Charter for improvements in rare disease drug evaluation launched

77 patient groups, including Aniridia Network UK are calling for improvements in rare disease drug evaluation in a ground breaking new report. This is the first time so many patient groups have come together to set out a united case for change.

Today Genetic Alliance UK launch 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 rare conditions are made available through the NHS.

The Patient Charter, sets out a list of recommendations to improve the way medicines for rare conditions are evaluated.  Published by Genetic Alliance UK it shows patients are concerned that, while greatly needed, the existing process is opaque, potentially disruptive to the wider NHS and risks downplaying the patient voice.

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.

77 patient groups agree on these 29 recommendations for progress. Never before have we seen so many patient groups from across the rare disease community come together with a united call for change. Let’s use this is as strong starting point from which to move forward and develop the evaluation process that the rare disease community needs.”

The Patient Charter is launched today (Wednesday 2nd April) at a breakfast meeting hosted by the Association of the British Pharmaceutical Industry (ABPI) at their headquarters in central London. Present will be representatives from patient groups, the National Institute for Health and Care Excellence (NICE), NHS England, clinicians, the pharmaceutical industry and other stakeholders.

The meeting is being chaired by Alastair Kent OBE and on the panel are

  • Steve Bates (Chief Executive Officer, Bioindustry Association)
  • Josie Godfrey (Associate Director, NICE)
  • Dr Robin Lachmann (National Hospital for Neurology and Neurosurgery)
  • Frances Macdonald (Association of the British Pharmaceutical Industry).

Commenting on the publication of Genetic Alliance UK’s Patient Charter, ABPI Director of Value and Access Paul Catchpole said:

“The ABPI welcomes the publication of Genetic Alliance UK’s Patient Charter to support further stakeholder engagement around the development of the NICE Highly Specialised Technology (HST) Programme. Providing more meaningful and extended opportunities for patient involvement in decision making about medicines is very important. The Patient Charter will be particularly helpful when NICE reviews its interim processes and methods for the HST programme after it has gained more experience of the programme and a number of medicines have completed evaluations using the new process.”

The Patient Charter is a clear and unified communication of patients’ perspectives and priorities on NICE’s interim Highly Specialised Technology (HST) evaluation framework through the eyes of patients affected by those diseases most likely to require an HST evaluation for newly developed medicines in order for them to be accessed through the NHS.

The Patient Charter is intended to directly inform NICE in advance of its planned review of the HST evaluation framework, which it has committed to undertake before the end of 2014, and to provide a future reference point for the patient view on this issue. The recommendations have been endorsed by 77 patient groups who support the need for change.

Aniridia Network UK is a member of Genetic Alliance UK. With drugs such as potentially curing START eye drops currently being researched these issues are becoming increasingly relevant to people with aniridia.

For more information visit Genetic Alliance UK

About Aniridia Network

A charity support group for people with the genetic visual impairment aniridia and their families in the UK and Ireland. Our vision is that people with/associated with aniridia are hopeful, confident, supported and well informed regarding aniridia. Founded in 2000. First registered as a charity in 2011 and fully in 2018.
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