Rare Disease Archives - SAI MedPartners

Challenges and Concerns Raised by EFPIA on Joint Clinical Assessments

In the healthcare regulation sphere, proposed EU-level joint clinical assessments (JCAs) have sparked debate in the pharmaceutical industry. A recent EFPIA report highlights concerns about the rigidity of the guidelines, particularly in oncology, and suggests revisions to ensure a more efficient and adaptable evaluation process that accommodates diverse stakeholder needs across Europe.

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Challenges in Hemoglobinopathies Projects and the Support of KOL Match

Missy Heizmann / September 19, 2022

There are over 7,000 rare diseases described, affecting about 20-30 million people in the U.S. alone, according to the NIH. While each condition has its specificities, there are common challenges in the rare disease space, such as finding patients and leveraging centers of excellence and referral networks. This article discusses such challenges and how partnering with SAI’s subject matter experts can help overcome them.

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Challenges and Concerns Raised by EFPIA on Joint Clinical Assessments

Challenges in Hemoglobinopathies Projects and the Support of KOL Match