as discussed over breakfast.

The future of market access

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CONTENTS

Foreword 3

Professor David Taylor 4 Emeritus Professor of Pharmaceutical and Public Health Policy, University College London

Leela Barham 5 Independent Health Economist and Policy Expert

Rt Hon Lord Hunt of King’s Heath 6 Labour Spokesman on Health

Dr Paul Catchpole 7 Value and Access Director, Association of the British Pharmaceutical Industry (ABPI)

Dr Nick Crabb 8 Programme Director, Scientific Affairs, The National Institute for Care and Health Excellence (NICE)

Biographies 9

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Foreword:

Lexington Health Practice recently held a breakfast event to discuss the future for market access in England. The breakfast, the first in a series, examined the Health Technology Appraisal (HTA) environment and facilitated a discussion amongst individuals who work closely with and amongst the pharmaceutical industry, examining how the mechanisms in place can be improved to ensure equitable access to medicines.

Since our breakfast the issues surrounding the future of market access in England have continued to evolve. The future of the Cancer Drugs Fund (CDF) and the Accelerated Access Review (AAR) are providing challenges to the appraisal and uptake of medicines with a widening gulf in opinion appearing between industry, the National Institute for Health Excellence (NICE) and the National Health Service (NHS). Industry and patient groups continue to claim the NHS is slow to adopt new technologies and have hit out at NICE calling its appraisal model ‘outdated’. Equally, NICE have derided industry for submitting poor quality evidence to support their cost-effectiveness calculations.

These divisions have been further intensified by NHS England’s (NHSE) recently announced plans to restrict access to hepatitis C treatments approved by NICE. This new decision will result in only 10,000 courses of treatment being funded in 2016-17, meaning NHSE now has the remit to restrict access to medicines already found to be cost-effective by NICE. Putting caps on NICE approved treatments has concerned patient groups and campaigners alike, with this shift in decision-making potentially fundamentally changing the role of NICE from being a decision making executive non-departmental public body to an advisory body to NHSE. In the future, NICE plans to play a more proactive role in ensuring the NHS uses the medicines recommended through tailoring the price and reimbursement model to the product type. Further improvements rest on the delayed AAR however the detail of this report remains to be seen as silo access funds continue to be shaped irrespectively of NICE.

Following their excellent contributions during the breakfast debate our speakers have kindly provided additional insights and analysis, which are captured in this pamphlet. These contributions consider the challenges facing the NHS, as well as looking more widely at the way we appraise medicines within England. For further information about Lexington Health or if you would like to receive details about our breakfast series, please contact

Joanne Bullen, Head of Lexington Health at joanne.bullen@lexcomm.co.uk or on 020 7025 2328.

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With the introduction of the new CDF, the expected publication of the AAR and the forthcoming renegotiation of the Pharmaceutical Price Regulation Scheme (PPRS), much is going to be at stake in relation to the UK public’s interests in medicines innovation and the biopharmaceutical industry during the next 12-18 months.

In contexts where state funded or other neo-public monopsonist purchasers are negotiating with private intellectual property right holders about the costs of and access to medicines and, indirectly at least, how much in future should be invested in high risk research there are always going to be tensions and controversies. It is often claimed that medicines costs are rising too fast, but in reality they have in most relatively prosperous nations been more or less stable as a proportion of total health spending for several decades. This is because as medicines lose the short term intellectual property protection needed to ensure ongoing private (and arguably public) sector research and development investment their prices drop, a factor which conventional HTA fails adequately to accommodate.

There is a greater future danger of failing to invest sufficiently in public and private biomedical research than there is of new treatments making health services such as the NHS ‘unaffordable’. Key hazards can be summarised as:

• The creators of the NHS wanted health for all. The pioneers of universal health care were not primarily motivated by a desire to see the UK’s health and social services spend significantly less as a proportion of GDP than equivalent social or private insurance based systems. But because the NHS is tax funded this has been the paradoxical result, as compared to the situation in many other OECD countries.

• Global life expectancy has risen by about 30 years since the 1950s. Medicines, vaccines and other pharmaceutical products have helped achieve this. In future, innovative biomedical technologies will not only continue to improve health outcomes in fields such as oncology and neurology but will also contribute to biologically based solutions to problems in areas such as energy and food production and environmental protection. However, progress will be delayed if the illusory pursuit of short term financial savings undermines awareness of the long term prizes humanity will win by investing adequately in the ongoing development of the biopharmaceutical (and regulatory) sciences.

• Population ageing has not been the main cause of increasing health and social care costs. It has rather brought social and economic benefits which politicians often fail to understand, and agencies such as the medical profession and pharmaceutical companies frequently fail to communicate.

The continuing health of people and the economies they inhabit will depend on spending enough on biopharmaceutical/genetics based innovation for the future. Nobody wants to waste public or indeed private money. Yet although the dangers of spending too much on ‘high science’ products as opposed to every-day consumer goods are widely perceived, the more serious dangers of investing too little on what is fundamentally important can be difficult to understand.

