1. The data landscape is fragmented

The NHS is seen by many as a single, national organisation – but in practice this is not the case for health data.

Health data is collected by a large number of different organisations, including over 200 legally separate NHS trusts across England, autonomous research organisations (including UK Biobank), government-owned companies (such as Genomics England), disease registries (collecting information on, for example, patients with cancer, heart disease and diabetes) and national-level bodies (such as, in England, NHS Digital).

The devolved nature of the UK health system complicates this picture further, with each devolved nation maintaining separate legal and data governance structures for their health systems. They can decide their own approaches to promoting their data and responding to requests from researchers, and the risk is that – as stated above – implementation of the OLS’s second guiding principle on health data exacerbates rather than alleviates this situation.

The following anecdotal examples illustrate this fragmentation:[53]

• The UK might have been able to take part in a real-world study in type 2 diabetes (sponsored by a global company), but the available datasets were much smaller than those accessible through US administrative claims databases, which were able to provide data on around 700,000 patients.
• The UK had the opportunity to take part in a multi-country real-world study in inflammatory bowel disease (also sponsored by a global company). However, lack of central data access across the NHS in England meant there was information on too few patients per centre for the UK to be readily included.

We recognise that important steps are being taken to address this fragmentation: for example, the UK Health Data Research Alliance was launched in February 2019 to bring together the many organisations in the UK which hold health data – including academic institutions, NHS England, NHS Scotland, NHS Wales, NHS Digital, Genomics England, Public Health England, CPRD and others [54] – and to bring about a consistent approach to data access for research.

However, membership of the Alliance does not require specific organisational commitments to data access, and it is not comprehensive: with 18 members as of December 2019, its membership does not include many relevant UK data custodians, including many disease registries and the majority of NHS organisations.[54] The first priority action area is to address fragmentation.

2. Processes to access data are inefficient

As Boxes 10 and 11 make clear, the biopharmaceutical industry encounters processes to access data in the UK which can be slow, bureaucratic and unpredictable, which require multiple applications and agreements, and which are guided by data controllers who are under pressure to be risk averse.

These processes can take months and sometimes years to respond to requests from researchers for health data (see Box 10), frustrating the development both of new treatments and new technologies (see Box 11). Slow and inefficient processes also impede steps to improve the quality of the UK’s health data, since the longer the time between generating and accessing data (known as ‘latency’), the more difficult it is to make corrections while the clinical team is able to recall the case.

Where processes are more efficient elsewhere in the world, some elements of global new medicine development will be done there rather than in the UK. However, significant improvement could ensure that the UK can attract more R&D investment.

We hope that the Government’s plans to create a new ‘National Centre of Expertise’ in NHSX, to provide commercial and legal expertise to NHS organisations, and tools such as standard contracts and guidance, will help the NHS respond more efficiently to requests for data from researchers.[35] In addition, we will continue to support efforts to improve digital and data-handling skills in the NHS as recommended in Dr Eric Topol’s 2019 review for Health Education England.[55]

3. The NHS is not sufficiently digitised to allow data to be linked and accessed readily

A particular issue for clinicians and researchers seeking to work with UK health data is that much of the data is unlinked. For example, one global biopharmaceutical company seeking health data to support its cancer research found that the UK’s SACT and cancer registry datasets could not help because it was not possible to follow a patient between primary and secondary care.[61]

Although commitments have been made to a ‘paperless NHS’ in England – which would allow data on an individual patient to be linked seamlessly across care settings – the timetable for delivery has repeatedly slipped: the NHS in England was first challenged to ‘go paperless’ by 2018,[62] and then by 2020;[63] and the NHS Long Term Plan’s current target is 2024.[4]

We hope that, in time, the NHS’s digital infrastructure across the UK will be sufficiently mature to allow the easy use of health data for the purposes identified in Box 11 – for example, trial recruitment, demonstrating value, and understanding and stratifying disease. In relation to the former, this will – for example – create the conditions to allow further trials of the kind exemplified by the Salford Lung Study to be located in the UK. However, in order for the UK to secure such clinical trials investment, researchers must have the confidence that the UK’s digital infrastructure will be able to identify patients who might benefit from a treatment under development in near-real time, secure their consent for participation, enroll them into a trial, and report on results to the standards that traditional clinical trials would offer.

We recognise that the UK is taking important and positive steps to help digitise and link the UK’s health datasets, and achieve this goal:

NHS England is supporting ‘global digital exemplars (GDEs)’ in secondary care and ‘local health and care record exemplars (LHCREs)’ to join together health and care records to test the most efficient approaches to achieving its aim of a ‘paperless’ NHS.[4]

HDR UK’s ‘Sprint Exemplar Projects’ aim to test technologies and methodologies to enable the utilisation of linked datasets,[64] and include a project led by the University of Leicester to make patient datasets safely linkable and discoverable so that a complete patient profile can be readily located.[65]

Seven ‘Health Data Research Hubs’ were established in September 2019, and offer the prospect of creating rich disease-focused datasets that will enable new clinical trials and real-world evidence studies to be undertaken in the UK.[66]

Whilst we recognise the progress that each of these initiatives will deliver, it is important that timetables for delivery do not slip, and that new initiatives are not created on top of older initiatives, which would further fragment the health data landscape.

