PoliticalSpeak: more on off-label prescribing

Is off-label prescribing the solution to the high-cost of MS DMTs in resource-poor settings? #MSBlog #PoliticalSpeak

“Did any of you read yesterday’s article by Sarah Boseley in The Guardian? If not you should. Almost all the points she makes about HIV, hepatitis C and cancer therapies are relevant to the management of multiple sclerosis (MS). The issues in the disease areas discussed in this piece dovetail to some extent with our off-label prescribing initiative and the Barts-MS essential off-label DMT list.”

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Sarah Boseley. Big Pharma’s worst nightmare. The Guardian, Tuesday 26 January 2016.

Excerpt: …..Fifteen years on, it is no longer just the poor who cannot afford the drugs they need. New medicines for lethal diseases such as hepatitis C and cancer have been launched on the global market at such high prices that the richest countries in the world are having to find ways to ration them…..

“I assume most of you have read my previous posts on this issue that provide some context; firstly my reflections on my sabbatical visit to South Africa and my latest New Year’s resolution post. Over the last few week’s I have been trying to get support for this initiative from several of the MS charities and seem to have hit a brick wall. In short, most of the charities are not keen to endorse off-label prescribing. I get the sense the charities have concerns about undermining the bureaucratic decisions of esteemed bodies such as the EMA, FDA and NICE who make judgement calls based on the benefits and risks of drugs, and in the case of NICE their cost-effectiveness. I see the Charity’s point in relation to the off-label promotion of drugs that have been rejected by these organisations in the past. However, most of the proposed DMTs on our essential drug list have never been submitted to the EMA and FDA for approval in MS. Some of my colleagues have argued that most of these charities are conflicted as they receive some support from Pharma. This is a possibility, but their primary responsibility has to be to their constituents, which are pwMS in rich-countries, and not to pwMS in resource-poor countries.”

“Another route to getting wide acceptance of off-label prescribing would be via the WHO essential drugs list. I am aware this was recently tired for azathioprine and failed. I suspect MS is not on the WHO radar; we need to change this. I have recently been asked to join a working group of the MSIF  that will address this issue.”

“Some of the charities are however prepared to work with the Pharmaceutical industry to get affordable drug access schemes implemented in countries with unmet need. The latter however, requires logistics, time and money and it may be difficult to implement across the globe simultaneously. In addition, drug access schemes are unlikely to be implemented in middle income countries where there are established and profitable private healthcare markets, but still a large unmet need for large sections of the population that are not insured (e.g. South Africa, Egypt, etc.).”

“It is great to see that majority of you who responded to our off-label prescribing survey support the practice. Thank you we find this reassuring and hopefully we can extend this to our colleagues, politicians and other stakeholders. If we don’t it is pwMS living with untreated active MS who will pay the price.”

“At Barts-MS we need your help and are appealing to people who are interested in helping with this initiative to contact us. Please remember that outside of developed countries there is a massive unmet need with regard to the diagnosis and management of MS; we can do something about it. If you are interested in helping can you please sign-up to our initiative. Thank you.”

CoI: multiple

20 thoughts on “PoliticalSpeak: more on off-label prescribing”

  1. Prof G don't you think that the low uptake of DMTs in rich countries is also linked to the high-cost of drugs? Surely this is a broader issue than simply a focus on developing countries?

    1. Re: "Prof G don't you think that the low uptake of DMTs in rich countries is also linked to the high-cost of drugs?"Yes, that is why we have organisations such as NICE to make sure the NHS only pays for cost-effective treatments. Despite NICE greenlighting DMTs in the UK we have a slow and low uptake; this is not due to price a complex mix of cultural and local economics. If these drugs were cheaper may be would be allowed to prescribe them more broadly. However, NICE guidance is usually with the licensed indications. I think your point is more relevant to healthcare systems other than the NHS.