Professor David Taylor Emeritus Professor of Pharmaceutical and Public Health Policy, University College London

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Looking back at NICE I believe it’s fair to say that NICE has reformed and naturally, my prediction is that it will continue to do so. These reforms may be subtle but important; a review of the Board at NICE is either underway or about to be. Could we see a patient representative sat around that illustrious table to join GPs, Consultants, a Nurse, an Auditor, academics, businessmen and a civil servant? Could we see re-shaped agendas for the Appraisal Committee meetings, starting first with the patient perspective, before adding in the clinical and then the economic?

The more realistic changes might be that we really see a shift towards conditional access. Those ideas are already out there in the CDF consultation but it seems to me that this is not a cancer issue, but one that makes much sense for any area where we face uncertainty yet there is reason to believe that a new medicine will bring a positive risk, benefit balance and offer value for money in the fullness of time. The attractiveness of the concept lies in its ability to allow some access - better than none - but not unfettered access - not necessarily good for patients or for spend in the NHS, but good for companies. It also means that patients can get on with getting treatment and not have to lobby for access; leaving companies and Government to get on with working through uncertainty and a reasonable price.

But this idea is not new. It’s been around for some time, under different names such as coverage with evidence development. The tricky issues are many and varied; who pays and how much whilst there is uncertainty, who and how data is collected and for how long, who and how data is analysed and so what? Will price become conditional too? Perhaps one of the biggest issues is a shift in mind set. Looking back at NICE recommendations there has always been this ‘maybe’ style of recommendation – Only in Research. To quote NICE, this recommendation is when “The drug or treatment is recommended for use only in the context of a research study, for example a clinical trial. Often, particularly in the case of promising new technologies, sufficient clinical evidence has not been collected at the time of the appraisal and so the Appraisal Committee is unable to recommend the technology for use in the NHS until further evidence on its effectiveness is available for re-appraisal. The Committee will take into account whether there is a reasonable prospect of the technology being cost effective and that the research will inform future NICE guidance, whether the research can realistically be set up or is already planned or in progress, and whether the broad balance of the benefits and costs of conducting the research is favourable.

In these cases NICE will recommend further research to investigate whether the promise of the technology can be realised. In the meantime the NHS should only use the new treatment as part of a well-designed programme of research intended to answer these questions.” But it’s been used only 26 times since NICE’s work began in 2000, that’s 4% of Technology Appraisal recommendations. Although it’s only an assertion on my part, I suspect that perhaps it might have been a useful approach in more.

Leela Barham Independent Health Economist and Policy Expert

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The NHS has always been a slow adopter of new medicines. Given the current parlous financial state of the NHS, that is not going to improve any time soon as the NHS share of GDP looks like returning to its pre Labour Government levels.

Rt Hon Lord Hunt of King’s Heath Labour Spokesman on Health

I was the Minister in charge when NICE was created to speed up the introduction of clinically and cost effective treatment and drugs. We also legislated for technology appraisals to be implemented by the NHS locally. But increasingly, the NHS is using devices nationally and locally to restrict this.

We only need to look at the CDF to see what is happening. Once it got over budget, NHSE essentially set up a new rationing tool to exclude new patients from accessing drugs, which had originally been agreed through the CDF.

The PPRS was another promising step for the NHS and the pharmaceutical industry to incentivise the use of new drugs. But, it has so far proved to be a wasted opportunity. Rebates have been made but do not seem to have reached the front line. And the NHS continues to pare down on the use of medicines. NHSE do not seem to have bought into the idea and without their support the scheme did not take off as the industry would have hoped.

On a more positive note, the interim outcomes of the AAR seem to be promising and tribute should be paid to George Freeman and Sir Hugh Taylor. However, it is questionable whether NICE will be able to deal with the pressures put on them when the AAR report is published later in the year. Coupled with the extremely difficult financial problems facing the NHS, my fear is that it will work for a few selected medicines but not help the speed up of adoption more generally.

The UK continues to offer many advantages to the pharma industry but its future success cannot be divorced from the need for the NHS to be properly funded. It’s a debate in which industry may well need to join in.

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The ABPI continues to work with our members, the Government, NICE, NHSE and key stakeholders to ensure that the full value of medicines is understood and assessed appropriately so that patients can access the medicines they need when they need them. The Department of Health and the ABPI made a joint investment in the development of a common database for new medicines, UK Pharmascan, some time ago which is being used by NHS horizon scanning organisations to support better financial and service planning for the introduction of medicines into the NHS. It is likely that the AAR will build further on these concepts for all significant innovations.

We need to continue to have conversations about how we can predict important innovations coming down the line. ‘Breakthroughs’, ‘high-impact innovations’, ‘high unmet need’, ‘significant clinical promise’, are terms which all boil down to the need for us to be better at picking up these signals from the regulatory system and act on them earlier. The new Hepatitis C treatments being a great case in point. We need to have earlier conversations to better plan for new innovations so that patients can benefit from them.

