Unique challenges require unique approaches to development
Most people are familiar with the leading causes of morbidity and mortality in the United States—heart disease, cancer, and diabetes.
Rare diseases affect fewer than 1 in 2000 people. Each one affects only a small number of patients. Yet, there are over 7000 rare diseases. And, there are no treatments for 95 percent of them.
The concept of evaluating the value of pharmaceutical products is not new though the changing dynamics of the healthcare system have brought it to center stage. 薬価と最適な損益分岐点の関数として市場における需要を単純に判断する時代は既に過去のものです。
The state of gene therapy faces much uncertainty as recent regulatory and clinical setbacks have raised questions about its promise. With only two gene therapies FDA-approved for rare diseases to date, the path to developing these medicines is still a difficult one.
Achieving regulatory approval alone no longer determines a drug’s or therapy’s commercial success or even guarantees its market launch. Today, each product must be evaluated from a value perspective by payers and health authorities to be placed on the formulary, factored into reimbursement rates, and put into treatment plans before it is available for healthcare providers to prescribe.
The National Institute of Diabetes and Digestive and Kidney Diseases considers nonalcoholic fatty liver disease (NAFLD) as a condition wherein the liver stores excess fat and nonalcoholic steatohepatitis (NASH) as one type of NAFLD. Individuals with obesity and type 2 diabetes appear to be at greater risk of developing NAFLD.
350 million patients worldwide suffer from 7,000 rare diseases, yet only 300 of these diseases have approved treatments. This gap, impacting 95% of rare disease patients, represents a huge unmet medical need. 希少疾患治療薬（オーファンドラッグ）の開発には、臨床・規制・商業面において多様な課題を伴います。