Progressive familial intrahepatic cholestasis type 2 (PFIC2) is a rare condition that affects the liver. People with this condition generally develop signs and symptoms during infancy, which may include severe itching, jaundice, portal hypertension (high blood pressure in the vein that provides blood to the liver) and hepatosplenomegaly (enlarged liver and spleen). PFIC2 generally progresses to liver failure in the first few years of life. Affected people also have an increased risk of developing hepatocellular carcinoma (a form of liver cancer). PFIC2 is caused by change (mutations) in the ABCB11 gene and is inherited in an autosomal recessive manner. Treatment options include ursodeoxycholic acid therapy to prevent liver damage, surgery, and/or liver transplantation. There are three known types of PFIC: PFIC1, PFIC2, and PFIC3.
Market Dynamics
Increasing awareness about progressive familial intrahepatic cholestasis type 2 disease is expected to provide more opportunities for players in the progressive familial intrahepatic cholestasis type 2 treatment market in the near future. For instance, on October 3, 2019, Albireo Pharma, Inc., a clinical-stage orphan pediatric liver disease company developing novel bile acid modulators, announced its support for PFIC Awareness Day and reaffirmed its commitment to patients and families living with progressive familial intrahepatic cholestasis (PFIC).
However, higher economic growth in low- and middle-income countries has not closed the gap in health spending, and global inequity in health spending has remained largely unchanged. For instance, according to a report on ‘New Perspectives on Global Health Spending for Universal Health Coverage’ by WHO published in 2015, high-income countries with only 16% of the world’s population, account for 80% of global health spending. Conversely, 76% of the world’s population live in middle-income countries, but these account for less than 20% of global health spending. Low-income countries, with more than half a billion people, accounted for less than 1% of the world’s health spending in 2015. Thus, low healthcare spending in low income countries limits such countries for research and development of new drugs for treatment of rare diseases.
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