Press Release: Genetic Alliance UK welcomes new fund launched by
the Scottish Government for individual patients needing orphan
drugs
The Scottish Government has announced that it is launching a fund to cover the cost of medicines for
individual patients with rare conditions, which are not available for routine prescription. £21 million
will be invested to pay for the cost of medicines known as “orphan drugs”. These are medicines for
illnesses which affect fewer than the EU’s definition of 1 in 2,000 people.
Genetic Alliance UK welcomes this announcement as it will go some way towards addressing the
difficulties which those affected by rare diseases in Scotland have in accessing appropriate
treatments. Genetic Alliance UK’s Director Alastair Kent said: “we welcome this initiative as it means
patients living in Scotland will be able to access and receive medicines that will make a substantial
difference to their lives.”
Genetic Alliance UK has been campaigning in this area for several years and will continue to do so to
ensure the fund is used to its best effect and to ensure that all patients affected by rare diseases get
access to the treatments they need.
Only those medicines not recommended for routine use by the Scottish Medicines Consortium will be
covered by the fund. The fund will be available from March this year through to April 2014 and will
help to cover successful new Individual Patient Treatment Requests. The £21 million is in addition to the
current board funding allocations.
The Scottish Medicines Consortium (SMC) appraises all newly licensed medicines and provides advice
to NHS Boards on their clinical and cost-effectiveness.
Currently, if a medicine is accepted for use by the SMC then individual health boards set the criteria
for prescribing it. If the medicine is not accepted then health boards do not make it routinely
available. However, clinicians can prescribe medicines that are not accepted for routine use by the
SMC for individual patients in certain circumstances by special request through Individual Patient
Treatment Requests (IPTR).
Patients with a rare disease often experience difficulties in achieving a successful IPTR outcome as the
IPTR referral criteria are extremely difficult to satisfy for orphan medicines and are more likely to
result in rare disease patients with the greatest clinical need being refused access to potentially life
changing treatments. Genetic Alliance UK and Rare Disease UK have been campaigning for a
thorough and transparent review into the current IPTR process and has been assisting the Scottish
Parliament Health and Sport Committee with their inquiry into access to medicines. Whilst we welcome
the Scottish Government’s announcement of a £21million fund for rare disease, we will continue to
campaign for a fairer IPTR process in Scotland to ensure patients with rare diseases are able to
access this fund successfully.
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Genetic Alliance UK:
Unit 4D, Leroy House, 436 Essex Road, London, N1 3QP
020 7704 3141
contactus@geneticalliance.org.uk
Charity registered in England and Wales (no. 1114195) and in Scotland (no. SC039299)
Company limited by guarantee, registration no: 05772999
www.geneticalliance.org.uk
Genetic Alliance UK looks forward to the outcome of the Health and Sport Committee inquiry and the
Scottish Government Review and are hopeful that it will result in a fairer system for access to new
medicines for rare diseases.
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Genetic Alliance UK:
Unit 4D, Leroy House, 436 Essex Road, London, N1 3QP
020 7704 3141
contactus@geneticalliance.org.uk
Charity registered in England and Wales (no. 1114195) and in Scotland (no. SC039299)
Company limited by guarantee, registration no: 05772999
www.geneticalliance.org.uk