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AMA

I'm a Health Economist (I submit new medicines to NICE for use on the NHS) AMA

45 replies

QALY · 14/11/2018 00:01

I work in the pharmaceutical industry and quantify the long term medical outcomes and economic case to NICE and other Health Technology Assessment bodies in Scotland (SMC) and Wales (AWMSG) with the aim of getting a positive recommendation and reimbursement for the new medicine for use within the NHS. I have worked on a number of cancer drugs as well as drugs for other non-cancer medical conditions.

Ask me anything and i'll do my best to answer your questions.

OP posts:
MotherForkinShirtBalls · 14/11/2018 00:10

Do you do assessments for rare disease drugs?

ShovingLeopard · 14/11/2018 00:11

Do you believe in the value of what you do?

How do you feel about people who die because a drug isn't funded? Does it affect your feelings for the job?

PerkingFaintly · 14/11/2018 00:12

Wow, interesting area.

Want to think of questions.

OK, I get that you work on the pharmaceutical industry side of the fence, so obviously are answering from that perspective.

How does the role of NICE in the UK compare to what's done in other countries? Do most countries do some sort of formal cost-benefit or QALY calculation, for use by healthcare providers?

catlovingdoctor · 14/11/2018 00:16

What is your academic background/history?

QALY · 14/11/2018 07:55

ShirtBalls yes, I've been involved in assessing rare diseases. I find this particularly rewarding as there is a real need for new treatments for these patients.

catlovingdoctor I am a pharmacist and worked for many years in this role before moving into the pharmaceutical industry. I also have a Masters in health economics. Health economics is still a relatively new discipline so you tend to get people with very diverse academic/employment backgrounds, the vast majority will have a Masters in health economics or a similar subject. There are a lot of health economists with pharmacy or other scientific/healthcare degrees (biochemistry, biomedical science, public health, epidemiology, etc) as well as mathematics/economics graduates and others with academic backgrounds.

OP posts:
QALY · 14/11/2018 08:47

Leopard great questions!

Do you believe in the value of what you do?
Yes, I do. Healthcare systems don't have bottomless pits of money and have to make rational decisions about which new medicines offer value for money. The system for assessing new medicines in the UK is very transparent (e.g. you can access all of the documents on the NICE website) so by providing the health outcomes and cost data to the assessment body (NICE, SMC, AWMSG, etc) the committee of experts can make a decision in the interest of patients and the public/taxpayer as a whole. The process is very robust and our submissions are thoroughly critiqued and challenged.

How do you feel about people who die because a drug isn't funded? Does it affect your feelings for the job?
I have only worked on cancer drugs for advanced cancers where the disease has already spread and the new medicine offers a palliative treatment. Typically, patients with this advanced stage of disease would have, on average, less than a year or two of expected life with the currently available treatments. This means that whilst the new drug aims to improve symptoms and quality of life it sadly doesn't offer a potential cure. I have worked on some new cancer drugs that on average can extend life by a year or more (not all patients will respond to the new drug so some patients may die much sooner and others respond well and may live much longer) and other drugs where the clinical trial data has shown survival benefits of a few weeks or months.

This means that the vast majority of patients will sadly die eventually, with or without the new medicine. However, having the option of a new medicine that can extend life for a few weeks, months or a year or more offers hope to a lot of patients and their families and can help them live longer to see a child/grandchild born or get married. I have worked on some new drugs that are the only new treatment option in recent years and we were able to get these funded. In the situations where other drugs were considered to be of lesser value and not funded, there were other new drugs also being assessed, so whilst the drugs I worked on did not get funded, there were other treatment options available for these patients.

It is a huge responsibility to be leading the submissions and I'm motivated to get a successful outcome for patients and their families, so that they have another treatment option available to them. It is very frustrating and disappointing to get a negative decision when you've worked for so long on a submission and you really believe in the value the new drug brings to patients and to the NHS. The negative decision could be due to not having robust enough data to convince the committee about the benefits or cost impact of the new drug, or it could simply be that I have had my hands tied from the outset with a high price that I struggle to justify when we present the case for the benefits and costs. This really frustrates me and frankly pisses me off and that's when I question whether I'm in the right job or the right company, but I'll have been fighting internally for months if not years to get a realistic UK price.

