NICE and market access: time for reform?
pharmafile | August 26, 2014 | Feature | Business Services, Manufacturing and Production, Medical Communications, Research and Development, Sales and Marketing | ABPI, Bristol-Myers Squibb, NICE, Yervoy, reform
Earlier in the year, at the ABPI’s annual conference, the UK pharma lobby group released a new report suggesting that market access in the UK was a major problem.
And the source of its concerns was the role of England’s health technology assessor, NICE, which was created in 1999 to help ensure new medicines were a cost-effective use of NHS resources.
It’s a powerful organisation as it can restrict or completely bar a new drug from being funded by the NHS in England. Even when it does recommend a treatment, it can still take between nine months and a year before a patient can access it, and it regularly denies new and expensive drugs – especially cancer treatments – given their high cost.
The ABPI’s new president Jonathan Emms, who also heads up Pfizer UK, has been a vocal critic when it comes to some of NICE’s decisions and echoes the trade body’s concerns that market access is being hindered by the watchdog.
He tells Pharmafocus that enough is enough and that NICE needs major ‘political reform’, rather than internal tweaking, to help increase market access in England.
But this kind of reform is not on the cards. The new chairman of NICE, Professor David Haslam, tells Pharmafocus that the body is going through a consultation process which could open it up to saying ‘yes’ to more medicines, but this is not being directed from the government.
Haslam says the consultation is still valid and is quick to dismiss that issues of poor uptake are all centred on NICE.
“I don’t think it’s specific to the UK: the issue of market access is worldwide,” he explains. “I’ve certainly been involved in discussions in several countries – before becoming chair of NICE in 2013 – about this issue and there is a desire from everybody to get the right drugs on the market as speedily as possible.”
He says that this may require new systems of adaptive licensing, which is something EU drugs regulator the European Medicines Agency (EMA) has now begun, with several pilots being launched in June.
Adaptive licensing, as discussed by Steve Bates at the start of this supplement and also called ‘staggered approval’ or ‘progressive licensing’, is a new policy that the EMA hopes will allow quicker uptake of new medicines.
“If that were to be adopted in Europe, then those discussions need to be had. But I understand that pharma is keen to get drugs out earlier.”
But when it comes to access it goes further than just NICE being – or not being – a barrier, he argues, and things could be sped up in many ways, not least by changes within the industry. “I think the large, multi-centre, double-blind, control-trial methodology isn’t necessarily appropriate for many of these conditions.
“As we go forward […] maybe trials need to be run differently. That’s because there are new ways of looking at how research and evidence can be developed and, really, we’re right at the beginning of that.”
The need to collaborate
But NICE is still central to market access and fixing issues around uptake will need to focus primarily on the pricing body.
One way this may be done is by closer collaborations, rather than the industry pointing the finger at NICE, whilst it simply points straight back.
Speaking to Pharmafocus Johanna Mercier, the new general manager of Bristol-Myers Squibb in the UK and Ireland, explains how her company’s innovative immuno-oncology product Yervoy (ipilimumab) eventually passed through a tough NICE appraisal after receiving help from the watchdog.
NICE initially rejected Yervoy as a first-line treatment for melanoma patients in its draft appraisal in 2013, saying it didn’t think it was cost-effective given its £80,000 price tag for a full course of treatment.
Mercier says: “When you look at Yervoy’s first-line licence, you can see that its EMA approval was based on real world data, and there was no Phase III trial data, so we knew that, given how NICE looks at these things, it was always going to be an uphill battle.”
NICE has a fairly strict set of rules it wants pharma to follow when submitting an appraisal, and this requires Phase III data for most new medicines undertaken by the traditional randomised clinical trial method although as Haslam tells Pharmafocus, NICE are opening themselves up to become more receptive to new ways of proving clinical efficacy.
Mercier says that she hoped the Cancer Drugs Fund (CDF), which pays £200 million out a year for new cancer drugs not recommended by NICE, would help bridge the gap, but the Fund wouldn’t pay out for Yervoy first-line, only second, which surprised Mercier given its positive overall survival data.
Asked why they didn’t recommend funding, she says: “I think they felt it wasn’t their responsibility because they really thought it was up to NICE to make the decision. I mean if you look at the review, the CDF panel actually thought that Yervoy first-line was absolutely producing a strong benefit for patients, so they supported it.
“I think the thought was that if NICE doesn’t come through [and recommend Yervoy first-line], that we’d go back to them for funding, but they just wouldn’t recommend it before NICE had made a decision.”
But BMS did not want to rely on the CDF to fund its drug in a lower setting, and pushed on with the appraisal. From the outset the entire appraisal seemed as if it would be a major battle but Mercier says that on the contrary, NICE actually helped the UK team along, and even came out publically saying Yervoy was a ‘breakthrough’ drug, a label it rarely gives to medicines it is assessing.
