NICE-Approved Products: Patient Access

Anne Marie Morris Excerpts

Tuesday 30th November 2021 Commons Chamber

[Anne Marie Morris (Newton Abbot) (Con)]

I rise to address the challenging issue of access for patients to medicines and medical devices. We all believe absolutely passionately that we should have access to doctors and nurses, good hospitals and operating theatres, but I think the pandemic has shown beyond question that access to medicines and medical devices goes hand in hand, and without that we do not have the NHS that I think we all believe everyone deserves.

What is the problem? The problem, I shall explain, is as follows. When a medicine is approved, it goes through two processes: first, with the Medicines and Healthcare products Regulatory Agency, which checks whether or not a drug is actually safe and does effectively what it says on the tin; and then it goes to a separate process run by the National Institute for Health and Care Excellence, which looks at cost-effectiveness and value for money. The theory goes that, once those two hurdles have been passed, the medicine is then accessible to anyone. It is very clear in the NHS constitution, which explains that there is a legal right for people to have NHS NICE-approved drugs if it is right for their particular circumstances. Indeed, the NICE guidelines say that there should be automatic adoption, if clinically appropriate and relevant, within 90 days of approval. So where is the problem?

The problem is partly in the system and partly in the words. The words in the constitution are effectively caveated: people can have a medicine if it is right for their particular circumstances. Likewise, according to the NICE guidelines, it will be automatically adopted if clinically appropriate and relevant. The challenge is that, in the current system, NICE will approve medicines for the condition for which they are most cost-effective, so in this country we do not have the ability to approve a medicine for multiple conditions—multiple indications, in the jargon. It is what is most cost-effective that gets approved, and others do not, and there is not a system, either than paying privately, to ensure that the medicine that has been approved for the condition for which it is most cost-effective is available to those with other conditions, but that medicine may in fact still be the only possible solution.

Assuming we get over that hurdle, there is a second hurdle, because not only must NICE have approved the drug, but it has to go on the approved list of drugs locally in the local health authorities—now integrated care systems. The problem is that to get on those formularies, somebody has to put it on those formularies. Currently, while in theory under the NHS NICE guidelines there is a system, it does not actually happen. There is currently a drug for multiple sclerosis, and research shows that people are still waiting after 150 days for it to go on the formularies in something like 25% of the local health systems across the country. So the system, fundamentally, does not work.

What does this result in? It results in a postcode lottery. If someone has type 1 diabetes, it is absolutely crucial that they monitor their condition. There is a device, a flash monitor, that is state of the art, and research shows that the uptake across the country varies between 16% and 65%. What is most worrying is that those parts of the country with the greatest levels of deprivation have the lowest levels of uptake. We all think we have access to medicines for cancer given that we now have the highly innovative and very welcome cancer drugs fund. However, that drugs fund is only relevant when the particular drug is approved for a particular type of cancer. So there will be some drugs—Avastin, for example—which those with the appropriate cancer can get through the CDF, but those with a different type of cancer or who do not fit the profile again have to pay for it privately, costing £252 to £1,088 per cycle, which is every three months.

Sadly, NICE does not approve much for those with skin conditions. For those with mastocytosis—blotches on the skin and boils causing vomiting and diarrhoea—the only solution is usually NICE-approved food allergy drugs, but they are approved for NICE allergies not skin conditions so they have to be paid for privately. For cystinosis, the accumulation of amino acids, which gives rise to kidney problems and kidney damage, the drug Procysbi has been approved by NICE but, bizarrely, there seems to be no uptake of that to date at all. That is important because that drug is, unlike the existing drug, a slow-release drug and therefore mums and dads do not have to keep waking up their kids in the middle of the night to give them the next dose, which, as we can imagine, takes a real toll on family life.

For those with an obesity problem there is a good solution in Saxenda, but that is approved for diabetes. So those whose obesity does not give rise to diabetes will not get access except by case-by-case approval. I am pleased to say that Imperial has finally accepted and approved.

How are we going to resolve some of these issues to make sure there is no longer a postcode lottery? First, let us look at the simple case of those drugs that are deemed to give the most health benefit and are therefore in theory approved and people can get hold of them. What can we do to make sure they do actually finally appear on those formularies and how can we then make sure the system for take-up is actually in place? One of the problems is that there is nobody sitting in these health bodies who monitors NICE drugs coming up, and therefore no one who looks to see whether in their health community they may be of benefit.

[Jim Shannon (Strangford) (DUP)]

I congratulate the hon. Lady on securing a debate on this topic. As she says, this is about NICE-approved products as well as drugs. One of those products is cognitive rehabilitation therapy, an important intervention for those with dementia, enabling them to live independently for longer. However, despite being listed in the NICE recommendations there is a barrier to delivery. Occupational therapists and other staff in memory clinics do not have the capacity to deliver programmes that are National Institute for Health Research and Alzheimer’s Society-funded. Does the hon. Lady agree that the NICE recommendations must address not just drugs but also products?

