A survey conducted by the Association of the British Pharmaceutical Industry (ABPI) looks at the main reasons for the National Institute for Health and Care Excellence (NICE) issuing optimised recommendations and the reasons for terminated appraisals.
According to the ABPI, over the last 20 years, 82% of technology appraisal recommendations have been positive (ie recommended or optimised); around one third of these are optimised recommendations though in the past five years this has risen to around 43% of positive recommendations.
To better understand the reasons for optimised recommendations, the ABPI conducted a survey of member companies in March 2021 requesting details of appraisals with an optimised recommendation over the period 2016-2021 and on terminated appraisals to gather information on the main reasons for non-submission of evidence by companies. The survey looked at 48 technology appraisals (around 45% of all optimised recommendations issued over the last five years).
According to the results of the survey, the most common reason for optimised recommendations (33% of cases) was that the company did not submit clinical/cost effectiveness evidence for the full licensed population. The next most common reason was cost-effectiveness (20% ie drugs that NICE determined to be clinically superior in the full licensed population but not cost-effective in the full-licensed population), clinical effectiveness (17% ie drugs that NICE judged to have a lack of evidence of clinical effectiveness in the full licensed population) and stratification of the patient population to reflect clinical practice (11%). Notably, whilst company submission was the most frequently cited reason for optimised recommendation, ABPI members commented that in practice this represents the companies’ expectation of a positive NICE recommendation in only a subset of patients, usually on the grounds of cost-effectiveness.
The survey also looked at the reasons for 30 NICE technology appraisals that were terminated because of non-submission of evidence. In 34% of cases, the main reason was a lack of flexibility to pursue differential net pricing or complex schemes with consequent risk to the revenue from previously approved indications. The second most common reason (24%) was the inability to present a cost-effective case due to pricing of other medicines in the combination. Lack of clinical demand (no positioning of the product in English clinical pathways) accounted for 14% of cases. Inability to provide a cost-effective net price and too much clinical uncertainty due to immature data each accounted for 3%.
The ABPI goes on to note that medicines indicated for solid tumours represented the largest group among optimised recommendations (46%) whereas, among terminated appraisals, medicines for haematological cancers represented the largest group (53%).
- www.pharmaphorum.com, “Why companies don’t submit to NICE”, 22nd April 2022
- www.abpi.org, “Member survey: Reasons for NICE optimised recommendations and terminated appraisals”.