final-logo-genetic-alliance-uk-colour-jpgToday Genetic Alliance UK launched a landmark Patient Charter that highlights the concerns patients have about the new approach the Government and NICE are using to determine whether or not medicines for very rare conditions are made available through the NHS.  Here they write to explain more.

The new process at NICE is the Highly Specialised Technologies (HSTs) evaluation programme. Following a workshop hosted by Genetic Alliance UK, patient representatives expressed their concerns that, while greatly needed, the existing HST evaluation process is opaque, potentially disruptive to the wider NHS and risks downplaying the patient voice.

The Patient Charter sets out a list of recommendations to improve the interim framework and ensure that it is fit for purpose. 77 patient groups signed up to the Charter to endorse the recommendations. This is the first time that so many patient groups from across the rare disease community have come together with a united call for change.

The launch took place at a breakfast meeting hosted by the BIA and ABPI at their headquarters in central London. It was chaired by Alastair Kent OBE (Director, Genetic Alliance UK). On the panel were Steve Bates (BIA), Josie Godfrey (NICE), Dr Robin Lachmann (National Hospital for Neurology and Neurosurgery) and Frances Macdonald (ABPI).

Alastair Kent OBE, Director of Genetic Alliance UK, said: “The public have consistently given support to the concept of providing highly priced medicines to patients with rare diseases. It is right that we have the HST evaluation process, but given what is at stake, it is critical that the process is fit for purpose. It needs to be transparent, it needs to integrate with the health service, and the patient voice needs to be central to the entire process.”

Steve Bates, BIA Chief Executive Officer, said: “Genetic Alliance should be commended for the launch of this Patient Charter which articulates many of the core areas that all stakeholders interested in the development of new, innovative medicines for patients with rare and very rare diseases need to be concerned with. In particular, how NICE chooses which medicines go through this new evaluation route through topic selection will be incredibly important, not only for the many patients who want to access new medicines but for the developers of those products who need predictability and transparency.”

The key recommendations in the Patient Charter included:

  • Ensuring there was a continued dialogue between NICE and NHS England to prevent NICE recommendations disrupting access to other, currently prescribed treatments by pushing them beyond the NHS budget.
  • Improving the transparency of the process during topic selection – the stage which determines which medicines will or will not be evaluated through the HST process. In comparison to the remainder of HST evaluation, topic selection is a black box. Patients are neither involved in the process nor able to access the deliberations of NICE’s topic selection committee.
  • Updating the criteria used in topic selection, in particular the interpretation of the term ‘clinically distinct’, which does not currently recognise patient subgroups defined by symptoms, genetics or biomarkers, meaning many medicines for rare diseases are currently ineligible for HST evaluation.
  • Calling for NICE to provide third-party, contracted support for patient communities involved in HST evaluations. If a patient group does not exist, or if the group is small, poorly resourced or unable to produce an evidence submission alone, NICE risks the patient voice being absent unless targeted support is provided. As yet, NICE has made no provision for this.

You can see more in the BIA’s recent report on the evaluation of medicines for rare and very rare diseases.