Value Frameworks For Rare Diseases: Should They Be Different?

The US health care system is increasingly focusing on value as a basis for reimbursement of pharmacotherapies and devices, and as a result the use of “value frameworks” for measuring and comparing treatment value has grown in recent years. However, the therapies assessed by most frameworks frequently apply to modest-to-large disease populations, rather than the smaller populations affected by rare diseases, where the factors driving value may differ. Rare diseases are different from diseases affecting larger populations in several fundamental ways. In the United States, a rare disease is defined as one that affects fewer than 200,000 people a year, or one that affects more than 200,000 people per year but for which the costs of drug development are expected to exceed revenues for the product. Because rare diseases are frequently characterized by their high complexity, small patient populations that limit the speed of clinical trial enrollment, and limited availability of clinical experts, the development of therapies for rare diseases is particularly challenging. Within a small market of patients for which the per-patient costs of research and development may be sizable, higher per-patient prices are needed to generate sufficient investment returns on successful innovations. The difficulty of justifying much higher prices may result is an “unmet need trap” that impedes innovation and leaves behind a significant number of people who do not have adequate...
Source: Health Affairs Blog - Category: Health Management Authors: Tags: Costs and Spending Drugs and Medical Innovation Quality Orphan Drug Act Precision Medicine rare disease treatment treatment value value frameworks Source Type: blogs

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Publication date: Available online 13 December 2019Source: Academic RadiologyAuthor(s): Farideh Mohtasham, Jamal Rahmani, Yousef Khani, Siamak Sabour
Source: Academic Radiology - Category: Radiology Source Type: research
Conclusion: Although left-sided gallbladder is a very rare disease, it is possible to diagnose it preoperatively and perform laparoscopic cholecystectomy safely by adjusting port position. The common important features of left-sided gallbladder include distribution of the left portal vein crossing over to the right side of the liver and increased size of the left portal vein. These variations may have important clinical implications in the management of hepatic resection including donor hepatectomy. PMID: 31824885 [PubMed]
Source: Annals of Surgical Treatment and Research - Category: Surgery Tags: Ann Surg Treat Res Source Type: research
Authors: Knight O, Carter CR, Loranger B, Rea PM Abstract Pancreatic cancer is a leading cause of cancer related deaths in the UK. However, public knowledge and understanding of the pancreas is generally poor, therefore pancreatic cancer patients often have to contend with understanding large quantities of new information at a pivotal time in their lives.Despite utilisation of digital visualisation techniques in medical education, very rarely are they being used to help clinicians communicate information to their patients. Specifically, there is no literature describing use of an interactive digital application for...
Source: Advances in Experimental Medicine and Biology - Category: Research Tags: Adv Exp Med Biol Source Type: research
Recent advances in molecular studies, especially genome-wide analyses, have revealed the landscape of genomic alterations present in endometrial carcinomas, and have provided valuable insight into the pathogenesis of this disease. The current challenges are in developing a molecular-morphologic classification system to enhance traditional pathologic diagnosis and in determining the optimal approach to using this new information to guide clinical management. Molecular assays may be particularly beneficial in allowing the earlier detection of endometrial cancer or precursor lesions and in guiding personalized treatment appro...
Source: International Journal of Gynecological Pathology - Category: Pathology Tags: PATHOLOGY OF THE CORPUS: REVIEW ARTICLE Source Type: research
Cowden syndrome (CS) is a multiple hamartoma syndrome associated with the development of various tumors, including endometrial cancer. However, the histology of CS-associated endometrial cancer remains to be fully described. To our knowledge, this is the first report of a patient with CS having endometrial endometrioid carcinoma with ovarian metastasis demonstrating morula-like features. A 31-yr-old, nulliparous, Japanese woman presented with abnormal genital bleeding. Endometrial biopsy revealed endometrioid carcinoma with an extensive morular formation, partially resembling atypical polypoid adenomyoma (APAM). Moreover, ...
Source: International Journal of Gynecological Pathology - Category: Pathology Tags: PATHOLOGY OF THE CORPUS: CASE REPORT Source Type: research
Brain metastasis is a rare and generally late manifestation of an advanced-stage, high-grade serous ovarian carcinoma. Nowadays, the improved control of intra-abdominal disease by surgery and platinum-based chemotherapy results in a longer survival, allowing distant metastasis to implant and grow in the brain parenchyma. Herein, we describe a unique case of a cerebellar metastasis from clear cell ovarian carcinoma that initially presented as a FIGO Stage IC cancer. Surprisingly, 6 mo after surgery, the patient was in good condition with complete disappearance of symptoms and no evidence of recurrence. This relativel...
Source: International Journal of Gynecological Pathology - Category: Pathology Tags: PATHOLOGY OF THE UPPER TRACT: CASE REPORTS Source Type: research
No abstract available
Source: International Journal of Gynecological Pathology - Category: Pathology Tags: ONLINE ARTICLES: LETTERS TO THE EDITOR Source Type: research
Journal of Women's Health, Ahead of Print.
Source: Journal of Women - Category: OBGYN Authors: Source Type: research
The active ingredient in psychedelic mushrooms is called psilocybin -- and a new study suggests it could safely be used as a therapeutic approach to treat depression when other drugs have failed to work.
Source: CNN.com - Health - Category: Consumer Health News Source Type: news
FDA officially has a new boss. The U.S. Senate voted 72-18 on Thursday to confirm Stephen Hahn, MD, as the new FDA Commissioner. The news was largely well received by industry advocacy groups like AdvaMed and professional societies like the American Heart Association. “Dr. Hahn is an excellent choice to lead FDA, and we congratulate him on his swift and overwhelming bipartisan confirmation," said AdvaMed CEO Scott Whitaker. "During the confirmation process, Dr. Hahn demonstrated his deep understanding of the agency’s mission to pr...
Source: MDDI - Category: Medical Devices Authors: Tags: Regulatory and Compliance Source Type: news
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