5 results
Search Results
2. Eltrombopag for the Treatment of Chronic Immune or Idiopathic Thrombocytopenic Purpura: A NICE Single Technology Appraisal.
- Author
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Boyers, Dwayne, Xueli Jia, Jenkinson, David, and Mowatt, Graham
- Subjects
- *
IDIOPATHIC thrombocytopenic purpura , *CLINICAL trials , *RITUXIMAB , *QUALITY-adjusted life years - Abstract
The National Institute for Health and Clinical Excellence (NICE) invited the manufacturer of eltrombopag (GlaxoSmithKline) to submit evidence for the clinical and cost effectiveness of this drug for the treatment of patients with chronic immune or idiopathic thrombocytopenic purpura (ITP), as part of the their Single Technology Appraisal (STA) process. The Aberdeen Technology Assessment Review (TAR) Group, commissioned to act as the evidence review group (ERG), critically reviewed and supplemented the submitted evidence. This paper describes the company submission, the ERG review and NICE's subsequent decisions. The ERG critically appraised the clinical and cost-effectiveness evidence submitted by the manufacturer, independently searched for relevant literature, conducted a critical appraisal of the submitted economic models and explored the impact of altering some of the key model assumptions as well as combining relevant sensitivity analyses. Three trials were used to inform the safety and efficacy aspects of this submission; however, one high-quality randomized controlled trial (RAISE study) was the principal source of evidence and was used to inform the economic model. Eltrombopag had greater odds of achieving the primary outcome of a platelet count between 50 × 10^9/L and 400 × 10^9/L during the 6-month treatment period than placebo (odds ratio [OR] 8.2, 99% CI 3.6, 18.7). In the eltrombopag group, 50/83 (60%) of non-splenectomized patients and 18/49 (37%) of splenectomized patients achieved this outcome. The median duration of response was 10.9 weeks for eltrombopag (splenectomized 6 and non-splenectomized 13.4) compared with 0 for placebo. Eltrombopag patients required less rescue medication and had lower odds of bleeding events for both the splenectomized and the non-splenectomized patients. For a watch-and-rescue strategy of care, the comparator was placebo and the ERG found that substantial reductions in the cost of eltrombopag are needed before the incremental cost per QALY is less than d30 000. There was significant uncertainty, with the incremental cost-effectiveness ratio (ICER) reported varying from d33 561 to d103 500 per QALY (splenectomized) and d39 657 to d150 245 per QALY (non-splenectomized). All costs are presented in d, year 2008 values, as this was the costing year for the manufacturer's model. Other than bleeding, no adverse events were modelled. In relation to the long-term treatment model, the ERG questioned the robustness of the use of non-randomized non-comparative data. The base-case results restricting the time horizon to 2 years and prescribing eltrombopag as second-line treatment post-rituximab were found to be favourable towards eltrombopag. As rituximab is not a licensed treatment for ITP, the ERG were concerned that its inclusion may not be reflective of clinical practice. None of the treatment sequences resulted in an ICER approaching the recommended threshold of d30 000 per QALY gained. Eltrombopag appears to be a safe treatment for ITP (although longterm follow-up studies are awaited) and has short-term efficacy. However, NICE found based on the evidence submitted and reviewed that there was no robust evidence on the long-term efficacy or cost effectiveness of eltrombopag and a lack of direct evidence for eltrombopag tested against other relevant comparators. [ABSTRACT FROM AUTHOR]
- Published
- 2012
- Full Text
- View/download PDF
3. Value of Information and Pricing New Healthcare Interventions.
- Author
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Willan, Andrew R. and Eckermann, Simon
- Subjects
- *
MEDICAL care costs , *CLINICAL trials , *DECISION making , *PUBLIC spending - Abstract
Previous application of value-of-information methods to optimal clinical trial design have predominantly taken a societal decision-making perspective, implicitly assuming that healthcare costs are covered through public expenditure and trial research is funded by government or donation-based philanthropic agencies. In this paper, we consider the interaction between interrelated perspectives of a societal decision maker (e.g. the National Institute for Health and Clinical Excellence [NICE] in the UK) charged with the responsibility for approving new health interventions for reimbursement and the company that holds the patent for a new intervention. We establish optimal decision making from societal and company perspectives, allowing for trade-offs between the value and cost of research and the price of the new intervention. Given the current level of evidence, there exists a maximum (threshold) price acceptable to the decision maker. Submission for approval with prices above this threshold will be refused. Given the current level of evidence and the decision maker's threshold price, there exists a minimum (threshold) price acceptable to the company. If the decision maker's threshold price exceeds the company's, then current evidence is sufficient since any price between the thresholds is acceptable to both. On the other hand, if the decision maker's threshold price is lower than the company's, then no price is acceptable to both and the company's optimal strategy is to commission additional research. The methods are illustrated using a recent example from the literature. [ABSTRACT FROM AUTHOR]
- Published
- 2012
- Full Text
- View/download PDF
4. Recruiting children into cancer trials--role of the United Kingdom Children's Cancer Study Group (UKCCSG).
- Author
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Ablett, S, Pinkerton, C R, and United Kingdom Children's Cancer Study Group (UKCCSG)
- Subjects
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CANCER , *ASSOCIATIONS, institutions, etc. , *TUMOR treatment , *CLINICAL trials , *MEDICAL referrals , *ONCOLOGY , *PATIENT participation , *ACQUISITION of data , *PATIENT selection - Abstract
The UK Children's Cancer Study Group (UKCCSG), established in 1977, provides a highly organised structure for both service provision and research, and represents the model to which the adult cancer community is currently aspiring. Since childhood cancer is so rare, it is both essential and feasible for the vast majority of children to be referred into the network of specialist centres, and also for the maximum number of children to be recruited into national and international clinical trials. Over the last 30-40 years there have been major advances in treatment, such that now approximately 70% of children diagnosed with cancer will be cured of their disease. The conduct of clinical trials in this patient population does, however, raise a number of specific issues and these are discussed in the paper. [ABSTRACT FROM AUTHOR]
- Published
- 2003
5. Advances in orthodontic anchorage with the use of mini-implant techniques.
- Author
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Cousley, R. R. J. and Sandler, P. J.
- Subjects
- *
ORTHODONTICS , *DENTAL implants , *CLINICAL trials , *DENTISTS - Abstract
Orthodontic mini-implants (OMIs) represent a new form of anchorage provision and appear to provide a variety of benefits for both anchorage-demanding and complex orthodontic cases. This paper reports the latest perspectives on OMIs in terms of the emerging clinical evidence base coupled with their varied clinical applications. [ABSTRACT FROM AUTHOR]
- Published
- 2015
- Full Text
- View/download PDF
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