PoliticalSpeak: High-cost drugs NICE or not so NICE

How long can we continue to ignore the impact high-cost drugs are having on the treatment of MS? #PoliticalSpeak #MSBlog

“I note my post on the Norwegian health technology appraisal on the cost-effectiveness of DMTs in Norway generated a relative muted response. I am surprised considering how hot the topic of high-cost drugs is at the moment, particularly in the USA. An online BMJ article by Narcyz Ghinea and Wendy Lipworth discuss the issue and cover many of the points we have highlighted many times on the blog in the past.”

“What surprises me, and upsets me, is how passive the MS community seems to be about the issue of high-cost drugs when you compare it to communities in other disease areas. Why? To me the cost of MS DMTs is one of the main reason why we are not managing MS actively, or aggressively, enough. The price of DMTs forces healthcare systems to rank DMTs as first, second and third-line and to impose a whole raft of restrictions to prescribing. More importantly the price of these drugs means that the majority of pwMS in resource poor environments have no access to treatment. In my opinion this is a scandal. High-cost drugs are responsible for a new form of therapeutic nihilism; dare I call it nihilism?”

“I am aware that many of you disagree with me on this issue, but seeing patients with highly-active MS on no DMTs, in South Africa and India, when I was on my sabbatical still haunts me. They weren’t on DMTs because their respective healthcare systems couldn’t afford the price of licensed DMTs and they couldn’t afford to pay for it privately. In response to this dilemma I generated our Barts-MS Essential Off-label DMT List that has drawn criticism, some of it very vocal, from many quarters. To be honest I don’t care; at the end of the day I am a clinician and if faced with the option of not-to-treat, or to-treat with an off-label drug, I will always put my patient’s interest first and offer them an off-label drug. I know enough about MS that if it was left to its own devices, and given sufficient time, it will cause the majority of people with the disease to become disabled. Since promoting off-label DMTs in resource poor settings it has become clear that these drugs need to be prescribed in a safe and appropriate way. This has highlighted the need for education of neurologists in these environments and for improving MS services in these environments so that the drugs can be prescribed safely. To do this we are starting an initiative to raise awareness of the issues faced by patients and clinicians in resource poor environments. What I have learnt from our ‘Brain Health: time matters’ policy initiative, is that simply standing on a soap box and shouting makes little difference and simply adds to the noise; in short nobody listens. This is why we need discussion forums, lobby groups and policy initiatives to address the issue of high-cost DMTs; if we don’t we would be letting these people down.” 

“I am pleased to see that the high-cost drug debate, and what is cost-effective, is still very active and ongoing. Have you ever considered why NICE, health economists and payers set the cost per QALY (quality adjusted life year) at a particular level and then base their cost-effectiveness, or rationing, decisions on this level? Last week’s BMJ highlights this issue and gives the rationale for why NICE has set the cost per QALY between £20,000 and £30,000 (see Timmins’ & Appleby’s articles below). In relation to DMTs for people with MS we think the threshold has been set slightly higher at ~£36,000. Why? This level was set more than a decade ago when they assessed the cost-effectiveness of interferon-beta and glatiramer acetate. The threshold was set to cover direct medical costs and does not include societal, or indirect costs, and the threshold is not index linked. So overtime DMTs are getting relatively cheaper and hence more cost-effective. Please note this statement refers to the NHS and does not apply to other countries, for example, in the USA the real cost of MS DMTs have soared well above the price of inflation, including drug-price inflation.”

Article 1: Narcyz Ghinea & Wendy Lipworth. Propaganda or the cost of innovation? Challenging the high price of new drugs. BMJ 2016;352:i1284

Key messages: 

  • The cost of drugs, particularly new biological agents, is overwhelming health budgets around the world
  • Little is known about how much it really costs to develop new medicines and, therefore, what they are really worth
  • This limits the ability of public and private payers to negotiate affordable prices and show that they are achieving “value for money”
  • Basic information about drug development costs needs to be available to both payers and the public to ensure greater accountability

Article 2: Nicholas Timmins. At last, NICE to take over the Cancer Drugs Fund. BMJ 2016;352:i1324.


….. The National Institute for Health and Care Excellence (NICE) celebrates its 17th birthday in April. By any standards it must be judged to have been one of the more successful pieces of public policy in the past couple of decades…..