    2. Re: "Surely this is a broader issue than simply a focus on developing countries?"Yes, it definitely broader than developing countries, which is why I refer to resource-poor settings. The latter is relevant to pwMS living in middle and high income countries without socialist healthcare systems; in these situations if you are not insured you simply can't afford expensive DMTs unless your are wealthy. I am aware that in some countries, e.g. the US, pharma run drug access schemes. These schemes however only cover the cost of the drug and not their administration or monitoring costs. The latter is hurdle for many pwMS accessing some of the DMTs that require infusions and blood monitoring; the latter costs money. If you have MS and are unemployed how can you pay? This is why I so grateful we have the NHS.

    3. I agree this problem is biting pwMS (and pw other diseases as described in Sarah Boseley's article) not only in resource-poor settings, but also in parts of the World that are in the position to afford high-cost healthcare: After all there's quite a number of not so affluent people living in affluent countries. In places like the UK, tax-funded healthcare has to walk the line between affordability for the taxpayer and keeping the industry & shareholders happy. Regulators charge incredible fees that can only be paid by Big Pharma, which in turn makes the regulators dependent on them. NICE converts efficacy into effectiveness which dictates the price the industry will be able to achieve. If the drug is not considered cost-effective, an undisclosed discount will be agreed that allows NICE to give it the go ahead, and patients access to the drug (excellent!). However, neither the original price nor the discount agreed in secrecy has much to do with the actual development or manufacturing, but very much with the maximum achievable within the confines of the system. NICE lives quite comfortably with this arrangement as they are just as dependent as the regulators on Big Pharma producing expensive drugs that create the need to assess cost-effectiveness. A drug like generic Cladribine that costs (in the UK) £165/10mg vial when you only need 6 vials/year, and perhaps for only 2 years in total, is too disruptive to this system and will be ignored by the NICE Health Technology Assessment (HTA) department until the day Movectro (oral Cladribine) comes along, enters the high-price market, and justifies their existence.

    4. The NHS is dying. The Tories are killing it. Thatcher never dared to privatise the Royal Mail, but she implemented a culture of privatising public assets during her reign that Cameron came along thirty years later and did the job for her.This is happening right now with the NHS. The system is being inextricably undermined so that within a decade or two it will be privatised.

    5. I sadly think you are right however I do not think you can lay all the blame at the Tories feet. As ProfG wrote there has been cross party support for these moves and the Labour laggards were gagged when when these aspects are being slyly pushed through. I don't here much uproar about the deal where the trusts get 39% if they under spend their drug budget but get lumbered with 30% cost if they over spend, so incentivising the trusts to ration drugs so they make money to offset other funding deficits and ensuring that the trusts are not going to want to fund expensive drugs. We simply to pay more ring fenced tax if we want an NHS.We wll see that certain centes become allowed to certain drugs and it will be all well and dandy if you live near one of these centres, but if you don't it makes me worried.

  2. So if you have a patient who is newly diagnosed with MS and the NHS wants to take a wait and see approach, what would happen to you as a physician if you offered an off-label drug?Maybe physicians can start a trend of dis-regarding the NHS recommendations of the wait and see aproach.

    1. The wait & see approach has very few friends at BartsMS, but I dont't think the NHS as a whole endorses this approach either. Delays arise for various reasons, sluggish translation of policies into action being one of them. Off-label drugs can be used whenever your doctor considers it to be the best option.

  3. I'm looking to get support from some charities on certain topics is quite difficult, and so I'm noticing is not only here in the UK and the US, but here in Brazil it's the same situation. I sent some emails to 02 large charities that support individuals with MS in Brazil, to build support for a discussion on the feasibility of treatment with Cladribine Generic Injectable for MS and so far got no answers … seems to be a "fear" on the issue of off-label treatments …

  4. There seems to be some misinterpretation about this initiative. Off-label prescribing is not a solution for drug discovery and development. All the drugs we have on our list were discovered and licensed for other indications. Without the necessary R&D that led to their licensing we wouldn't be able to repurpose them and use them to treat MS. Simply because they are 'cheap' we can use them to address and unmet need in parts of the world where pwMS can't afford expensive innovator drugs. This initiative is not for parts of the world where pwMS and their healthcare systems can afford expensive innovator drugs unless they fall outside of treatment guidelines and their is a compelling reason for these patients to on a DMT off-label.