The evidence base for new medicines is changing. We need to get better at managing risk where medicines do have an immature evidence base. The plans for the new CDF begin to address this, but we haven’t got it right yet. This means there is a risk of significantly setting back patient access to cancer medicines, now and for the foreseeable future. If cancer medicines go through more or less exactly the same NICE appraisal process that was in place five years ago - which necessitated the setting up of the CDF in the first place - we will largely get the same answers as before - the majority of medicines will be turned down. Turning the clock back five years just doesn’t make sense; more substantial change is needed to the way that NICE appraises cancer medicines to prevent this.

Dr Paul Catchpole Value and Access Director, Association of the British Pharmaceutical Industry (ABPI)

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Facilitating timely patient access to clinically and cost-effective medicines and medical technologies is the key objective of NICE’s health technology evaluation activities. Regulatory developments and initiatives such as the AAR and Early Access to Medicines Scheme (EAMS) and broader European initiatives such as the European Medicines Agency (EMA) adaptive pathways pilots are strongly influencing developments at NICE. These developments mean that products addressing unmet medical need are likely to come to NICE sooner in their development cycle with less evidence.

A key challenge is to work with partner and stakeholder organisations to find a way to provide patients access to promising new treatments while the evidence is still emerging, in a financially sustainable way. Developments are likely to include the wider use of managed access agreements together with agreeing plans for continued evidence development beyond the initial marketing authorisation. The recent consultation document on reforms to the CDF addresses several of these issues and includes mechanisms for handling situations where high potential is combined with immature evidence – the CDF essentially becomes a managed access fund for cancer medicines.

In parallel with developments to policies and methods, NICE is actively engaged in related research such that we are well positioned for the adoption of robust strategies to support timely patient access. NICE is leading work packages within major European Innovative Medicines Initiative projects on real world evidence (GetReal project) and adaptive pathways (ADAPT-SMART project) as well as actively contributing to the EMA adaptive pathways pilots.

In the rapidly changing market access landscape, opportunities for life sciences companies to engage with NICE are important. NICE Scientific Advice has been providing advice related to prospective clinical studies for the past six years and this service has expanded considerably in recent times. Parallel advice with regulators (Medicines and Healthcare products Regulatory Agency (MHRA) and EMA) is also available. NICE has also opened its Office for Market Access recognising the need for dialogue over a broad range of market access related issues. Enquiries to date have covered diverse product types (biologics, cell therapies, small molecule pharmaceuticals, medical devices, diagnostics, digital health) and many issues. Early discussion of potential managed access arrangements have been included in a number of company discussions. Recognising that in some cases, meaningful discussion of market access issues requires input from multiple agencies and stakeholders, the office is also establishing facilities for early multi-stakeholder “safe harbour” discussions.

Dr Nick Crabb Programme Director - Scientific Affairs, NICE

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Biography: Leela Barham Independent Health Economist and Policy Expert

Biography: Rt Hon Lord Hunt of King’s Heath, Labour Spokesman on Health

• David Taylor is Professor Emeritus of Pharmaceutical and Public Health Policy, University College London.

• David’s current work includes studies on public health and pharmaceutical policy formation and the future development of pharmacy, the pharmaceutical industry and globally accessible health care provision.

• Leela is an independent health economist and policy expert who has worked on health policy for over 10 years, after qualifying with an MSc in Health Economics from York University and BSc Economics from Nottingham University.

• Leela is also a freelance writer, and has written for the IMS Health publication Pharma Pricing and Reimbursement, FirstWord, PharmaTimes, EyeforPharma and Pharmaceutical Executive.

• Raised to the peerage in 1997, Lord Hunt has had a long interest in health, previously working as a health administrator.

• He was the Director for the National Association of Health Authorities and Trusts, and spent a year as the Chief Executive of the NHS Confederation. He became President of the Royal Society for Public Health in 2010.

• Paul Catchpole is Value & Access Director at the ABPI working across an extensive industry-wide portfolio covering the PPRS, evaluation of medicines, pricing and reimbursement, health technology assessment, the CDF, biosimilar medicines, specialised commissioning and market access.

• Paul has worked in the healthcare, local government and pharmaceutical sectors for over 30 years.

• Nick had a 20 year career in analytical science, process technology and general management in the chemical, pharmaceutical and contract laboratory industries prior to joining NICE as the associate director for the Diagnostics Assessment Programme in 2010.

• Nick was responsible for the establishment and management of the programme. In 2014 Nick was appointed Programme Director for Scientific Affairs where he oversees NICE Scientific Advice, the Science Policy and Research programme and the NICE Office for Market Access.

Biography: Professor David Taylor Professor Emeritus Professor of Pharmaceutical and Public Health Policy, University College London

Biography: Dr Paul Catchpole Value and Access Director, Association of the British Pharmaceutical Industry (ABPI)

Biography: Dr Nick Crabb Programme Director, Scientific Affairs, The National Institute for Care and Health Excellence (NICE)