…… It has shielded politicians from many of the really difficult decisions about what the NHS should and should not provide. It has made cost effectiveness, as opposed to purely clinical effectiveness, a key part of that. And—amid repeated controversies—it has sought with considerable success to balance an essentially unequal equation. One between the interests of the taxpayer in cost effectiveness; the interests of individual patients, who when they are not paying simply want the treatment whatever it costs; the interests of clinicians who, by and large, welcome guidance but detest instruction; and the interests of the life sciences industry, which needs a market for its products and ideally wants one that encourages the best sort of innovation while delivering the best possible price. Navigating that equation—seeking to balance it—has never been easy and has often been controversial……

……. However, by applying a set of social values to its judgments and seeking to operate to a set of principles—that it will be robust, inclusive, transparent, independent and contestable—it has done a pretty good job. One that other countries have sought to emulate…… 

Article 3: John Appleby. Crossing the line: NICE’s value for money threshold. BMJ 2016;352:i1336.


….. For a publicly funded health service, deciding what services and treatments should be available to patients is not just a matter of getting the best value for every (scarce) pound spent but an ethical duty too…. 

….. Choosing “what’s in and what’s out” quite literally involves decisions about life and death; the cost of getting a decision wrong can be measured not just in wasted resources but in avoidable deaths and lower quality of life. In this respect, the creation of the National Institute for Health and Clinical Excellence (NICE) 17 years ago was a watershed moment. For the first time there was an organisation required to advise the NHS explicitly on what it should offer to patients, drawing on evidence of the clinical benefits and the cost of obtaining those benefits……

…… For some obvious practical reasons, this is not how NICE arrived at its threshold range of £20 000 to £30 000. (It chose a range rather than a single number to allow some flexibility in decision making to reflect uncertainties in cost and effectiveness data and the use of criteria other than cost effectiveness in making decisions….

…… The exact origin of this range remains somewhat shady, emerging as a reasonable benchmark from NICE’s early evaluations of various healthcare interventions. Compared with the World Health Organization’s suggested cost effectiveness range based on gross domestic product (GDP) per capita of a country (“very cost effective”) to three times GDP per capita (“cost effective”), NICE’s threshold is much less generous (fig 2). On the other hand, recent analysis by Claxton and colleagues suggests NICE’s threshold is too generous relative to the cost effectiveness of treatments and services currently provided by the NHS….

…. Whatever the starting point for NICE’s threshold, because the NHS budget has roughly doubled in real terms since NICE started using its threshold range and the NHS has also increased outputs relative to inputs (that is, increased its productivity) over time, the threshold should have more than doubled over the past 15 years—becoming more generous to less cost effective treatments (fig 3). To the possible chagrin of drug manufacturers, this hasn’t happened—not least because moving the threshold goal posts would create problems. Previously rejected treatments could become cost effective as the threshold moves; what does NICE do then, and what incentives does this create for drug manufacturers? But we shouldn’t get too fixated by the cliff edge nature of thresholds. As Helen Dakin and colleagues have shown, while cost effectiveness explains most of the decisions made by NICE, other criteria play a part, and there is little indication of a sharp accept-reject divide at the supposed fixed threshold of £20 000 to £30 000 per QALY……

12 thoughts on “PoliticalSpeak: High-cost drugs NICE or not so NICE”

  1. As always so much to think about. My limited comments: I live in the USA and fully understand the costs of DMT and my ability to be allowed to take one or another based on random criteria. I am currently on Tysabri but am JC+. I now know that if I ever take a drug holiday, I will not be allowed to go back on. I am deemed to high a risk for PML by the insurance company.The second issue is that as health care has changed in our country (I leave commentary on that for another day) our out of pocket costs have risen dramatically. So all of the DMTs whether oral or infusion cost the patient now. For those limited in their ability to work, this also determines their decisions.Lastly is physician risk in the USA. So many doctors who do not specialize in MS are hesitant to prescribe the DMTs for fear of the side effects coming back to haunt them. The DMTs are complicated albeit effective for pwMS and take time and effort to prescribe confidently. We also need honest and well understood answers from our Neuros to help evaluate the risk of each DMT.And lastly, that leaves maybe why we have been quiet. IF we are happy and steady why rock the boat?!? Furthermore as has been discussed many times: if there is no profit motive for big Pharma it is hard to get their attention to look at low cost DMTsThank you for all of your insight.

  2. It would seem that the MS community isn't motivated to make much of a noise in this area! Most of the Ms charities have a campaigning section but maybe what's needed is a group of people solely dedicated to campaigning/lobbying? maybe people are so busy putting their limited energy into everyday living that they don't have the time or energy to take on NICE as well? I for one appreciate your efforts at raising this issue. I would like to see the 'cost effectiveness' question properly aired and a sensible conclusion arrived at, to include symptom relief drugs – I am particularly thinking of Fampridine which can help someone stay on employment with all the benefits that brings to the exchequer amongst others.