    1. With the NHS culling drugs off the NHS England Cancer Drug Fund list it won't be long before their sights are trained in MS drugs. When will the NHS be classed as a healthcare system that can't afford new innovator drugs? Will people with MS in the UK ever have the opportunity to be treated with ocrelizumab? Will we be forced to receive rituximab off-label?

    2. Re: "When will the NHS be classed as a healthcare system that can't afford new innovator drugs?"I suspect if you had certain types of cancers that one of these culled drugs are licensed to treat and you need the drug you would already be in a healthcare system that can't afford innovator drugs. What this debate is highlighting is that there is a limit to what a tax-funded healthcare system can fund. The proposed solution is rationing and allowing patients to pay for these expensive drugs out of their own or their families pockets. Sadly this is what has destroyed the NHS; it is no longer equitable nor free at point of service. Those who can afford get to be treated with these drugs at great personal expense and those who are 'poor' or even 'relatively poor' can only watch from the bed next door.At the moment this has not happened in MS; what does happen is that access to treatment varies based on where you live and who you see. There is a great variability in prescribing habits between neurologists. Let's call this a type of postcode lottery.

    3. Re: "Will people with MS in the UK ever have the opportunity to be treated with ocrelizumab?"I suspect yes. Ocrelizumab still needs to be assessed and given a marketing authorisation (license) by the EMA and only then can it be assessed by NICE. This all takes about 2 years. I think there won't be a problem with its RRMS indication. The PPMS indication may get more scrutiny. Regarding NICE that will come down to a cost benefit analysis. In reality most Pharma companies have done a deal with the NHS to get a NICE greenlight.

    4. Re: "Will we be forced to receive rituximab off-label?"I suspect not; we have already tried to get NHS England to give us permission to use rituximab post-natalizumab in our patients who are JCV-seropositive. We are waiting to hear back from them, but the indications are that we won't be able to do so. It is also incorrect to assume that rituximab is a biosimilar of ocrelizumab or vice versa. They are different products. Rituximab is less humanised and hence there is a higher incidence of anti-drug antibodies and more infusion reactions. The drugs also are slightly different in their effector mechanisms, or the way they work. Based on these differences it is very unlikely that NHS England will treat them as interchangeable drugs.

  5. Is there a criteria you are using to recruit volunteers for this effort? For eg, are you looking for people who work in MS centers, or are you looking for people with MS in countries with less resources?

    1. Re: "Is there a criteria you are using to recruit volunteers for this effort?"No specific criteria; what we really need is local champions to make this happen and ideas to get people to adopt off-label prescribing. If you have active MS it is probably better being treated with an off-label drug than being left to the ravages of the natural history of MS.

  6. But when I say that I fully support the Off-label treatments, especially Cladribine for the treatment of MS is not because I think they "the solution to everything," but I see them as a cheap way out for countries that have not one public coverage system to pay for treatments licensed, or even those that have an NHS have problems in the distribution of medicines. Here in Brazil we are facing an economic and political crisis that has only increased. You know what you read every day in groups about MS in Brazil on Social Networks people constantly complaining that they can not take their medicines in public pharmacies of expensive drugs in Brazil because Brazilian states have not requested the provision or not paid suppliers for lack of funds, the money for this? People who are 2, 3, 6 months without being able to deal with Interferons, Fingolimode, etc., simply because the states have no way to pay them … If the situation in Brazil is already well, you imagine in Venezuela for example, facing an inflation rate of 200% per year, almost waging a civil war, which has neither toilet paper nor toothpaste for people to buy ?! That is why I support the off-label initiative, not because I think the the panacea for MS, to why those who have the means to pay for what they see fit and that is regulamentadoa but is a viable solution for poor or developing countries …

    1. Thanks for sharing your perception and experience. Only a fool will not understand where you're coming from.

  7. This is why some MSers are removing themselves from this "game" and are choosing to have HSCT. There is nothing to guarantee that NICE will not, at some date in the not-too-distant future, declare that natalizumab and alemtuzumab are no longer affordable. The idea that bean-counters could affect the level of my future disability is repugnant to me.

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