OP posts:
KanyesVest · 14/11/2018 09:02

Why is pricing different in different countries?

rogueantimatter · 14/11/2018 09:06

Why do you think governments and international bodies don't fund more large scale pharma reseach? The current system appears so exploitative. I get that there isn't a finite budget for healthcare but it seems unethical to deny people in great need something that will help them unless they give the pharma seller a huge amount of something they want, but don't actually need.

It's like this: my only cow dies. You have three (probably through good luck at least as much as merit). I ask for one of your cows. You (pharmaceutical company) refuse to give me one unless I give you something in return, something I may not have. Consequently my family has no milk and suffers accordingly.

Do you feel that big pharmas epitomise the problems with capitalism?

QALY · 14/11/2018 09:11

Faintly

OK, I get that you work on the pharmaceutical industry side of the fence, so obviously are answering from that perspective.

Naturally, my main perspective is from the pharma side, however, having worked as a pharmacist I can also see it from NHS side too, and here's my disclaimer though of course all views are my own and not necessarily representative of the pharmaceutical industry or the NHS.

How does the role of NICE in the UK compare to what's done in other countries? Do most countries do some sort of formal cost-benefit or QALY calculation, for use by healthcare providers?

NICE makes decisions for the NHS in England and Wales. SMC assesses new medicines for use in Scotland and AWMSG assesses new medicines for Wales where NICE is not reviewing it. They all have similar processes and requirements for assessing the cost-effectiveness of new medicines with cost per QALY as the default requirement.

Many other countries use formal cost-effectiveness assessments including Australia, Sweden, Canada, France, Germany, Netherlands, Ireland, etc although they all have their own nuances in terms of their processes and assessment methods. However, NICE is extremely robust and transparent so influences decision making in many other countries that don't have their own formal assessment body.

OP posts:
KatyMac · 14/11/2018 09:12

How do you feel about the areas where little or new new research is being done (small cell lung cancer - my dad was told the treatment hadn't changed in 30 years or B12/Vit D where more work needs doing)

Or when you hear that someone prints out the NICE guidelines and takes them into the GP but the GP ignores them

QALY · 14/11/2018 09:16

I have to work now so I will come back later tonight and answer more questions.

OP posts:
PerkingFaintly · 14/11/2018 11:42

Thank you very much for answering – and for partly seeing it from the NHS side.

That's very interesting that the NICE process is so robust and transparent, that it actually influences what happens in other countries.

I think you've already answered most of my next question, but feel free to expand more!

My question had been going to be, whether you feel the NICE process delivers a reasonable balance of value-for-money vs availability of drugs? And whether the UK is fairly well-served by it?

QALY · 14/11/2018 22:11

vest

Drug pricing is very complex and I'm not an expert in international pricing by any means. However, in the simplest terms so that I understand it many countries have free pricing so companies will set a price in individual countries at a level the market will accept. It's no so different to any other product, be it a book or a car, being priced differently in UK and other countries.

Some countries reference UK, and other country, prices so this can drive prices higher in the countries referenced due to the impact downstream.

Many other countries reference UK prices, but at the same time we have NICE, etc which means medicines still need to be considered cost-effective and a good use of NHS resources to get NHS funding.

OP posts:
QALY · 14/11/2018 22:30

rogue

Why do you think governments and international bodies don't fund more large scale pharma reseach?

Again, not my area of expertise but I guess it's down to available funds and other priorities such as existing NHS services, education, military, etc. Also, the odds of discovering a new drug and bringing to market and the humungous costs involved mean that drug development is extremely high risk.

The current system appears so exploitative. I get that there isn't a finite budget for healthcare but it seems unethical to deny people in great need something that will help them unless they give the pharma seller a huge amount of something they want, but don't actually need. It's like this: my only cow dies. You have three (probably through good luck at least as much as merit). I ask for one of your cows. You (pharmaceutical company) refuse to give me one unless I give you something in return, something I may not have. Consequently my family has no milk and suffers accordingly.

Sorry, but I don't understand how your example with the cows relates to medicines and pharma. If you'd like to ask again in a different way, hopefully I'll get it next time and be able to reply.

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QALY · 14/11/2018 23:04

Katy

How do you feel about the areas where little or new new research is being done (small cell lung cancer - my dad was told the treatment hadn't changed in 30 years or B12/Vit D where more work needs doing)

Some diseases attract more research funding than others and not necessarily based on greatest need. Great strides were made in HIV treatment I understand due to the extensive lobbying by patient advocacy groups and subsequent research funding. Similarly, breast cancer has attracted a lot of funding and a lot of new treatments have been developed. In some diseases it may be that the science just isn't there yet to enable new drugs to be developed, so more research is needed to understand the disease process first which creates a lag for subsequent drug development in that area.

