Review of Access to New Medicines

6.4 How the acceptance rates for end of life, orphan and ultra-orphan medicines have changed as a result of the new approach

6.4.1 The general perception of stakeholders is that acceptance rates, both with restrictions and without, have improved although there was a sense that this might not apply equitably to all three definitions, end-of-life, orphan and ultra-orphan.

6.4.2 It is not possible to draw definitive conclusions because of the short time scale of two years since the new approach was introduced and the relatively small number of end-of-life, orphan and ultra-orphan medicines considered in that period, namely fifty-seven. The drawing of conclusions is further hampered because the definitions in use have only applied since the inception of the new approach and therefore there are no reliable baseline figures against which to measure change. Outwith this Review consideration has been given by SMC to retrospectively applying the new definitions to enable comparison but this has been deemed not feasible. The only definition in use by SMC before the T&FG's work was the orphan medicine definition used by the EMA. The definitions in use for end-of-life and ultra-orphan are new and unique to SMC.

6.4.3 Some retrospective data is available. From 2002 - March 2014, SMC assessed 65 full submissions for EMA defined orphan medicines. Of these 16 (25%) were accepted for use, 25 (38%) were accepted with restrictions and 24 (37%) were not recommended for use. The combined acceptance rate for orphan medicines was 63%. (For the purposes of comparison medicines not recommended through non-submission are not included.)

6.4.4 The T&FG used submissions for designated orphan medicines and medicines within British National Formulary ( BNF) chapter 8 (Malignant Disease) as a proxy for previous acceptance rates for medicines used at the end of life or for very rare conditions, the assumption being that cancer medicines would fit the definition for end-of-life medicines. In the period November 2011 to October 2013, the combined acceptance rate for orphan/cancer medicines was 48%.

6.4.5 Published SMC data shows an acceptance rate of 75% for all submissions for end-of-life, orphan and ultra-orphan medicines considered under the new approach. This encompasses the total of 57 submissions however sub-analysis by definition is complicated by several medicines falling under more than one category ie orphan/end-of-life and ultra-orphan/end of life.

6.4.6 Taking account of the earlier comments about "true ultra-orphan medicines" analysis has been undertaken for this Review according to the indication for which the medicines were being assessed. Two broad categories were used, cancer ( BNF Chapter 8.1) or rare/very rare condition. The results of these analyses are shown in Tables 2 and 3.

6.4.7 Table 2 shows results for ultra-orphan medicines broken down by whether the medicine is solely an ultra-orphan or whether it meets dual definitions and also by broad therapeutic indication. Table 3 shows similar data for orphan and end-of-life medicines including those satisfying both definitions.

6.4.8 In summary the analyses appear to show a range of acceptance rates which in the main are higher than the figures from before the new approach used by the T&FG. There is however one significant outlier, ultra-orphan medicines indicated for very rare conditions, with an acceptance rate of only 14% representing one out of seven submissions. Arguably these are the "true ultra-orphan medicines." What all of these medicines appear to have in common, with the exception of the one medicine that was accepted (Pasireotide) is significant cost whether expressed as an Incremental Cost Effectiveness Ratio ( ICER) or Year 1 budget impact.

Table 2

Table 3

*All medicines designated end-of-life or combined orphan/end-of-life are indicated for cancers or other malignant conditions

Recommendations

12 Refine data collection systems to enable meaningful year-by-year comparisons and the monitoring of emergent trends.