Rapid regulator decision making

The second presentation of the day was given by Keith Tolley, a health economist working with the Scottish Medicines Consortium (SMC) who gave a detailed insight into the workings of the SMC in assessing new drugs and the role of external experts. Despite geographical constraints, the influence of the SMC extends beyond Scotland and Tolley mentioned instances where SMC rulings have been used to inform access decisions in the UK, in the absence of NICE guidance.

The SMC is one of three health technology appraisal boards. Although broadly analogous to NICE, the SMC differs from NICE in its scope of interest and the speed of its decision making. Unlike NICE, which is selective in its appraisals, the SMC covers all new pharmaceuticals, new formulations and new indications. The SMC aims to provide guidance as close as possible to product launch.

The principal distinction of the SMC is the speed of its assessment process, with decisions typically reached within 4 months of submission. Three possible decisions are used - accepted for use, restricted use or not accepted for use. Restricted use can encompass limitations of dosage, or restrictions to second-line usage for instance. In the last couple of years about 20% were accepted, 30% given limited use and 50% were not accepted for use

Submissions have a 2-part process comprising assessment and appraisal. The assessment team mainly examines the manufacturer's data for clinical and cost effectiveness evidence. The assessors provide a draft advice document which is then submitted for appraisal. At this stage, unmet needs and patient perspectives are incorporated and a final guidance is issued.

The critical step in all this is the New Drug Committee (NDC) at which expert opinions are solicited. Key individuals are the economic assessor and the committee chair who sets the tone. Cost effectiveness is pivotal. The committee also comprises clinical assessors, internal experts and external experts. The external expert corps contains mainly pharmacists, with very few physicians, and the occasional pharmacologist.

External experts are typically NHS experts but are not necessarily KOLs. Tolley emphasised that few nurses are used and that they would like to see this group better represented.

External experts are selected by a series of 6 generic questions that address current guidelines, unmet needs, disease prevalence, and current treatment practice. The experts are important to the process, especially at the NDC stage. Tolley emphasised that decisions were not influenced by KOLs writing to the committee and that media ‘noise’ has a minimal effect beyond emphasising the need for media training of experts.