I'm not aware of the B12/VitD research needs, but given these drugs have been around for decades there would be no incentive for a pharma company to conduct new research as the medicines will be off patent. This means that the pharmaceutical company would not benefit (as they wouldn't get more sales/profit) from conducting further research to extend the licence to new indications.

Or when you hear that someone prints out the NICE guidelines and takes them into the GP but the GP ignores them

NICE guidelines are evidence based guidelines that make recommendations that are suitable for the majority of patients with a specific condition. They are however a guide to clinical practice, and the GP, or other clinician, has to take many other factors into consideration, such as the age and overall heath of the patient, other conditions the patient has and other medicines the patient takes. The NICE guidelines include the following advice for clinicians to take the guideline recommendations in context (Source: www.nice.org.uk/guidance/cg146):

"The recommendations in this guideline represent the view of NICE, arrived at after careful consideration of the evidence available. When exercising their judgement, professionals and practitioners are expected to take this guideline fully into account, alongside the individual needs, preferences and values of their patients or the people using their service. It is not mandatory to apply the recommendations, and the guideline does not override the responsibility to make decisions appropriate to the circumstances of the individual, in consultation with them and their families and carers or guardian.

Local commissioners and providers of healthcare have a responsibility to enable the guideline to be applied when individual professionals and people using services wish to use it. They should do so in the context of local and national priorities for funding and developing services, and in light of their duties to have due regard to the need to eliminate unlawful discrimination, to advance equality of opportunity and to reduce health inequalities. Nothing in this guideline should be interpreted in a way that would be inconsistent with complying with those duties.

Commissioners and providers have a responsibility to promote an environmentally sustainable health and care system and should assess and reduce the environmental impact of implementing NICE recommendations wherever possible."

If I or a family member/friend experienced a GP ignoring NICE guidelines I would want to understand what factors meant that an alternative clinical management plan was considered more appropriate in this instance. And/or I'd see a different GP to get a second opinion.

OP posts:
QALY · 14/11/2018 23:29

Faintly

My question had been going to be, whether you feel the NICE process delivers a reasonable balance of value-for-money vs availability of drugs? And whether the UK is fairly well-served by it?

NICE does a huge amount of work above and beyond the technology appraisals of new medicines that I am mainly involved in. As I mentioned above, this process is very robust, but of course that makes it very costly and onerous, however overall I think it does benefit England and Wales. Currently the new drug appraisals are funded by the Government (Dept of Health) through taxpayers money, but recently NICE have consulted regarding pharma paying for the assessment of their new medicines, so this may change things. In Scotland, the SMC tends to make very similar decisions with a more streamlined and some would say more efficient assessment process. I have a huge respect for the work that NICE, SMC and AWMSG does and all three bodies are well regarded around the world.

If you're interested in the detail, the NICE website has a summary of decisions made on new medicines, which shows that 15% were not recommended at all after assessment. www.nice.org.uk/about/what-we-do/our-programmes/nice-guidance/nice-technology-appraisal-guidance/summary-of-decisions Some medicines get an optimised recommendation, which means that the drug isn't recommended for the full licensed population but a sub group of patients where the drug will be more cost-effective.

OP posts:
PerkingFaintly · 14/11/2018 23:30

Some countries reference UK, and other country, prices so this can drive prices higher in the countries referenced due to the impact downstream.

Many other countries reference UK prices, but at the same time we have NICE, etc which means medicines still need to be considered cost-effective

Wow. Do I understand correctly that NICE is effectively helping to keep drug prices lower across the world?

Because if a pharmaceutical company prices too high, it loses almost all of the UK market. But the UK price acts as a benchmark for other countries.

PerkingFaintly · 14/11/2018 23:32

Thank you for your answer and further links.

ShovingLeopard · 14/11/2018 23:44

Thanks for answering, QALY.

QALY · 14/11/2018 23:53

Faintly

I may have inadvertently opened a can of worms here!

