The Accelerated Access Review (AAR) is being undertaken by the Department for Life Sciences, with th…
The Accelerated Access Review team released their Interim Report in October 2015, setting out 5 propositions to accelerate access to ‘transformative health technology’. In our response to the interim report, we highlight some of the key concerns for patients with rare, genetic and undiagnosed conditions.
The key thing for our community, unsurprisingly, is to get effective treatments. Foremost this means starting right at the beginning, making sure that researchers are looking at the aspects of conditions that patients feel most strongly about. The key to this is patient involvement – making sure that patients are consulted right from the start.
The way treatments are assessed by commissioners, when they are finally ready to be accessed through the NHS, is often the most visible barrier to accessing medicines. In our response to the interim report, we make clear that the health economist’s toolbox needs updating to give patients with rare and genetic conditions access to new treatments.
In our response we discuss the missed opportunity that is value-based assessments of new medicines, and the places that rare disease patients are let down by QALYs and EQ-5Di assessments. Using the results from our Citizen’s Jury on weighing the risks and benefits of new medicines for serious conditions we address one of the fundamental problems with the way that commissioners evaluate medicines. The foremost question that regulators attempt to answer is 'do the benefits outweigh the risks?' We have identified that patients with rare and genetic conditions often perceive risk and benefit differently from the general public. As a result we must ensure that patient voice is placed at the centre of assessments.
We also call for clarity over how and why certain appraisal routs are chosen over others. There appears to be no cohesion between the different pathways and the system is over complicated with many different routes to access.