This week NICE issued guidelines surrounding the development of digital health standards that ensure new technologies are (1) clinically effective and (2) offer economic value. Both parts have a proportional approach to defining evidence standards which recognizes: (a) the sparsity of available evidence in the field of digital healthcare, (b) the challenges of developing traditional clinical trials for digital health technologies, and (c) the significant opportunities offered by digital health technologies to collect real-world data to inform effectiveness judgements. This is probably coming at the right time as a recent survey by Roche UK found that “82% of those in the 16-24-year-old age bracket want to see more use of digital technology on the NHS.” Great, maybe we can get past the point where conference calls (BTW, link is highly entertaining) are termed as “technology.”
Earlier this week the Scottish Medicines Consortium (SMC) rejected NHS funding for four new therapies, including Bristol-Myers Squibb and Roche’s cancer medicines Opdivo and Gazyvaro. If you don’t know, those are pretty popular drugs. Opdivo recorded $1.2B in worldwide sales in the 2nd quarter of 2017 (that’s ~$13.3M per day). The reason for the rejections? Lack of evidence that the price justified the effectiveness (vs. current treatments). The SMC’s decisions mirror those made by NICE, which rejected their use in England and Wales. Hard to argue with the logic, but we just can’t help but ask; If these medicines were made by Scottish drug makers, would they be approved? Because we all know, if it’s not Scottish, it’s crap. Either way, drug makers better bring their cost-effectiveness A-game if they want future approvals.
Earlier this week the National Institute for Health and Care Excellence (NICE) said it is not recommending NHS funding for Eisai’s Kisplyx (lenvatinib) plus everolimus for treating advanced renal cell carcinoma in adults who have had one previous vascular endothelial growth factor (VEGF)-targeted therapy. The reason? “The cost-effectiveness estimates compared with all comparators were much more than what NICE normally considers acceptable (£30,000 per quality-adjusted life year gained).” Gotta hand it to NICE, a rule is a rule. The decision did recognize there was a 10.1 month improvement in life expectancy, but this figure was based on a small sample size in the clinical trials. While we’re not market access experts, good chance Eisai will be firing up some RWE/HEOR/Late-phase studies fairly soon.
Beginning in April of this year, NHS England will have the ability to withhold patient access to select approved medicines, according to The Times. Even if NICE considers a new drug of appropriate value, it may be withheld or rationed if it costs the NHS more than £20 million per year. While this might not be too alarming for those seeking help to lower cholesterol, the prospect of a drug being withheld for a rare disease – even if it is considered good value – is no bueno. Or what if one of the many potential Alzheimer’s drugs in development works? The size of the addressable patient population could make the £20 million threshold look puny. Stay tuned for more unpopular decisions to come.
Want to shave 60 days off your drug’s approval by NICE in the UK? No problem, just ensure your product (drug, device, treatment) adds one year of quality life for a patient for less than £10,000. Done. Last Thursday, NICE announced it is seeking comments on a proposal to cut the approval time from 90 days to 30 days, stating products that “have a likely cost per QALY (quality adjusted life year) of up to £10,000 would be dealt with more quickly under a ‘lighter touch’ process.” There is also a provision for treatments that address rare diseases (aka – the Highly Specialised Technologies programme). PS, you have until January 13, 2017 to respond to NICE with your comments. Pencils up.
It turns out the relationship between regulatory approval and drug pricing isn’t a simple one. On the one hand, regulators and payers are pressured to discourage the development of me-too products because being rewarded for not adding value feels… well… wrong-headed and simply adds to the number of new products, which typically command higher prices. On the other hand, some are arguing that these product cost – drug value decisions are actually driving prices up because denying approval of low-value-add products leaves existing products with little competition. And we all know what limited competition breeds. Just ask. Gilead.They’re not a cheap date. Only time will tell how the gatekeepers’ strategies will evolve but the law of unintended consequences will not be ignored!