I wouldn't say NICE are explicitly keeping drug prices lower across the world. It's very complex and I don't understand all of the intricacies of reference pricing, but rather if the pharma company wants to get a positive recommendation for a new cancer drug then the price can only be so high otherwise there is no way it could be considered cost effective, when the benefits and other non-drug costs are taken into account. There is an official list price, which is what is referenced by other countries, but companies can set a list price and then offer a discount to the NHS, which is included in confidence as part of the NICE process, so NICE calculations take account of the discounted price. This allows companies to maintain a higher UK list price for international drug reference pricing purposes and at the same time offer a confidential price to the NHS that offers better value for money.

The US tends to have much higher drug prices than we do in the UK.

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PerkingFaintly · 15/11/2018 00:53

Very interesting! Thank you for answering.

KatyMac · 15/11/2018 10:51

Thanks

It's sad when some old things (like B12 and D) can be used in so many different ways now more is known about how they work

A bizarre thing I've always felt is that if you have ME/CFS you can't then be tested for B12 (according to NICE - if I read it right)

QALY · 15/11/2018 12:04

Katy

I've just had a quick look at the NICE website & the current guideline (www.nice.org.uk/guidance/cg53) is now 11 years old and being updated with publication due in 2020.

I can't find any reference to Vit D in the original guidance doc but regards to B12 it states as part of initial investigations: "1.2.2.5 Tests for vitamin B12 deficiency and folate levels should not be carried out unless a full blood count and mean cell volume show a macrocytosis." and in respect of complementary & supplementary therapies "1.4.7.2 There is insufficient evidence for the use of supplements – such as vitamin B12, vitamin C, co-enzyme Q10, magnesium, NADH (nicotinamide adenine dinucleotide) or multivitamins and minerals – for people with CFS/ME, and therefore they should not be prescribed for treating the symptoms of the condition. However, some people with CFS/ME have reported finding these helpful as a part of a self-management strategy for their symptoms." (I've added the bold). I think this latter sentence leaves it very much open for clinicians to support patients if they find it helps relieve symptoms. And re the test, it recommends that B12 testing isn't done except in patients where full blood count and mean cell volume show a macrocytosis. This may be because it isn't clinically relevant to test B12 without macrocytosis being present (my clinical knowledge of this area is not up to date so I don't know the actual reason), or the evidence at the time wasn't sufficient to enable NICE to make a recommendation for wider testing.

The new guideline www.nice.org.uk/guidance/gid-ng10091/documents/final-scope will be reviewing the latest evidence and will cover diagnosis and management so this may change the formal recommendations. The stakeholder meeting mentions vitamins as one of the priorities for the new guideline: "Guidance on interventions, including but not limited to exercise, CBT, pacing, dietary supplements / specific vitamins, Vitamin B12 treatment for children, pain management" www.nice.org.uk/guidance/gid-ng10091/documents/interim-findings and the draft scope consultation responses mention vitamin D & B12 in a few places (it's a big document but you can search for vitamin to find each time it is mentioned. www.nice.org.uk/guidance/gid-ng10091/documents/consultation-comments-and-responses-2

However, given the current guideline is being updated I would expect clinical practice to have moved on in line with any new evidence. The updated guideline will review all of this new evidence in a systematic way and assess what good clinical practice should look like now so that they reflect the latest and most robust evidence base.

If you are interested, take a look at the patient groups involved (look at the Stakeholder List and the patient groups who have already responded to the draft scope) and get involved via the patient group you feel is most appropriate for you. NICE really does take patient and clinician views into account as well as the published evidence base so the more proactive the patient groups are in getting involved in the process the better.

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KatyMac · 15/11/2018 12:40

Thanks - I'll have a look

At least we aren't being told about graduated exercise anymore!

My problems are being aleviated by a cocktail of supplements started by a referral to oral health for recurrent ulcers - who tested for Zinc and suggested testing for B12 (as they weren't allowed to it was a GP thing) and in reading (MN mostly) asked to be tested for D

I self medicate and it changed my life - placebo or no placebo it works for me Wink Sorry, not relevent to the thread anecdotes not data!! sorry

Thanks for telling me about the change - there is a big one for B12 recently which I didn't know about either

PerkingFaintly · 15/11/2018 17:55

Oh I know something else I wanted to ask.

QALY, has your employer joined All Trials, the project to ensure all clinical trials report their results, not just trials that show what the companies wanted?

www.alltrials.net

If so, do you know if it actually does report all its trial results?