While industry continues to support the digitisation of the NHS, the issues of linkage and accessibility are addressed through the first two action areas. The third priority action area for industry is specifically to harness health data to support clinical development of new medicines:

4. The quality and accessibility of the UK’s health data resources is opaque

Researchers engaged in biopharmaceutical R&D frequently struggle to understand the quality and accessibility of the UK’s health data, encountering particular challenges in answering the following common questions:

• Consent: does the patient consent cover the intended use of the health data?
• Permissions: am I legally able to use the dataset in the way I intend?
• Cost/terms: what are the costs or terms of accessing the dataset?
• Time: how long will it take me to get permission and then practically access the dataset?

As a result, researchers look elsewhere in the world for health data to meet their research needs, rather than work in a UK health data environment where the discovery of relevant health datasets and information about their quality is a challenge (see Box 12).

We are nonetheless encouraged by a number of initiatives:

• In Scotland, the electronic Data Research and Innovation Service (eDRIS) offers an effective, single point of contact to assist researchers with their data access questions.[67]
• The Health Data Research Innovation Gateway has been established in January 2020, with the aim of allowing data from the Health Data Research Alliance members to be discovered quickly.[68]
• The new National Centre of Expertise being established in NHSX is due to set clear and robust standards on transparency and reporting.[35]

Therefore, the fourth priority action area is to make it easy for researchers to find out what datasets are available, and what the quality and utility of each dataset is.

5. Regulatory and reimbursement processes do not recognise the potential that developments in health data can offer

Many of the challenges that the biopharmaceutical industry encounters in securing approval of new medicines for use in healthcare systems – including the NHS – relate to the methods used by regulatory and reimbursement authorities to assess them. For example:

• The clinical endpoints required by regulatory authorities are naturally based upon historical experience in clinical trials of medicines with established measures of efficacy. 
  As innovation proceeds, new mechanisms with new measures and endpoints are discovered and novel designs for clinical trials are developed to assess these. The adoption of these new approaches in the regulatory process needs to be accelerated through early dialogue.
• NICE’s methods of evaluation rely mainly on the use of research findings in academic journals (or syntheses of these research findings), despite the growing possibility of using broader types of data – including data captured from the NHS during routine clinical care – to inform its assessments. This gap is itself recognised by NICE, which launched a consultation on making use of broader types of data in June 2019.[70]
• Current, international approaches to the regulation of clinical trials are based on standards agreed by the International Council for Harmonisation of Technical Requirements for Registration of Pharmaceuticals for Human Use (ICH) in 1995, before health data healthcare institutions became digitised.[13]

These characteristics pose particular challenges for the new categories of treatments which the industry is researching and developing. For example:

• The efficacy of a variety of new treatments for cancer may need to be tested on multiple tumour sites, leading to a need for novel trial designs (for example ‘basket’ or ‘umbrella’ trials).
• The need to address smaller patient populations due to biomarker-led stratification, personalised treatments or rare diseases also requires novel designs of clinical trials.

Clear demonstration of the long-term value of emerging curative technologies such as cell and gene therapies is challenging and may require new approaches including long-term follow-up.

We therefore hope to see that the advances being made in biopharmaceutical R&D and healthcare systems’ digital capabilities are accompanied by advances in the approaches taken by regulators and reimbursement authorities.

This will enable the use of data captured digitally, allowing the use of real-world evidence to support the approval and adoption of new treatments. As the Life Sciences Industrial Strategy made clear, this will improve the speed and efficiency of regulatory studies, increase the cost effectiveness of trials, and reduce the cost of developing medicines.[13]

The UK share of global biopharmaceutical R&D spending is, at 7%, relatively small [71] – but the UK has an excellent reputation for regulation and health economic assessment. The UK could lead the way in the development of a regulatory and reimbursement environment which harnesses the potential improvements that health data can deliver – as the MHRA and NICE are already considering.[72]

The rapid evolution of clinical trial designs and endpoints, coupled with the potential for health data to be accessed and analysed in near-real time, leads to the suggestion that regulatory and health technology assessment authorities will in future have a wider variety of data to consider – some of it eventually enabling more precise estimation of both the clinical and economic value of an intervention.

At the same time, novel treatments that promise to provide long-term remission or even cure after a single (or only a few) administrations will need specific programmes of long-term data collection, to demonstrate or confirm their value and perhaps to underpin new approaches to reimbursement (for example outcomes-based payments).

Therefore, industry’s fifth priority area is to work with the authorities to understand new types of health data, how they can be factored into evaluations, and how standards can be developed.