Coverage Pathway to Accelerate Patient Access to Life-Changing Medical Devices

Genomic pathway managers: a novel role in the genomic medicine care pathway in France-overview and perspectives.

Global Access to IMD Drugs: Bridging the Gap Between High-Income Countries and Latin America.

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