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How real-world evidence is changing Health Technology Assessment (HTA) submissions

The EU joint HTA procedure: a gate or barrier for access?

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Alagille Syndrome
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Alagille Syndrome
Wilson’s Disease
Bile Acid Synthesis Disorders
Niemann-Pick Type C
Cushing Syndrome
Progressive Familial Intrahepatic Cholestasis
Adrenal Cortical Carcinoma
Sickle Cell Disease

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