The Road to Early Access:Latest developments from Empower Spring 2015

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Introduction

In November 2014 Empower: Access to Medicine brought together the Medicines and Healthcare products Regulatory Agency (MHRA), Department of Health, charities, politicians, leading academics and campaigners for a discussion led by the President of the Royal Society of Medicine, Babulal Sethia, and Chairman of GW Pharmaceuticals, Geoffrey Guy.

The meeting received an update from Daniel O’Connor, MHRA, on the progress of the Early Access to Medicines Scheme, and featured a wide ranging discussion exploring the barriers to earlier access, financial hurdles in drug development, gaps in the relationship between health bodies, and the role of the patient voice.

This latest report from Empower: Access to Medicine is based on many of the key discussion points from the November

meeting; and our plans for the future.

The Road to Early Access • Latest developments from Empower

The Early Access to Medicines Scheme – initial progress

Launched with much fanfare in the spring of 2014, the Early Access to Medicines Scheme (EAMS) was the culmination of a drive by Government, industry and patients to find a faster pathway for innovative medicines to those who needed them.

However, many months have passed since the scheme went live, and there is still an unclear picture regarding the take up of EAMS and its role as an enabler for future change.

Hearing from experts in industry and regulation, those gathered met reports of the initial applications of the Promising Innovative Medicine designation (PIM) with mixed emotions. As of late November 2014, some seven months after the scheme went live, only two PIM designations had been given (there are now 3).

At this stage, only one of the two designates, an oncology drug, had published their receiving of the PIM designation; the other had not (as is their right).

The fact that two medicines had received such a designation was clearly welcomed by those present at the RSM gathering; that an oncology treatment might be better expedited to patients was surely a good thing. What was more of a concern was that only two products had been approved at this stage; and even more worryingly that there had been an underwhelming number of applications.

Whilst it was highlighted that the Early Access to Medicine Scheme was indeed a faster process than conventional methods (as the treatments are not licenced), it was also made clear that there remained a strong case for strict oversight and safety checks before administering the drug to patients.

Explaining the low number of applications was less straightforward. Indeed it was emphasised that when the USA’s Food and Drug Administration (FDA) created the Breakthrough drug designation, a system EAMS was to some extent designed to emulate, the agency was swamped with applications.

The lack of applicants, compared with the US, was surprising for a number of reasons.

Firstly the number of disease areas that claim to have promising treatments waiting for an opportunity to get their drugs to patients created the expectation that more companies may have come forward for the scheme.

Secondly though the scheme is largely intended to accelerate medicines through Phase III (and generally final) clinical trialling, it can, in certain circumstances, be applied at Phase II – a move Empower: Access to Medicine has been driving at for a number of years.

The scheme certainly presents a clear opportunity to accelerate drugs to patients, and it is indeed early days. However, slow progress is a luxury few patients can afford.

The Early Access to Medicines Scheme (EAMS)

The scheme is voluntary and companies can put their product forward if they meet the MHRA criteria:

• The condition is life-threatening or seriously debilitating and there is a high unmet need

• The product is likely to offer major advantage over methods currently in use in the UK

• Reasonable expectation of a positive benefit risk balance based on preliminary evidence

The scheme has two key stages with the regulator:

• Step I: MHRA considers whether to award a Promising Innovative Medicine (PIM) designation based on early data

• Step II: An EAMS scientific opinion to describe the benefits and risks and if positive, a Public Assessment Report (PAR) and an EAMS treatment protocol to support prescribing decisions before licensing. A risk management plan will be required and opinion holders will collect patient data whilst the EAMS is in operation, to ensure the safe and effective use of the product

Companies pay fees to the MHRA for both Step I and Step II of £4,027 for applying for a PIM, £29,000 for the scientific opinion. They must also collect real world evidence to inform the future reimbursement decisions.

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The Road to Early Access • Latest developments from Empower

Cultural and Cost Barriers

It was commented on that doctors previously had greater scope to apply innovative treatments without having had to wait for a raft of approval processes further up the drug development cycle.

Instead of so heavily relying on ‘laboratory data,’ a doctor was required to report the new findings of medicine very early on. But over the past 30 years the burden of proof required to get something through has increased exponentially.

