GAO on the cost of rare diseases-healthcare economist

Last month, the Government Accountability Office issued a report on Rare disease costsThis report is very interesting, especially in Appendix 1, which contains a detailed list of rare disease cost studies on about 50 rare diseases. Here are some highlights:

Currently, there is no cure or cure for most rare diseases. Although the federal government encourages the development of products to treat these diseases, only about 5% of rare diseases receive FDA-approved treatments… Even if a drug can be used off-label, NIH officials explained that the drug can only be provided to patients if a doctor A rare disease is prescribed (they may be reluctant to do so if there is no clear evidence that it is effective for the patient’s specific condition), and if the drug is prescribed, insurance may not cover it.

The above-mentioned incentives include the Orphan Drug Act (ODA), which provides tax credits and exclusive marketing rights (except for treatment of non-rare diseases) to encourage the development of drugs for rare diseases.

Being diagnosed with a rare disease can also be challenging.

…Because there are no treatments for many rare diseases, testing for them may not be considered to affect health outcomes. Therefore, some insurance companies may not cover them on the grounds that they are “not medically necessary”. For example, whole-exome sequencing—a method of identifying variations in protein coding regions of genes—has been shown to be useful for detecting patients with suspected genetic diseases, but insurance companies may not cover its use.

A typical cost-benefit model quantifies the value of diagnosis based on how the new information provided by the diagnosis changes treatment behavior and the final health outcome. However, patients themselves may take very seriously just knowing that they have a certain disease. Lakdavala and so on. (2018) Call it “the value of reducing uncertainty due to a new diagnosis.”Part of the reason is that no diagnostic tests were performed. On average, patients with rare diseases visited 4.2 PCPs, 4.8 specialists, 3.7 emergency room visits, and 1.7 hospitalizations for disease-related reasons. Before being diagnosedSome of the challenges that were diagnosed are shown in the figure below.

Unsurprisingly, the cost of treating rare diseases is high.A summary or cost of rare diseases in rare diseases is National Economic Burden of Rare Disease ResearchThe study found that the total annual economic burden of rare diseases is US$966 billion.

The cost of rare diseases varies depending on a variety of factors, as shown in the table below.

please read Whole report Learn more.

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