[Anne Marie Morris]

I could not agree more. It is crucial to realise we are talking not just about medicines and drugs but also devices and, as the hon. Gentleman says, the processes, which are often the connection between the medicine, the device and the patient.

Within these health bodies, there is no training and nobody specifically focused on monitoring innovation, and there is no obligation to prescribe. Even more peculiarly, for these drugs there is an agreement between the industry and the NHS, the voluntary payment scheme or VPAS, under which manufacturers that are members of the scheme effectively agree with Government when the medicine or device is approved that it will be supplied at a well-discounted price. In addition, there is an agreement that sets a cap so that if, as it happens, more prescriptions are written for that particular drug, it is agreed that the extra cost that the NHS has incurred will be reimbursed by the manufacturer.

So if there is excessive prescribing—we assume that is why there is an attempt to limit how much goes on to formularies—why is that a problem when we have the VPAS scheme? It is a problem because the scheme does not the pass the benefit, other than the reduced price, down to the local health authority. The money is put into a separate pot, and that pot is then used generally to support the NHS writ broadly; it is not ringfenced, either for medicines or to be used, as it could be, to support local health authorities—integrated care systems—when their budgets are put under pressure, which is why they do not want too many things on their formularies. This would help them pay the price.

It seems to me, Minister, that there are some solutions here. You will be aware that I raised three of them in proceedings on the Health and Care Bill. To deal with the imperfections of the current arrangements, if those drugs that were approved by NICE—we are talking about the most cost-effective drugs—were mandated to be on formularies within 28 days automatically, so no one had to decide whether they went on or not, that would be a good system. It would also ensure that the decision was in the hands of the clinician and not of the bureaucracy of the health authority.

It seems to me, Minister, that the second suggestion I made—

[Madam Deputy Speaker (Dame Rosie Winterton)]

Order. It is quite important not to address the Minister directly; he should be addressed through me.

[Anne Marie Morris]

Thank you for the correction, Madam Deputy Speaker.

My second proposal is that there should be an innovation officer specifically appointed to look at and manage these issues. Failing all else, there should be a final provision that specifically puts an obligation on the health authority to provide any NICE-approved medicine. Then, as a matter of practicality that is not a matter for the Health and Care Bill, there should be a new arrangement under which the NHS would agree that this pot, rather than going into the general NHS coffers, is put aside specifically to reimburse local authorities.

What about the second category—those drugs that are approved by NICE but not used for the most cost-effective indication? That is called off-label prescribing. We know that a drug in that category does what it says on the tin and we know that it is cost-effective, but it is not sufficiently cost-effective to have got a tick in the box from NICE. Access then is a matter for negotiation between the integrated care system, the NHS and the manufacturer. Often, the starting point will be the list price, but bear in mind that that list price is a lot more than the NHS is paying. The result is that in different health authorities and different hospitals, different patients are offered different costs to be able to access the drug.

There is a real problem on top of all this. To ensure that there is no gaming of the system between access to private healthcare and state healthcare, someone cannot mix the two—quite rightly, we do not want gaming of the system so that people can effectively jump waiting lists—so there is an agreement to enable people to do a bit privately and then jump straight back into the NHS. If there is an episode where someone takes a drug and pays for it privately, technically they cannot then have the ongoing monitoring of their cancer—screening and so on—on the NHS. Individual health authorities recognise that that is rather absurd when we are talking not about a private patient with health insurance but about someone for whom the only way to get the drug is to pay for it, but that recognition is not universal.

So what is the solution for this one?

We need to look at NICE, supported by NHS England, accepting multi-indication approvals. That means a drug company can take a drug and apply, at the same time, for the drug to be approved for different health issues that have different ranges of health benefit. Clearly, the NHS will say, “Hold on a minute. I am not going to pay the same price for something which delivers less health benefit.” That means we need a system of differential pricing, so that a different amount is paid by the NHS for the drug, depending on the use to which it is to be put. That is entirely possible. It is done all over the world.

The Office of Health Economics put out a report, an international study, just this month on “Payment Models for Multi-Indication Therapies”. It concluded:

“Inflexible uniform pricing does not optimally support innovation and access. The most important consequence is lost treatment opportunities for patients.”

I respectfully suggest that the NHS and the Department of Health and Social Care might sensibly look at that. I also suggest, given the clear importance of payment support through industry, that as we move to the next iteration and renegotiation of VPAS, the agreement under which the industry agrees reduced pricing for mass purchase above a certain cap, it will reimburse the Government. That renegotiation needs to include provision for the multi-indication approvals process and differential pricing. 2023 is not very far away and I urge the NHS and the Government to take it forward very quickly. Meanwhile, there should be agreed standard pricing which is effectively paid for and underwritten by the pot set aside as a result of the cap.

