In 2016, we released an article entitled “The future of ICER in the US: Are HTA assessments here to stay?” The Institute for Clinical and Economic Review (ICER) had been making headlines, after publishing Health Technology Assessments (HTAs) of then-newly launched drugs Repatha and Praluent. The PCSK9 inhibitors, indicated in cardiovascular disorders, were the first drugs to be assessed by the 2015 ICER program Emerging Therapy Assessment and Pricing (ETAP). These reports suggested that the current drug prices were too high and that the ex-factory price should be reduced by 17 – 85% to justify the broad label population according to the label. It was a landmark moment for the prominence of ICER in the US and posed questions about the future implications for US HTAs and drug prices in general.
The ICER Process
ICER’s HTA review process is similar to that of other countries, with an assessment undertaken by one of three appraisal committees over 30 weeks. An evaluation is performed after initial discussions with stakeholders, and following the release of a draft report, the appraisal committee meets publicly to make their final recommendations. Earlier this year, ICER updated their process to include some of the following procedures:
Changes to the ICER process are not the only developments since our original article was posted. The influence of ICER on payers has been steadily growing. Unlike HTA systems in other countries, such as NICE, ICER exists more as an informative resource for payers’ policy and strategy decisions, rather than a strict guideline on whether or not a drug should be reimbursed. However, this has not stopped some of the major payers in the US market from using ICER reports to make “one-size-fits-all” decisions. Most notably, CVS Caremark now provides clients (health plans and self-funded employer groups) the option to exclude drugs that do not meet the “high care value” benchmark of $100,000 per QALY. Nevertheless, ICER’s biggest impact has been as a bargaining chip on the conditions and utilization controls of reimbursement. A survey of major payers found that 35% used ICER reports to determine affordability and 13% directly used them in price negotiations.
Impact on Real-Life Examples
Remdesivier
The impact of ICER has been playing out in the public sphere, with the recent assessment of Gilead’s remdesivier, which is currently in the spotlight as an antiviral medicine for patients with COVID-19. ICER’s analysis, when evaluating remdesivier on top of dexamethasone as standard of care, gave a cost-effectiveness threshold of $2,520 to $2,800 per patient. Interestingly, Gilead launched the product at $2,340 per patient, slightly below the ICER threshold, perhaps reflecting their awareness of public sensitivity regarding COVID. This decision may also have been driven by the success of cheap and readily available dexamethasone, with its expected contribution to reducing mortality influencing ICER’s threshold, which was initially higher. So far, it seems remdesivier has successfully broken into the COVID market, although this may not be surprising with the need for treatments being so front-and-centre in today’s news cycle. And despite the lower-than-expected price, Gilead says it expects total sales of remdesivier to reach $1 to $3 billion for 2020 alone.
PCSK9 Inhibitors
But what about last time ICER was making headlines? In 2016, ICER had found that to be cost effective, Praluent and Repatha would need a price of just $2,177 per year, as opposed to the ~ $14,000 per year factory price the manufacturers were looking for. On-going negotiations have continued since then. Despite both drugs addressing a fairly large unmet need in familial hypercholesterolemia (an inherited disease causing high levels of LDL, or so-called “bad cholesterol”), and reducing the rate of heart attacks and strokes in some patients more effectively than statins, a final ICER report in 2019 found the drugs were still too expensive to be cost-effective. And the market agrees. Both drugs struggled to break into the market and are considered commercial disappointments. In December 2019, Sanofi, the manufacturer of Praluent, withdrew from the US cardiovascular market in favour of immunology, and released marketing rights of Praluent to its development partner Regeneron. It is difficult to ascertain the extent to which ICER’s reports influenced the failure of Praluent and Repatha in succeeding in the US, but there is no doubt that one reflects the other.
Spinraza
It is worth noting that this is not always the case. In April 2019, ICER released a statement that expensive rare disease drugs like Spinraza are “threatening the affordability and sustainability of the US health system”. Biogen’s Spinraza is an anti-sense oligonucleotide – a molecule which targets RNA to regulate gene expression – and is indicated as a therapy for spinal muscular atrophy. It was launched with a list price of $750,000 for the first year, followed by $375,000 per year, which works out to $125,000 per injection. At such a price, it is perhaps no wonder that it gained some negative public attention, or that ICER recommended an 83% price drop ($72,000-$130,000 per year) for it to be considered cost-effective. Despite this, Spinraza had, in Biogen CEO Michael Vounatsos’ own words, “one of the most successful rare disease launches of all time”. In Q4 of 2019, Sprinraza had revenues of $243 million in the US, with reimbursement for 80% of commercially insured patients and two thirds of those under Medicaid. In this case, the opinion of ICER that Spinraza is not cost-effective does not seem to have negatively impacted its market access in the US.
What Next for ICER in the US?
The pharmaceutical landscape is evolving, and gene therapy products are entering the market in increasing numbers. However, with large costs and high risks regarding both efficacy and safety, the role of ICER assessments could be a key factor in the outcomes of these cases.
As of now, it is clear that ICER analyses are of growing interest to payers, particularly those regarding new products with large price tags. Reports are being used by payers to both determine affordability and negotiate a more palatable reimbursement price. However, while this may be saving payers money, there is little evidence that ICER has had any influence on patient out-of-pocket costs. With drug prices being a contentious issue in the US political landscape, and a US election just around the corner, the impact of ICER here may be one to watch.