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Celgene UK: market access is a complex business

Published on 15/12/14 at 07:55am
Sam Pierce image
Sam Pearce, former vice president and general manager of Celgene UK

Being a firm that specialises in cancer drugs, Celgene UK is no stranger to England’s notoriously rigorous approach to expensive medicines, and the company has been through many ups and downs with NICE over the years. 

In fact, Sam Pearce, former vice president and general manager of Celgene UK, considers the pricing and market access environment in the country to have been the ‘biggest challenge’ of her time at the company. 

“The system in the UK really is quite complex,” she says, “and the decisions that we take here do have a global impact, so it’s really important that we get it right.” 

Pearce, who now heads up Celgene’s EMEA emerging markets unit, joined the company in 2010 – four years after it established itself in the UK as a subsidiary of the US firm, hoping to support its blockbuster multiple myeloma drug Revlimid (lenalidomide) as it entered the country.

In that time the firm’s offerings have of course been through NICE’s system, producing both positive and negative results. Most recently, the company’s drug Abraxane (nab-paclitaxel) was rejected for use in untreated metastatic pancreatic cancer because the watchdog found it to have ‘limited benefits’ compared to current treatments – on top of being more costly.

Revlimid itself has also faced a rocky road in the UK. It was initially rejected by the NICE in 2008 on the grounds of being too expensive, despite much anticipation from patients. The drug was only approved for use in combination with the standard treatment dexamethasone after Celgene agreed a patient access scheme with the pricing regulator.

Later, it was rejected for the treatment of myelodysplastic syndromes, but after a lengthy appraisal process it has now been approved in this indication. This year the pricing body once again issued a preliminary decision not to recommend Revlimid, this time for the treatment of multiple myeloma patients who have already received Janssen’s Velcade (bortezomib). 

Sir Andrew Dillon, chief executive of NICE, explained at the time: “Since 2009, no clinical studies have been undertaken specifically looking at how well Revlimid works compared with other treatments for these patients. Following the publication of draft recommendations earlier this year, [Celgene] provided the appraisal committee with further evidence and analyses to consider. 

“However, these further data did not lessen the uncertainty in the results presented because there is very little evidence in the population we are currently appraising this drug for, and the analyses indicate that lenalidomide is not a good use of NHS resources.” 

The drug costs £3,570 for the smallest dose of 5mg, and £4,368 for the highest of 25mg in 21-capsule packs – with most patients starting on a daily dose of 25mg, so it is easy to see why when price is a major deciding factor, the watchdog might balk at approving it.

Celgene says that it is continuing to work closely with NICE and understands that the final decision will be made at the beginning of 2015. “It’s a tough environment,” says Pearce. “We’ve seen very few cancer medicines actually being approved by NICE in the last couple of years.” 

In fact, last year the body rejected all of the cancer drugs for which it issued final guidance – although historically it has approved the majority of drugs that it has assessed, with a rate of 63% for cancer. 

This change is indicative of how price remains a sticking point for both sides as cancer drug costs increase, with pharma blaming NICE’s system for being too strict and the watchdog condemning the high prices set by firms. 

Strong growth

Celgene has had impressive growth over the last few years, which means that any difficulties with NICE may do little damage to the company. Becoming a strong contender in the oncology market, it ranked fourth in sales worldwide for 2013, with third quarter revenues of $1.9 billion this year – an increase of 19% over the same period last year.

It also has the funds to recently enter into a new $1 billion dollar agreement with Sutro Biopharma to develop immuno-oncology therapies. 

Pearce says that Celgene UK in itself has seen nothing but growth since its inception, before adding: “When I arrived there were 48 people and there’s now 160. We had revenues of $80 million dollars back then, and now we’re pushing $300 million. The company had a tiny little office in Windsor, and now we’re in a fantastic building in Stockley Park. It’s really been quite a dramatic change.”

But even though NICE is not yet starting to bite Celgene’s profits, Pearce still feels that the uptake of cancer medicines in the UK is too slow. “It’s obviously a big issue for patients. So I do think that it needs reforming. We’ve got some of the lowest prices in Europe, and yet we still have a reputation in the UK for low and slow uptake. And we have a record in cancer that could definitely be improved upon.”

She argues that the inevitably low numbers of patients in studies for rare diseases can be one of the main roadblocks when seeking approval, besides high prices. “There’s always so much uncertainty in the modelling that’s done, and NICE will often point to that as a reason for either not approving the medicine or demanding a lower price.”

The government is suggesting that two new criteria be introduced for assessing a medicine’s cost – ‘burden of illness’ and ‘wider societal impact’ – which may give some drugs a better chance than they would have otherwise had. The future of these proposals remains in doubt at this stage however. 

Cancer Drugs Fund: the right solution?

For the next couple of years, both cancer patients and pharma firms have the Cancer Drugs Fund (CDF) to fall back on in the event of NICE rejections. The Fund began in April 2011 with a budget of £200 million a year.

Increasing cancer drug costs and the resulting failure of many treatments to be approved by the watchdog have now led the government to raise the budget to £280 million a year.
It has also introduced cost-effectiveness assessments for new drugs entering the list.

Celgene has benefited much from the silo budget so far with Abraxane, Revlimid and Imnovid currently being funded by the CDF. But Pearce thinks that the Fund is a short-term solution, and an unsatisfactory one at that.

