New Medicines Approval
Why aren’t all new medicines used in Scotland?
The Scottish Medicines Consortium (SMC) carries out an evaluation of the clinical and cost effectiveness of all newly licensed drugs and advises Health Boards and their Area Drug and Therapeutics Committees, on their use in NHS Scotland. This process has to be seen to be fair and understandable.
Everyone wants to have the most effective medicines for themselves and their families but unfortunately all health services have limited budgets. This means that difficult choices have to be made around what treatments can be bought with health service funds. This is an uncomfortable fact but most people will agree with it. For example a medical treatment costing a million pounds resulting in just one patient living one day longer would not be acceptable to the majority of people. If the same expenditure gave a thousand people five years extra life it may be acceptable. In real life the choice is not so clear-cut but it has to be made.
SMC’s procedure for evaluating new medicines
When new medicines are licensed for use the manufacturers are asked to submit details of the medicine to SMC. A licence indicates that the medicine works and is safe to use for specific conditions but it does not imply that it is good value for money or more effective than other medicines.
Pharmaceutical companies submit details of their new medicines, including results of drug trials and costs, to the SMC. The New Drugs Committee (NDC) of the SMC examines the submission with the assistance of medical, pharmaceutical, statistics and economics experts. Provisional advice from the NDC is passed to the SMC and the pharmaceutical company concerned. This advice and feedback from the manufacturer is then considered at the next monthly SMC meeting. Patient Interest Groups are also encouraged to make a submission to the SMC, focussing on the difficulties the disease presents for patients and the place of the medicine in addressing patient needs.
At the SMC meeting the submissions are considered in detail. Will the medicine be effective? Are current treatments better? Does the medicine give value for money compared to existing treatments? These are the main questions asked. Members of the SMC appointed from the general public represent patient interests at the meeting. They also present Patient Interest Group submissions to the meeting.
An important issue is how health benefits to patients are captured in the economic analysis. A widely used economic indicator is the “Quality Adjusted Life Year” (QALY). A treatment giving an extra year of good quality life to a patient has a QALY of 1 whereas an extra year of life with pain and disability could have a QALY of 0.5. The cost per QALY of a medicine is commonly used when evaluating the economics of a treatment.
Shortly after a submission from the manufacturer has been evaluated the results are published in the form of a Detailed Advice Document. This is posted on the SMC website about four weeks after the SMC meeting. The whole process from submission to publication takes around eighteen weeks.
Reasons for non-approval of a medicine
Some of the most common reasons for SMC not approving a medicine for use are shown below.
- Non-submission.
Medicines can only be approved if the manufacturer makes a submission to the SMC. If the manufacturer does not make a submission then SMC will advise that use of the product cannot be recommended. - Inappropriate comparator.
During drug trials a new medicine may be tested against a placebo (a substance that contains no medication at all) or a currently used medicine (comparator) to see if it is better. If the comparator medicine is not the one normally used in Scotland then the submission may be rejected even though the new medicine appears to work. Pharmaceutical companies may be asked to repeat the submission with an appropriate comparator. - No better than a cheaper medicine.
Sometimes a medicine is produced which is as effective as one that is currently used but is no better and more expensive. In this case it would probably not be recommended. - Not sufficiently strong economic case.
This is a common reason for non-approval and one which is often misunderstood. Very high cost per QALY figures may be why the economic case is rejected. Sometimes it is because the SMC feels that wrong assumptions are made in the submission. In this case the company may decide to resubmit to address any shortcomings that SMC may have highlighted in the economic case and which prevented them from accepting the medicine for use.
Scottish Medicines Consortium and the public
The SMC encourages public interest in its work. Its website www.scottishmedicines.org.uk contains details of how the SMC works and a timetable of future submissions. The Detailed Advice Documents for each submission can be seen on the website.
An individual submission of evidence from either a patient or carer cannot be considered by SMC. Individuals interested in expressing patient or carer views on a medicine that SMC has scheduled for assessment are encouraged to contact a relevant Patient Interest Group. These individual responses form part of the evidence in the Patient Interest Group submission to SMC. All Patient Interest Group Submissions are carefully considered and they do make a difference to the approval process.
Compiled by the Patient Public Involvement Group of SMC
October 2008