HTAs and access to rare diseases therapies: How can clinicians assist in the healthcare assessment of treatments for patients with primary immune deficiencies?

Abstract

1. Background Health Technology Assessments (HTA) in terms of therapies depend on established treatments for defined conditionswith provenoutcomes; the various treatment regimes can be compared with each other or with outcomes in the absence of treatment, in various healthcare settings. For example in the UK, the National Institute for Health Research – NIHR states that “The HTA programmeproducesindependent research about the effectiveness of different healthcare treatments ...for those who use, manage and provide care in the NHS”. Given limited financial resources, it is appropriate that HTA agencies are increasingly involved in assessment of the cost effectiveness of well -established therapies, includingthose for rare diseases. Ultimately HTAs are mechanismsthat help funders and governments to control healthcare budget expenditure. An excellent example is therecentdecisionoftheIrishAuthority’sHealthTechnologyAssessmentDirectorate “not to introduce a new technology to screen donated blood for Creutzfeldt-Jakob disease because it would not be cost effective to do so”. Such tools are widely used, though for rare diseases the background is often poorly understood. However their popularity is exemplified in the Journal Citation Reports for 2009, which shows that "Health Technology Assessment", the journal of the HTA programme, has seen a significant rise in its impact factor rising from 5.01 last year, to 6.91. In the current world-wide economic climate, clinicians, patients, healthcare providers and those with the budgets need to understand the economic strategy of HTAs. This is particularly true for those involved with rare diseases, such as primary immune deficiencies (PIDs), and applies in countries with good access to PID centres of excellence that may be at risk, as well as in those in which therapies are restricted for financial reasons. The role of HTAs in assessing the efficacy and cost effectiveness treatments for patientswithPIDsisdifficultforavarietyofreasons: difficultiesinsettingupclinical