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We continuously monitor the assessments published by Health Technology Assessment (HTA) organizations around the world to identify the trends in payer decision making shaping our industry. The most recent trends aren’t likely to shock anyone: HTA authorities want a better demonstration of value, and they want it now.

Payers want more proof of value

While the number of assessments published by health technology assessment organizations in 2015 remained steady, the message remains clear: these organizations want robust effectiveness and cost-effectiveness data and more definitive proof that a product provides added-value over existing care. When developers can’t provide the outcomes data to validate these claims, they are facing lower prices and more restricted access than they would like.

Our research shows that agencies across the United Kingdom, including the All Wales Medicines Strategy Group (AWMSG), the Scottish Medicines Consortium (SMC) and the National Institute for Health and Care Excellence (NICE), are publishing an increasing number of restricted positive recommendations, implying that limiting submissions to certain subgroups or lines of treatment may help to demonstrate cost-effectiveness and secure reimbursement approval within the United Kingdom.

The UK is also replacing its Cancer Drugs Fund with a new managed-access fund controlled by NICE, which is a blow for many oncology drug developers. The CDF was set up in 2011 as an interim measure to ensure patient access to high-cost oncology treatments that NICE had rejected. Beginning in July responsibility for administering the fund will be handed to NICE, which is revising its process so that products with insufficient evidence for approval can be made available. It is meant to be a cost saving measures that will make it easier to collect longer-term data on the benefits of drugs, according to Sir Andrew Dillon, chief executive of NICE, but critics say it will eliminate drugs currently used by more than 12,000 patients per year. 

In France developers are also facing a tougher time, with HAS being more conservative in their judgements of the added benefit new products provide. The data in our HTA accelerator showed the French National Authority for Health (HAS) issued no ASMR I rating in 2015 and only 2 ASMR II ratings, a decrease from the 9 attributed in 2014. It should also be noted the percentage of ASMR III ratings was notably lower.

In Germany, G-BA adopted a positive opinion, including restrictions, on 59% of appraisals (vs 51% in 2014). 82% of positive recommendations without restrictions were in the categories of minor or non-quantifiable benefit. On the flip side, ~69% of appraisals with a considerable or major benefit included some type of restriction mostly with respect to the patient population.  Hence, higher added benefit can mostly be achieved in specific patient populations with high unmet medical needs. G-BA wants to see more head-to-head comparisons in indications with a sizable number of high-cost medications already available. One example is Rheumatoid Arthritis.

The message is clear: developers who want to market their products in these countries have to improve the quality of real world outcomes data they include their submissions if they hope to secure desirable ratings going forward.

Interestingly, the US was the only country to see a substantial uptick in assessments. The number of published reports more than tripled between 2014 and 2015, reflecting an increasing focus on drug pricing in the US that is evident in both the press and the presidential campaign. The Institute for Clinical and Economic Review (ICER) was particularly noteworthy in 2015, rolling out its Emerging Therapy Assessment and Pricing program. The program analyzes the comparative effectiveness, cost-effectiveness, and potential budget impact of new medicines and calculates a benchmark price for each new drug based on ICER’s view of the real benefits the drug brings to patients.  It remains to be seen what impact these reports will have on drug pricing in the near future, but it is further proof that the US is become more price wary than it has been in the past.

Finally, Japan is reforming drug pricing to incorporate cost-effectiveness analysis in its assessment of new products. As part of its national medical fee revision for 2016, The Central Social Insurance Medical Council (Chuikyo) will require products to undergo cost-effectiveness analysis  in order to set reimbursement rates.  While details are still being worked out, it is a bold move tied to the country’s growing interest in controlling healthcare costs.

EAMS hits the market

There was one other exciting bit of news to emerge from our most recent research. In March of 2015, Keytruda®, a cancer immunotherapy drug from Merck, was the first treatment to be accepted under the UK’s new Early Access to Medicines Scheme (EAMS). Seven months later, the NICE published a draft guidance recommending Keytruda as a first line treatment for adults with advanced melanoma.

It’s an exciting move for patients with advance melanomas, and an indicator that all of the talk about government agencies giving patients earlier access to treatments for life threatening diseases is driving real results. EAMS was launched in the UK in 2014 to promote innovation by giving these patients access to medicines that do not yet have a marketing authorization when there is a clear unmet medical need. The NICE decision in the case of Keytruda was based on preliminary data demonstrating that end of life and cost-effectiveness criteria had been met, and will help win the support of payers in the final step of this process.

While it is still early, and there are critics of EAMS who believe putting products in the market based on preliminary data is not a good decision, I’m optimistic that providing companies invest in real-world evidence generation Keytruda will be the first of many drugs for life threatening illnesses to move through this or similar schemes. 

Topics in this blog post: Biopharma, EMA, EU, Evidence, HTA, Market Access, Value