There is one final area that needs to be addressed: drugs approved by NICE through a managed access agreement. For some treatments—typically gene therapy, where you are taking body fluids out, effectively changing the genetic make-up, and then putting them back in again—a hub needs to be set up in a hospital. Often, when drugs are approved, exactly how they are going to be delivered is not approved at the same time. Those drugs and processes need to be agreed not just in principle, but together with a package that ensures they can be implemented. As things stand, it can often take three years and much argument before hubs are established and the funding can then flow.

In summary, I ask for a number of things. First, my three new clauses to the Health and Care Bill would require local health system formularies to include NICE-approved drugs within 28 days. Secondly, there ought to be included in the Bill an absolute obligation to provide NICE drugs. It would then be for the individual health authority to work out how to provide them, but there should be an underpinning payment mechanism provided by the NHS. Thirdly—again, I have tabled a new clause on this—we need an innovation officer to ensure the system runs smoothly and that the things that would give best benefit are put on those formularies.

Fourthly—forgive me, Madam Deputy Speaker, there are eight of these—I would like the Government to look very closely at a multi-indication approval system, fifthly, at a differential pricing system and, sixthly, at an appropriate VPAS agreement for 2023. My last asks are about how the money is used. Seventhly, the pot of money, an accumulation of money paid by industry because more of the drug was prescribed, should be made available to local health systems, the integrated care systems, to cover the costs that are not in their mainstream budget. Finally, we should put in place a formal delivery mechanism for every managed access treatment to ensure that it is not just a promise, but actuality. I wonder whether it might therefore be appropriate for this Minister, the life sciences Minister—the Under-Secretary of State for Business, Energy and Industrial Strategy, my hon. Friend the Member for Mid Norfolk (George Freeman)—and the innovation Minister, Lord Kamall, to meet me, because all three have a critical role, given their involvement in access, industry and licensing. I wonder whether it might be possible to persuade them to accept my three amendments to the Health and Care Bill.

It might also be helpful if these matters were raised with the National Audit Office, which could review the current system and look at whether it offers value for money. That would be a very good use of their time. I am sure that the doughty hon. Member for Hackney South and Shoreditch (Dame Meg Hillier), who chairs the Public Accounts Committee, might also be interested in looking at this issue. After all, value for money is critical.

In conclusion, we have a world-beating system but, as it currently stands, it does not provide value for money for all. It does not serve the best interests of all patients, and it does not serve the best interests of delivering the UK life sciences strategy. Indeed, I have one constituent who moved house from Devon to Southampton so that he could get a medical treatment.

[The Minister for Health (Edward Argar)]

As ever, I congratulate my hon. Friend the Member for Newton Abbot (Anne Marie Morris) on securing this debate and I pay tribute to her work in this area. That work has been sustained for as long as I have been a Member of the House—I was first elected in 2015—but I suspect that this area was of interest to her long before then. I pay tribute to her for her continued engagement on this area, which is not always top of the news or top of the political agenda, but is hugely important none the less, so I pay tribute to her work in this space.

I reassure my hon. Friend that the Government share this priority. She is right—I characterise this in these terms and I hope that she will not demur from this—that although the NHS workforce is the beating heart of our NHS, it is beholden on us to give them the tools that they need to be able to use their skills to treat and support the maximum number of possible patients in the most effective way. I think that is a fair reflection of the point that she made at the outset.

[Anne Marie Morris]

indicated assent.

[Edward Argar]

That is an encouraging start. That is why NICE exists. It plays a vital role in supporting patient access to new treatments. Through its technology appraisal and highly specialised technologies programme, NICE makes recommendations for the NHS on whether all new medicines represent value for money for the taxpayer.

NICE aims to publish guidance on new medicines as soon as possible after licensing and is committed to publishing draft recommendations on medicines around the time of marketing authorisation, wherever that is possible. It has a well-earned reputation as a world leader in the field of health technology assessment and it is right that, where NICE recommends a medicine or a medical device, it should be available to patients who stand to benefit. Value for money is important and I will come to that. I suggest that whether the NAO or the Public Accounts Committee wish to look at this is a matter for them, but as my hon. Friend said, she may wish to take that up with the hon. Member for Hackney South and Shoreditch (Dame Meg Hillier), who may well share her interest—I would not wish to presume, but the hon. Member may well.

