Question to the Department of Health and Social Care:

To ask Her Majesty’s Government what encouragement they are giving to the pharmaceutical industry to develop new treatments for patients with rare diseases.

The Government continues to implement its Strategy for UK Life Sciences, delivering its long-term programme of action to improve the wider environment for life sciences companies and overcome the barriers that allow discoveries to be translated into new treatments for patients with rare conditions.

We are making substantial investment in infrastructure for research into rare diseases and experimental medicine through the National Institute for Health Research (NIHR).

The NIHR BioResource, launched in March this year, includes a focus on rare diseases where ongoing genotyping of volunteers will speed up research into new treatments.

The NIHR Rare Diseases Translational Research Collaboration, launched alongside the UK Rare Diseases Strategy with an investment of £20 million over four years, is making good progress in developing a better understanding of the physical characteristics of rare diseases and supporting our drive for new diagnostics and treatments for patients with rare diseases.

The 100,000 Genomes Project is focusing on rare diseases and offers the strongest prospect of patient and scientific benefits and the ability to drive the transformation of the National Health Service in terms of application of genomic medicine. Furthermore, the application of whole genome sequencing may enable major new biological insights into the genetic make-up of rare diseases that will, in time, lead to new diagnostics and therapeutic innovation.

In March 2014, we announced the Early Access to Medicines Scheme to support access in the United Kingdom to promising new, unlicensed or off-label medicines in areas of unmet clinical need for patients with life threatening or seriously debilitating conditions without adequate treatment options. The Medicines and Healthcare Products Regulatory Agency has now issued two Promising Innovative Medicines Designations which is the first step of the scheme.

We are also commissioning an external review of the pathways for the development, assessment, and adoption of innovative medicines and medical technology. This review will consider how to speed up access for NHS patients to cost-effective new diagnostics, medicines and devices.