Top 5 Takeaways
- The UK wishes to ‘align as closely as possible’ to EU Regulation on medicines.
- There are several policies that will create a more positive prescribing environment in 2020.
- The MHRA could become the first choice regulatory authority for global pharma and biotech.
- The NHS Commercial Framework commits to making Commercial Access Agreements more widely available.
- The Medicines and Medical Devices Bill will ensure ‘faster access to innovative medicines’.
Will a raft of policies designed to create a more positive prescribing environment make a difference in 2020?
2019 was epitomised by two words ‘Brexit’ and ‘Uncertainty’ whilst the NHS and pharma had to deliver on ‘business as usual’, as well as work truly collaboratively to mitigate risk of a No Deal Brexit and avoid a potential national crisis of medicines shortages.
Every month the UK exports 47m packs of medicines and imports 35m packs of medicines, 90% of which leave and enter the country from Dover and Calais1. The Department of Health and Social Care (DHSC), pharma, biotech, and medtech collaborated in an unprecedented way to keep medicines flowing, including:
- building buffer stocks of prescription-only and pharmacy medicines in case of difficulties in shipping medicines using current transport routes2
- introducing arrangements for community pharmacists to dispense alternative equivalent medicine without redress to the patient’s GP in the event of an out of stock situation3
- procuring new cross channel ferry routes3,4.
The reality of the General Election result is that the Government got the majority it needed to get its Withdrawal Agreement through. By the time you read this, the UK will have ‘left’ the EU and be ‘in transition’, negotiating trade deals with the US, EU, Asia-PAC and implementing legislation to either UK-adopt or change the existing EU legislation it operates under.
“Moving from a world of uncertainty bundled up under the banner of Brexit, there is hope as we go forward that we can return to business as ‘unusual’ within the UK pharma and biotech market”
On medicines, clear statements have been made that the UK wishes to ‘align as closely as possible’ to EU Regulation. What does that mean in terms of keeping the UK as the third priority country in the world in which global pharma and biotech choose to launch medicines?
There is no doubt that Brexit was a catalyst for the Government to improve the environment for UK life sciences. The Life Sciences Sector Deal, with its commitment to increase the uptake of innovative medicines to the upper quartile of the Organisation for Economic Cooperation and Development (OECD) average by 2023, and its point to NHS England (NHSE) to improve its collaboration with industry, sent a strong message to DHSC and NHSE ahead of the renegotiation of the Pharmaceutical Pricing and Regulation Scheme – now the Voluntary Pricing Access Scheme (VPAS).
Government was genuinely concerned that a toxic combination of the historic low and slow uptake of medicines reputation and Brexit uncertainty would lead to global pharma reducing its investment in UK clinical research and delaying or not launching at all in the UK.
The positives for industry from the VPAS included:
- greater transparency of process for launch, culminating in the new NHSE Commercial Framework 5
- a commitment that the National Institute for Health and Care Excellence (NICE) would review its methodologies to ensure they are more aligned to the needs of complex specialised medicines and Advanced Therapy Medicinal Products than the traditional primary care blockbusters it was set up to review in 1999
- maintaining the cost-effectiveness threshold despite strong pressure from certain vociferous health economists to reduce it to £15,000.
Now there is a Government with a majority, industry can be certain about the implementation of several proposed policy initiatives during 2020, that are broadly positive for medicines access and uptake. The policies that will create a more positive prescribing environment in 2020 include:
- the Medicines and Healthcare products Regulatory Agency’s (MHRA) proposals to accelerate clinical trials and licensing approvals and be leaner and faster than the European Medicines Agency (EMA) and US Food and Drug Administration
- the Review of NICE Methodologies
- the implementation of the NHSE Commercial Framework
- Health Data Research Hubs
- expansion of the Accelerated Access Collaborative
- acceleration of the implementation of the NHS Long Term Plan
- the new Medicines and Medical Devices Bill6
Following the EMA leaving London for the Netherlands two years ago, the MHRA has made clear commitments to be not only open for business from Brexit, but importantly approving licensing within 150 days – and unlike the EMA it’s already taking account of real world studies in its considerations for license.
This could propel the MHRA to become the regulatory and licensing ‘Kitemark’ and the first-choice regulatory authority for global pharma and biotech. This would move the UK from being a ‘participatory’ country, as in the EMA days, to become a landmark registration country, running clinical research studies and not a small element of them as is the case today.
NICE Methods Review
NICE has now started the process of reviewing its methodologies to optimise its evaluation methods7. It has faced new challenges over the last five years and this major overhaul is designed to address issues, including:
- increasingly complex therapies being developed
- growing pipeline of cell and gene therapies e.g.: CAR-T and a UK view that they are gaining EMA approval based on limited evidence
- increasing levels of uncertainty, particularly regarding complex therapies and treatments for rare diseases, making it difficult for NICE to determine the true value of products, resulting in delayed access for patients.
Crucially, NICE is planning to increase its use of real world evidence in submissions, using broader sources of data including primary and secondary care databases, registries, surveys and national trend data. NICE is pace-setting amongst global Health Technology Appraisals (HTAs) in this respect.