Making treatments available is why NHS England and clinical commissioning groups are already under a statutory obligation to fund any treatment recommended by NICE through its technology appraisal or highly specialised technologies programmes, usually within three months of guidance. I will turn to my hon. Friend’s point on timing shortly. This will also be a statutory obligation for integrated care boards. As my hon. Friend has set out, the NHS constitution states that patients and the public

“have the right to drugs and treatments that have been recommended by NICE for use in the NHS, if your doctor”—

the treating clinician—

“says they are clinically appropriate for you.”

That is an important point, because we rightly rely on clinical judgment in such circumstances. As my hon. Friend will be aware, NICE recommends the vast majority of new medicines—indeed, in 2020-21 it recommended 100% of them, often thanks to commercial agreements negotiated between NHS England, NICE and companies in parallel with the NICE assessment process—but it is right that that clinical appropriateness filter is there.

I am conscious of time, but before I turn to specific points that my hon. Friend made, I would like to say a few brief words about the success of the cancer drugs fund in supporting patient access to new medicines. The cancer drugs fund was originally introduced to support patient access to medicines that NICE was unable to recommend as cost-effective. Since 2016, however, the fund has been linked to the NICE appraisal process and supports patient access to promising new medicines where the evidence is not mature enough for NICE to recommend routine funding at that point. This has benefited more than 73,000 patients, who have been able to access 91 medicines through the cancer drugs fund, treating more than 200 cancers.

We are building on that fabulous achievement with our manifesto commitment to extend the cancer drugs fund model to non-cancer treatments by creating an innovative medicines fund. I think that there are lessons that we can learn from the cancer drugs fund. NHS England has also recently launched a consultation on proposals for the establishment of the innovative medicines fund. I encourage anyone with an interest to engage; knowing my hon. Friend, I suspect that she has probably pre-empted me and done so already.

I turn briefly to medical devices and treatments. The hon. Member for Strangford (Jim Shannon) was quite right to highlight that, although debate often focuses on drugs and specific medicinal or therapeutic treatments, we also need to look more widely at medical devices and at treatments that take other forms than therapeutic treatment. I know that he takes a close interest in the treatment and support of those with dementia, as I did before I was a Minister; I am grateful, as ever, for his contribution to the debate.

As hon. Members will know, NICE can also make recommendations on treatments through its clinical guidelines programme. Guidelines provide authoritative, evidence-based guidance for healthcare professionals and play an important role in driving best practice in the health and care system and supporting improved patient outcomes. However, they often make dozens of recommendations that can be complicated to implement at a local level. For that reason, they are not mandatory, but I assure the House and my hon. Friend the Member for Newton Abbot that the Government expect the healthcare system to take guideline recommendations fully into account in designing services that meet the needs of the local population and in working towards their implementation over time. It is right, however, that that implementation reflects local circumstance and is done at a local level.

I turn to local formularies; time is getting short, but I may also turn briefly to the eight points that my hon. Friend made—let us see how we do. She has raised concerns, not only in this debate but during the Health and Care Bill’s passage through the Commons and in conversations with me, that despite positive NICE guidance, some medicines are not available to NHS patients because they are not included on local formularies. In addition to the statutory funding requirement that applies to NICE-recommended treatments, NICE’S guideline “Developing and updating local formularies” recommends that, when NICE approves the use of a medicine through a technology appraisal, it should be automatically adopted into local formularies.

The standard contract mandated by NHS England for use by commissioners stipulates that providers must ensure that formularies include all NICE-recommended treatments. That process should take place within three months, allowing services a realistic timeframe to prepare for the introduction of a new technology. I appreciate that my hon. Friend went a little further on tightening the timeframe and the compulsion element, but given the complexity of some new treatments, I think that three months probably remains an appropriate timescale. I suspect that she will continue to push me on it, but at the moment I believe it is the right approach.

My hon. Friend is right that the system needs monitoring. She called for an obligation on integrated care boards to report uptake of new medicines annually, which is effectively monitoring. I am pleased to say that the Government support the view that uptake of NICE-recommended medicines should be monitored. Since 2013, NHS Digital has published an innovation scorecard that reports, at a national and a local level, the uptake of selected medicines that NICE has recommended in the last five years. I believe that it is more appropriate and proportionate that that information is collected and published by a single national body using agreed methodology, rather than multiple organisations that may have different ways of measuring and presenting the data.

My hon. Friend made a number of other points. Given the time constraints, I fear that I cannot address them all, but I will reflect carefully, as ever, on what she has said. Given that neither of them are present in the Chamber, it is a pleasure, as always, to agree on their behalf that my noble Friend the innovation Minister and my hon. Friend the Member for Mid Norfolk (George Freeman)—the life sciences Minister—will be delighted to meet her to discuss the matter in more detail. I am very grateful to her for bringing this evening’s debate to the House.

Question put and agreed to.