Health Technology Assessment (HTA) agencies have been widely promoting their interest in taking relevant patient data into account during the drug review process. The effort to make a place for the patient voice in reviews is promising in an economic climate where the cost of innovation is under constant scrutiny. However, it raises two critical questions:
- How much weight does the patient data have in the HTA decision process?
- Are submissions by patient and professional groups associated with positive decisions?
Patient and professional groups can make submissions to some HTA agencies in order to point out aspects of the drug that may have been overlooked in the manufacturer’s submission. These submissions usually focus on quality of life issues for patients and carers, unmet need, and discussion of the relative advantages of the new treatment compared to existing therapies.
To learn about the impact of patient and professional group submissions, we looked at all HTA assessments from NICE, SMC, CADTH’s Common Drug Review, pCODR, and PBAC in the Context Matters data model from 2005-present. This represented a total of 1,605 reviews. 52% of all reviews included patient or professional group submissions. 31% of all reviews were for oncology disease conditions and 15% of all reviews for rare disease conditions.
Reviews of oncology drugs and drugs for rare diseases are more likely to include patient and professional group submissions than non-rare and non-oncology reviews, suggesting that disease severity and end-of-life considerations may be major factors in these submissions. Other therapeutic areas with significant impact on quality of life, such as ophthalmology and sleep conditions, also receive a high proportion of patient and professional group submissions.
More and more reviews have been considering patient and professional group submissions over time. Patient and professional group submissions are associated with higher rates of positive decisions for non-oncology reviews than for oncology reviews, but considered together they do not appear to have a substantial impact on decisions. The chart below shows that the overall rates of positive and negative reimbursement decisions for reviews with and without patient and professional group submissions are almost equal.
Why is this the case? Why aren’t drugs that receive significant support from patient and professional groups more likely to receive positive reimbursement decisions? In our experience, economic considerations almost always take precedence over other considerations, even when patient and professional groups strongly advocate for a particular drug. The following case study illustrates this by highlighting a specific case in which an effective drug with strong patient and professional group support failed to secure a positive reimbursement decision for economic reasons.
In September 2013, the Scottish Medicines Consortium (SMC) issued a “do not recommend” for pertuzumab (Perjeta) for breast cancer, arguing that the economic analysis was not robust enough to justify the drug’s cost in relation to its health benefits, in spite of a patient interest group submission arguing that the drug should be reimbursed in Scotland.
SMC considered the drug again in October 2014. This review included comments from a Patient and Clinician Engagement (PACE) meeting. PACE meetings are intended to give patient and professional groups a greater chance to discuss “the benefits of a medicine, including how it can impact the quality of a patient’s life” as “this information may not always be fully captured within the conventional assessment process.” They are specifically used in cases where the SMC originally makes a “do not recommend” decision about orphan or ultra-orphan drugs (SMC considers pertuzumab an “orphan equivalent” because of the particular subpopulation it treats).
In the resubmission, the PACE group “expressed very strong support for pertuzumab, describing it as a major advance” and noted that “the normality of life observed in patients treated with this medicine has a wideranging positive impact.” The SMC acknowledged that the drug significantly improved progression-free survival and overall survival. However, in spite of the improved clinical outcomes and the strong endorsement for patient and professional groups, the SMC still issued a “do not recommend” decision on account of deficiencies in the economic analysis.
We have seen this in numerous other cases. Even with strong support from patient and professional groups and even with significant demonstrated improvements in clinical performance, drugs seldom receive positive reimbursement decisions without robust economic analyses demonstrating acceptable cost-effectiveness levels. It is too early to say whether patient and professional group submissions will have major impacts in some narrow circumstances; however, on a whole, it appears that manufacturers stand to gain far more from a robust economic case than from the support of patient and professional groups.