On 23rd March, Genetic Alliance UK launched a new patient charter on access to medicines for rare disease patients in Scotland. This week’s blog highlights the important recommendations generated from the project, where a range of patient organisations gave their perspective.

Genetic Alliance UK is an alliance of over 180 patient organisations, set up to improve the lives of patients and families affected by genetic conditions. By working with patient organisations, they are able to get a broad range of patient perspectives and really understand how the patient is affected by current processes. Their new patient charter gathered views from a range of patient organisations on access to medicines for rare disease patients in Scotland. Our very own Patient Support Manager Lesley contributed to the charter, giving a valuable perspective on the issues affecting AKU patients.

Photo courtesy of Genetic Alliance/ Claire Tennant photography

How do Scottish AKU Patients Currently Access Treatment?

While there is currently no licensed treatment for AKU, patients attending the National AKU Centre are able to access nitisinone off-license. Fortunately NHS Scotland agreed to fund patients attending the centre and Scottish patients are referred via their GP. This is great news for our Scottish AKU patients, however the appraisal process for rare disease licensed medication is different in Scotland and we will continue to push for service improvements for all rare disease patients.

How Does a Patient Access a New Medicine on the NHS in Scotland?

The Scottish Medicines Consortium (SMC) appraises all newly licensed prescription medicines to decide whether the medicine will become available on the NHS. The appraisal considers several factors including effectiveness, current available treatments and value for money. Patient groups are encouraged to contribute to the process, giving a crucial patient perspective on how the medicine addresses their needs.

A review of the current Scottish process began in 2013 in response to 3 petitions from rare disease representatives. As a result, the SMC recognised orphan and ultra-orphan drugs should be assessed differently to other drugs due to high cost, limited data and greater levels of uncertainty. The SMC now considers additional factors when assessing orphan drugs, including improvement in quality of life and absence of alternative options.

Accessing rare disease medication has improved since the 2013 review, however Genetic Alliance found that while there has been a major increase in the number of assessments and approvals, the actual percentage of approvals went down slightly. Clearly more needs to be done and the suggested recommendations in the new patient charter offer solutions to some of the flaws in the current process.

Photo courtesy of Genetic Alliance/ Claire Tennant photography 

Patient Charter Recommendations

  • 1. There is a need for an increase in representation and type of patient voice on all decision-making panels

Patients provide an important and unique perspective on the evaluation process. Despite efforts to involve patients, their role is not clearly defined and it is thought that their input would be more valuable if made in person.

  • 2. Greater clarity as to the role of patient responses in Scottish Medicines Consortium decision-making is necessary

Patient representatives remain uncertain as to how their submission affects the decision making process and there is a clear need to communicate this back to them. It is also recommended that further training and support should be offered to patients groups to ensure their submission is fit for purpose.

  • 3. The New Medicines Fund should be retained with increased transparency on its use

While funds for the New Medicines Fund doubled in 2015/16, there are concerns over the sustainability of this fund.

  • 4. Clarity on the process and timeframe of the transition to Peer Approved Clinical System (PACs) is urgently needed

The introduction of PACs, a new system for accessing drugs in exceptional circumstances has been delayed. The details of this delay are currently unknown and a timescale for implementation is urgently needed.

  • 5. Careful monitoring of health board uptake of new medicines to local formularies may be necessary

This monitoring is important to ensure that everyone is following the correct processes and that a decision is reached in the 90 day period.

The reforms that occurred as a result of the 2013 review provided a valuable step forward for Scottish rare disease patients, with a large increase in the number of approvals. Yet clearly there is still scope for greater change and it is hoped that these 5 clear recommendations from the new patient charter will encourage service improvements for rare disease patients in Scotland.

Genetic Alliance are asking patient groups to endorse the recommendations made in the charter to help raise the volume of their voice. Email louise.coleman@geneticalliance.org.uk for more information.