“But working closely with NICE we were able to understand what their concerns were and then make sure we gave them the analysis they need, and it really helped when we got that feedback,” she explains.
In the end, NICE gave Yervoy first-line its blessing to be funded by the NHS in final guidance which was published in June this year.
“I’m really impressed that NICE came through with a positive decision,” Mercier says. “I mean I would not have been happy with a negative decision, but I would have understood, because of their process. So I think they were actually quite flexible in their approach because it’s rare that you get an HTA approval based on real world data.”
She says that in the case of Yervoy, NICE just needed to find a path forward. “So that’s a good example for me that NICE wants to do the right thing,” Mercier says. “It could have turned the other way and then maybe we’d be having a different conversation, but overall I’m really impressed with that, because I think that’s a good step forward not just for BMS, but for other organisations as well.”
UK still a ‘challenging market’
“Now, having said all of that, I would tell you that access in the UK is still very challenging,” Mercier admits. She adds that she has also worked in Europe, the US and her native Canada and come across a host of different, and sometimes similar, market access issues.
But it’s not just about NICE, she argues, but also about how market access is seen in the UK. She says: “When I began my job as general manager in the UK, here people were saying ‘Oh, it’s an early launch market’ and that this was a positive thing. But I was challenging my team saying you’re not an early launch market – you get an early price, but not an early launch because the NHS won’t pay for it until it’s been appraised by NICE, and then put onto a formulary, all of which can take up to a year.
“So there’s a disconnect between an early price versus early access, which are two very different things, and I think people are confusing the two. So I think there is an opportunity to bridge that gap and there are different ways of doing it.”
So despite the help NICE were willing to give with Yervoy, she still believes that “there is need of an overall reform of NICE”, adding that this is ‘around the corner’.
“We just need to partner together to make sure we get the right outcome for patients. It might take a couple of years, and I don’t think it will be immediate, but if there is intent on both sides, you can make it work.”
She points to the European Medicines Agency as one body that HTAs such as NICE can look to for guidance.
“The EMA is moving pretty quickly forward with filings with Phase II data only, or with real world data. They are really trying to progress with these new ways of gathering data. We in the UK and elsewhere just need to make sure that our reimbursement processes are falling in line with that, because at the moment there can be a real disconnect. And I think what happened with Yervoy first-line is a good example where they want to go.”
A need for reform?
So whilst collaboration is helping, the over-riding feeling among UK pharma is still that NICE needs a fairly big shake up if more medicines are going to come through to patients more quickly.
At the time of press, the consultation on NICE’s reform was still out for discussion and no changes have yet been wrought.
But Emms says he doesn’t expect any major re-working of its QALY cost-effectiveness formula or even its policies, given that the reform is not being directed by Whitehall.
Pharma has spent many years banging the drum over NICE and yet only small changes have ever been wrought, such as the ability to effectively lower prices with a patient access scheme (although this is done with the Department of Health, not NICE) and the end of life criteria that should see more drugs recommended, although it is very rarely used.
In truth the government tried to nullify NICE’s HTA role in 2010 when it also reformed the structure of the entire NHS. In the end, the then health secretary Andrew Lansley was defeated by strong opposition from medical groups that wanted to see NICE remain and its HTA status guaranteed.
This is, politically at least, why the government will not want to intervene in reshaping NICE. The latest consultation may see some changes, but the role and ethos of NICE will most likely remain intact.
But does it really need reform? The insight garnered from Mercier suggest that in fact, having pharma and NICE work together more closely could yield more positive recommendations from the watchdog whilst also ensuring a better collaborative relationship between pharma and HTA.
This is appears a more pragmatic, cheaper and positive way forward for all stakeholders, and would still lead to the ultimate goal, which is getting new medicines to patients as quickly as possible.
But Haslam will not be drawn on NICE’s role in the slow adoption of new medicines. The ABPI tells Pharmafocus simply that: “There is an issue with slow access to medicines”, but adds that there are avenues outside of NICE able to help speed things up. It says that NHS England is beginning to look at this issue, through areas such as commissioning through evaluation.
The ABPI explains that it is “working with NHS England to ensure that patients get access to the innovative medicines they need”, with some new innovations coming from new policies such as the Cancer Drugs Fund and more recently, the silo fund for Gilead’s hep C drug Sovaldi.
This is one of five special features from out latest supplement The Battle for Market Access, which is available to view online here.
Related Content
Digital mental health technologies – a valuable tool in supporting people with depression and anxiety
The potential benefits of digital mental health technology for managing depression, anxiety and stress, together …
Combination treatments: Takeda’s Implementation Framework and the broader landscape
Pharmafile talks to Emma Roffe, Oncology Country Head (UK & Ireland) about the combination treatment …
NICE recommends Pfizer’s new once-weekly treatment for haemophilia B on NHS
Walton Oaks, 21st May 2025 – Pfizer Ltd announced today that the National Institute for Health and Care …