The 2019 IQVIA White Paper, Orphan Medicines Launch Excellence8 evidenced that having real world data in a NICE submission increased the chances of a positive outcome for rare disease medicines by 20%. History has shown that where NICE leads, other countries’ HTA bodies follow. Having a real world strategy as part of a new medicines clinical research and HTA strategy is now a key launch differentiator.
NHS Commercial Framework
As part of the 2019 VPAS, it was agreed that NHSE would develop a Commercial Framework for branded medicines. The NHSE Commercial Framework9 was released in November 2019, with a consultation that closed on 10 January 2020. Key messages include:
- route maps in respect of how NHSE will implement additional commercial flexibility, and reiterate that simple patient access schemes (straight discount) remain the preferred option
- a commitment to make Commercial Access Agreements (CAAs) (as exist in the Cancer Drugs Fund (CDF), the Highly Specialised Technology (HST) programme, and the NICE budget impact test (BIT) available more widely – though there is limited detail on the circumstances that would warrant it
- at present, confidential complex CAAs are available within the context of the CDF and HST programmes, and products where spending exceeds NICE’s BIT. A key part of the Framework is a commitment to make these agreements available more widely where such flexibility is useful – again there is limited detail on what circumstances would warrant it
- other mechanisms that might be part of a negotiation such as a cap on the total budget, price/volume agreements, cost-sharing, or outcomes-based agreements
- quality-adjusted life years need to be closer to £20k than £30k
- NHSE Commercial Directorate’s message that companies should work with NICE’s Office for Market Access and/or NHSE as early as possible (ie during Phase III) is key and provides very clear guidance on how to effectively engage.
2020 will see more new medicines and devices added to the first round of the Accelerated Access Collaborative as the vehicle to implement the recommendations of the Accelerated Access Review (AAR). The AAR is working on developing innovative commissioning routes to accelerate transformative medicine and technology uptake at pace and scale in the NHS. To date, the medicines in the AAR include Merck’s Mavenclad for multiple sclerosis and Amgen and Sanofi’s PSCK9s.10
Health Data Research Hubs
A key differentiator for the UK on the competitively global clinical research stage is its uniquely rich health data ecosystem11 – the foundation for the UK to become a global leader in health data science. In 2020, the seven Health Data Research Hubs will be fully operational.12
- DATA-CAN – the national health data research hub
- Breathe – for respiratory health
- GutReaction – for irritable bowel disease
- DiscoverNOW – for real world research
- NHS DigiTrial – for clinical trials
- PIONEER – for acute care
- Insight – for eye health.
The Hubs are centres of excellence with expertise, tools, knowledge and ways of working to maximise the insights and innovations developed from the UK’s health data. As an example of how they will support industry, patients and academia, the DATA-CAN partners (UCLP, Genomics England, IQVIA, Leeds Teaching Hospitals, Queens University Hospital, Belfast) have set out to enable and expand UK-wide cancer dataset access for research.
DATA-CAN will provide high-quality real world data to support health and care transformation and use real world data to support patient enrolment in clinical trials, improve clinical trial design and reduce delays in start-up times through the IQVIA Oncology Data Network platform (ODN).
Crucially, DATA-CAN will be able to determine the real world impact of NICE approved cancer treatments, through the ODN platform, that can then be used to support evidence for new indications and budget impact discussions of the future. All the Hubs are seeking industry engagement and support, now they are through their initial start-up phase.
NHS Long Term Plan at local level
Accelerating the implementation of the NHS Long Term Plan at local level will see Integrated Care Systems (ICSs) bringing together local organisations in a pragmatic and practical way to deliver the ‘triple integration’ of primary and specialist care, physical and mental health services, and health with social care.
ICSs will work with local authorities at ‘place’ level, and through ICSs, commissioners will make shared decisions with providers on population health, service redesign and NHS Long Term Plan implementation.
Every ICS will need streamlined commissioning arrangements to enable a single set of commissioning decisions at system level. This will typically involve a single clinical commissioning group (CCG) for each ICS area. CCGs will become leaner, more strategic organisations that support providers to partner with local government and other community organisations on population health, service redesign and NHS Long Term Plan implementation.
The Medicines and Medical Devices Bill
The Medicines and Medical Devices Bill will ensure patients have ‘faster access to innovative medicines’ and support the growth of the UK life sciences sector. The Bill proposes cutting bureaucracy in clinical trials and licensing, making it simpler for NHS hospitals to manufacture and trial the most innovative new personalised and short life medicines, as their usage increases, and they are mainstreamed across the NHS.
The Bill also proposes increasing the range of professions able to prescribe and supply certain medicines to make the most effective use of the NHS workforce where recommended by experts, as well as developing more innovative ways of dispensing medicines in local pharmacies.
Hope on the horizon
Moving from a world of uncertainty bundled up under the banner of Brexit, there is hope as we go forward that we can return to business as ‘unusual’ within the UK pharma and biotech market. The collection and understanding of real world data sit at the heart of moving from a peri-Brexit fog with the promise of addressing some key pricing, funding and access issues that stand in the way of truly delivering medicines to patients that need them.
Angela McFarlane is Senior Market Development Director at IQVIA Go to www.iqvia.com