Progressive Familial Intrahepatic Cholestasis (PFIC) – Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2023 To 2033

Progressive Familial Intrahepatic Cholestasis (PFIC) Market Outlook

Thelansis’s “Progressive Familial Intrahepatic Cholestasis (PFIC) Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2023 To 2033" covers disease overview, epidemiology, drug utilization, prescription share analysis, competitive landscape, clinical practice, regulatory landscape, patient share, market uptake, market forecast, and key market insights under the potential Progressive Familial Intrahepatic Cholestasis treatment modalities options for eight major markets (USA, Germany, France, Italy, Spain, UK, Japan, and China).

Progressive Familial Intrahepatic Cholestasis (PFIC) Overview

Progressive familial intrahepatic cholestasis (PFIC) is an inherited liver condition that hinders bile drainage despite open large bile ducts, leading to cholestasis. It typically presents in early childhood with pruritus, dark urine, pale stool, decreased appetite, and fatigue. PFIC is categorized into three subtypes: PFIC type 1, PFIC type 2, and PFIC type 3, each linked to mutations in the ATP8B1, ABCB11, and ABCB4 genes, respectively. However, some PFIC cases lack these specific gene mutations. Signs and symptoms encompass severe itching (pruritus), inadequate weight gain due to impaired fat digestion and absorption, jaundice (skin yellowing), bleeding due to vitamin K deficiency, and poor bone growth from vitamin D deficiency. Various treatment options, both medical and surgical, are available. The primary medical treatment for all PFIC variants is ursodeoxycholic acid (UDCA). Nevertheless, most PFIC patients develop end-stage liver disease with significant fibrosis in adulthood. Due to the condition's progressive nature, those who do not undergo liver transplantation face a high risk of morbidity and mortality. Complications frequently stem from portal hypertension, including esophageal and gastric varices, leading to severe bleeding, ascites, hemorrhoids, and hepatic encephalopathy. Moreover, end-stage liver disease elevates the risk of hepatocellular carcinoma, particularly in cirrhotic patients. PFIC2 individuals may have an independent risk of malignancy regardless of cirrhosis.

• The estimated incidence of PFIC in the United States ranges from 1 in 50,000 to 1 in 100,000 births.

Geography Covered:

North America- the United States and Canada

Europe- EU5 (Germany, France, Italy, Spain, and the United Kingdom)

Other countries- Japan & China

Study Period: 2023-2033

Current Clinical Practice and Treatment Algorithm

This section of the study covers country-specific current clinical practice, the standard of care, and significant limitations around addressing the unmet needs. Retrospective analysis and bench-marking of clinical study outcomes are presented in terms of Pre-treatment & post-treatment clinical and demographic patient characteristics. Essentially, this section will cover the evolution of the current competitive landscape and its impact on the future treatment paradigm.

KOL Insights:

KOLs across 8 MM markets from the center of Excellence/ Public/ Private hospitals participated in the study. Insights around current treatment landscape, epidemiology, clinical characteristics, future treatment paradigm, and Unmet needs

Market Forecast: Patient Based Forecast Model (MS. Excel Based Automated Dashboard)

- Data Inputs with sourcing

- Market Event and Product Event

- Country-specific Forecast Model

- Market uptake and patient share uptake

- Attribute Analysis

- Analog Analysis

- Disease burden and pricing scenario

- Summary and Insights

NPV/ IRR Calculator-

Optimization of cash flow/ revenue flow concerning all fixed and variable investments throughout the product development process. The rate of return on an investment is a critical indicator to ensure the profitability and break-even of the project.

Competitive Landscape:

The competitive landscape includes country-specific approved as well as pipeline therapies. Any asset/product-specific designation or review such as Orphan drug designation, Fast track, Priority Review, Breakthrough Therapy Designation, Rare Pediatric Disease Designation, and Accelerated Approval are tracked and supplemented with analyst commentary.

Clinical Trial Assessment-

Detailed clinical trial data analysis and critical product positioning include trial design, primary outcomes, secondary outcomes, dosing and schedules, inclusion and exclusion criteria, recruitment status and essentially covers the reported adverse events. Majorly the trial analysis helps determine the potential of the critical assets and their probable filing and launch date.

Unmet Medical Needs Overview-

This report presents the most important clinical unmet needs in the treatment, according to Thelansis research and analysis. Other essential unmet needs identified through our study include decreased cost burden on patients, improved administration convenience, and improved patient compliance.

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