  3. As an MSer, I am profoundly grateful for your efforts. I am just too exhausted sometimes to add a comment; much less, go to battle over this. But, I thank you.

    1. Re: "I am just too exhausted sometimes to add a comment; much less, go to battle over this."This may explain it; people with MS are too tired to do anything that takes more energy than what they have got to give. The solution we need to help you do it!

    2. The rifts globally in the MS community of patients and entities have such a broad and wide impact its literally startling. The results are a near complete mistrust in a large segment of patient / caregiver populations having them feeling like leaves in a tornadic vortex. They will rapidly point fingers but when it comes to the work few engage it. I do not just speak of things such as activism but even the mechanisms towards personal wellness regiments.In a recent #chat for example with several entities including the National MS Society the MS Society quite correctly stated that everyone's MS is different. It was stated that as such best outcomes tend to also require a personal and refined regiment that is individualized. That care professional that are multiple disciplines are the best path towards current best outcomes. It was a well promoted discussion and literally, myself and a few other MS'ers even appeared for it.This stuff is hard work.Engaging costs of DMT's requires the leadership to activate patients and caregivers. Yet, even getting them to engage what really can have a great impact on the pathology of their MS is not accomplished in broad spectrum.Recently I have been telling people that I do not prescribe to "Spoon theory" as a person with MS. I tell them I prescribe to "Fork theory" meaning I continue to stab away at what I can do personally towards my best outcome given the current state of MS and practical knowledge.In the same frame of mind I was in a #chat with a whole slew of entities related to The Heart Association (USA). The conversation was the well known I/O. Eat / Exercise towards health. I put forward the question, "Why are these high profile med entities hammering away at what is well known amongst healthy populous .vs. expending energies towards those with other conditions that could see great benefits? NOT A SINGLE response. Not a one. It quite literally appeared as a "If you are enduring disability you cease to exist"

  4. I resoundingly agree with the points being made by the writer of this post.My personal trajectory amply emphasises this – 10 year RRMS, 10 year SPMS -currently effectively medicating with off-label slow-release 4 amino pyridine, AKA fampridine. My personal savings are of the order of A$3,000 per year for this medication that the Australian system does not yet fund publicly.Moreover, in response to the comment above, about the reaction of the MS Community my approach is actively not promulgated by the Australian MS Society.

    1. But David Australia is not a low-income country. I think Prof G is referring to people living with MS in poor countries.

    2. Re Fampridine being "off-label" in Aus – as far as I am aware it is not "off-label" at all – it is approved for use but just not subsidised by our Pharmaceutical Benefits Scheme. Mine cost me $A215 for each 28 days supply – I did a seven week trial to see if it would work for me but I was one of the 65% of non-responders. Good for my budget but not so good for my walking!The IFN Beta-1A I was on cost the Aussie taxpayer a bit over $A1,000 for each 28 days supply. That did me no good either – just made all of my symptoms worse so I gave up on that as well.Currently only taking LDN – cost at the moment is around $A120 per 100 capsules.No idea what the next thing might be – depends on the next MRI.MS Societies around the world seem to really only tread the middle ground and are more focussed on services than drug costs – to lobby about costs would probably threaten their fundraising efforts – "too political"!. They don't really speak for me either – my MS nurses were somewhat prickly because I asked questions, did my own research, and didn't put them on pedestals. Still, they are good for some people.However, Prof G makes a very important point – most of us in the so-called developed" world do have options. Many people in other countries don't. At least Prof G cares about outcomes for patients – there are too many neuros and other specialists around who don't and who are perhaps overly influenced by cost as a primary factor. I'm just glad that I don't have MS and live in India or one of the other resource poor countries – at least with living where I do I have options.

    3. My "off label" is compounded, slow release 4 amino pyridine and costs $50 per month so all my comments registered to that

  5. Convincing pharma to lower drug costs is not within my reach. I try not to stress too much about it, instead look at the positive. High cost of MS drugs makes it a very lucrative market. I was diagnosed in 2012. Since then Aubagio, Tecfidera, Lemtrada, and Plegridy have been approved, ocrelizumab is on its way. There are more trials being done for repair and restore. I am sure that soaring costs is one of the reasons pharmaceutical companies are investing so much in MS.

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