Nearly half of Phase III trials now fail; not necessarily because the drug does not work, but often because the trial has ‘failed’ from an empirical point of view. Meaning there now exists a situation where big pharma (let alone the smaller biotechs) cannot afford the money to see medicines through the long process.

It was highlighted that the remaining spectre of the Thalidomide scandal casts a long shadow over the sector and a general decline in the use of “named patient prescribing” continued to act as a break on innovation.

The recent debates on the Medical Innovation Bill in Parliament (instigated by Lord Saatchi) had served to shine a

spotlight on many of these issues.

Whole System Approach

Despite recent progress, there remains a serious ‘disconnect’ between the many health bodies that have a say along the road to drug approval. It was emphasised that too often in discussions, regarding how patients could be better served by the system, the people who ‘hold the chequebook’ are not present – i.e. NHS England.

There also exists a contrast between the UK and USA context in how medicines are pursued. Patient groups now play a much expanded role. Indeed it is often recognised that patient groups understand their respective conditions better than anyone. Therefore the FDA are obliged to involve patient groups from the start of the approval process – the UK does this at a pan-European level which can make it harder to engage with.

It was suggested that part of the problem in the UK was that the patient community often struggled to proactively engage early on in the development process, only coming to the table once a negative or positive decision on a treatment had already been reached.

This issue highlighted a wider communication problem - medical bodies need to communicate better with each other and proactively engage patients early on; and patient groups need to explore effective channels to engage the health bodies collectively on a disease area.

It was agreed by all that the NHS was in a uniquely strong position to implement and integrated approach to data collection. The National Institute for Health Research, the new Academic Health Science Networks, the Health Research Authority, the MHRA and the national oversight afforded by the NHS, present the opportunity to collate innovative medicine data nationally, and disseminate it to those who need it. Yet, there is currently no register.

The Early Access to Medicines Scheme, though unique to the UK, could provide the prospect of a test bed of innovation. But it is currently unclear if any of the innovative effects of the treatments that survive the PIM process are held centrally for the wider patient community to benefit from. If there are plans to do so, they are yet to be announced.

Way Forward

Empower will now drive to ensure that the gap in communication between the relevant health bodies is identified, and the avenues for a more appropriate forum to bring the approvers, payers and users together.

Furthermore Empower will seek to bring the question of a patient’s right to access back to the core of the argument. Recent consultations on the NHS Constitution have paid lip service to patients’ rights – but to what practical outcome?

The Medical Innovation Bill may further the rights of doctors to innovate more effectively in the hospital context, but in the emerging world of personalised medicine denying a patient’s right to bespoke access is unsustainable.

America’s Right to Try (a movement to give patients the right to try more experimental medicines) drive to change state-level legislation speaks to the heart of Les Halpin’s original mission, as enshrined in the Halpin Protocol. Real legislative change in this country may yet be the best course for patients.

Attendees

President of the Royal Society of Medicine Babulal Sethia

Geoffrey Guy, GW Pharmaceuticals

Daniel O’Connor, Medicines and Healthcare products Regulatory Agency

Richard Carter, Department of Health

Aisling Burnand, Association of Medical Research Charities

Emily Crossley, Duchene’s Children’s Trust

Alex Johnson, Joining Jack

Baroness Masham

Baroness O’Neil

Richard Barker, Centre for the Advancement of Sustainable Medical Innovation

Elin Haf-Davies

Peter Feldschreiber

Leela Barham

The Road to Early Access: Latest developments from Empower

Pamela Learmonth, BioIndustry Association

Jeremy King, Obillex

Jack Scannell, Centre for the Advancement of Sustainable Medical Innovation

Martin Smith, NHS

Alastair Kent, Genetic Alliance UK

Dr Hollie Chandler, Cancer Research UK

Caroline Brocklehurst, Teenage Cancer Trust

Elin Haf-Davies

Bina Rawal, Association of the British Pharmaceutical Industry

Jennifer Bryant-Pearson, Empower: Access to Medicine

James Hargrave, Empower: Access to Medicine