“On the whole I think it’s been a positive thing, because there have been thousands of patients treated who otherwise wouldn’t have been. But there’s probably need for a more holistic reform of the reimbursement approach that would enable medicines for rare diseases to be evaluated fairly.”

The CDF has been criticised as being unfair by favouring cancer over other serious diseases, but Pearce sees it more as a problem with the UK system as a whole.

“Obviously it depends on your perspective. If you think back to the reasons why the CDF was introduced, patients were demanding these medicines. There was a lot of disquiet with the patients and the patient organisations as to why people in the UK were being denied innovative new treatments for cancer. 

“Cancer is one of those diseases where there’s been so much innovation, and patients in the UK want access to the latest treatments. But I don’t think [the CDF] is a long-term answer.

“I would like to see a clearer link between the strategic health priorities in the UK and the investments that are made. So if cancer is a priority then yes, let’s invest in cancer. If dementia is a priority, let’s agree that we’re going to invest differentially in drugs for dementia.

“But that link doesn’t really seem to exist. [Almost everything] is taken with £20,000 or £30,000 per QALY. It’s very much a one-size-fits-all approach, and as a result it doesn’t really work in all areas.”

PPRS concerns

Pearce also voices disappointment in the new 2014 PPRS deal, which introduced growth caps for the pharmaceutical industry. If companies exceed these caps they must pay the extra money back to the Department of Health. So far the industry has paid nearly £180 million in rebates for 2014. 

Pearce was one of nine signatories of an open letter raising concerns about the deal. The countersigners, who also included the UK heads of Novartis, Pfizer and Sanofi, said that they believed ‘a critical opportunity’ had been missed to “improve the health of the British public and wealth of the British economy,” and that the deal had “failed to break down the barriers” preventing patients from being treated with innovative new medicines.

“We all accept there are increasing pressures on NHS spending, but there is a prevailing myth that medicines are expensive. 

They pointed to the fact that spend on medicines was less than 10% of total NHS expenditure in 2011 – though it has risen to more than 10% in the years since, as last year the medicines budget was £13 billion and the NHS budget was around £108 billion. 

The letter went on: “Medicines should not just be seen as a cost. They are an investment and an essential part of improving patient outcomes. At a time when there is fierce global competition to attract investment in life sciences, the commercial environment is critical […] Action to manage costs should be matched with action to drive better health outcomes.” 

ABPI chief executive Stephen Whitehead, while acknowledging that many companies would find the deal ‘extremely tough’, has strongly defended it – describing it as “fundamentally the best deal we could achieve in very tough times.”

He says that the fact that so many firms have agreed to it – 93% of the industry, up from 88% for the previous deal – means that it “provides a game-changing opportunity to improve the UK environment and address the long history of low patient usage of innovative medicines in the UK, which has been getting progressively worse over the life of the last scheme”.

Pearce stands by her initial criticisms, though. “The PPRS was described as the deal for innovation, but so far I think it has been a missed opportunity. I think what I would have liked to have seen is the NHS really grabbing this opportunity, and I don’t think it’s understood well enough by enough people.

“I think we would always still need a form of assessment to ensure that the right investments are being made, but why not take the opportunity now to loosen the purse strings in a controlled way knowing that any up uptick in spending is going to be rebated by the industry? 

“I think it would have been a good thing to see what kind of long-term value that would bring. It would have been a really nice experiment to see whether we got the health benefits going through with improved access to medicines.”

Communicating to patients

To the average patient, NICE rejections and disapproval of the deal may seem like little more than pharma firms valuing finances over the very patients they insist come first. 

“I think the industry as a whole really needs to engage proactively in those types of discussions,” says Pearce. She goes on: “[The industry] really needs to get clients to understand the value that we do bring, and I don’t think we’ve always necessarily done that as well as we could.” 

Digital media is one communication path many companies are increasingly taking to try and engage with patients more – from the high-profile gamification efforts of Sanofi and Boehringer to the strong social media presence of companies like Johnson & Johnson and GlaxoSmithKline. 

However, Pearce says that while Celgene certainly sees the potential of these efforts, it is cautious about jumping in too soon. “[Celgene] hasn’t been a pioneer in that space. We’ve been watching and evaluating the trends and the impact. But I do think the direction of travel is clear and we need to think forward – how will doctors or patients be engaging with healthcare five or ten years from now? And obviously we need to be a part
of that. 

“I think the opportunity is significant. There have obviously been concerns from a regulatory perspective as to how we use it in a responsible way, but I don’t think there’s any denying the direction that’s going in. 

“Celgene, like other companies and the industry in general, needs to figure out how we’re really going to maximise this for the benefits of patients.” 

The future of the UK market

Despite her criticisms of the UK system, Pearce says that she still sees significant opportunities in the future of the market. 

“It’s quite a stable environment, and we do see a real positive amount of rhetoric coming from both the Labour and Conservative parties about support for the life science industry, and a real appreciation of the kind of value that the industry brings – both economic and health value. 

“I can’t help but feel optimistic for the future because I think there is a real commitment to health, and there’s a real commitment to innovation, and I think there’s a commitment to the life sciences sector overall as well, and that’s got to be a good thing.” 

However its relationship with UK regulation goes, she is confident for the future of Celgene itself. “Celgene will continue to focus on bringing innovative medicines through and developing them in areas of high unmet need, and we will continue to be committed to ensuring that patients in the UK can access
those medicines. 

“The tough environment means that we have to work even harder to make sure we’re delivering real value to the NHS, value that they want to pay for and are prepared to pay for.” 

George Underwood

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