From lab to bedside: ensuring new medicines are available in a cost-effective and timely way

David Omojomolo writing at The King’s Fund: At a challenging time for the NHS in terms of finances, demand for services and patient expectations, policy-makers need to ensure patients have access to new medicines in a cost-effective and timely way.

Much of recent investment from the pharmaceutical industry has been in personalised medicines, using advances in the understanding of genetics to tailor medicines to individual patients. Individualisation does come at a cost, with associated larger costs for research and development, manufacturing and treatment dispensing. These high costs per patient may be unaffordable in the context of an economic evaluator like NICE, even if outcomes are better than best practice. For example Kymriah, a CAR-T cell therapy, has the potential to extend the lives of even the sickest blood cancer patients but at an estimated cost of $475,000 per patient.

Medicines

Other new medicines may offer affordable treatment on a per-patient basis but have such large potential patient populations that the total cost is deemed unaffordable, this time by the payer NHS England. In 2015 NICE deemed Sofosbuvir (a treatment for hepatitis C) cost effective and recommended several indications/treatments for routine use. NHS England was alarmed by projected treatment costs of £700 million annually – driven not by the cost of each individual treatment but by the large patient population. Through a protracted process NHS England capped availability to the sickest patients: its efforts were successful, restricting the bill to a fraction of projection, although medicine reached patients 10 months later than originally expected and some less serious cases are still ineligible for treatment.

In March 2017, NICE introduced the Budget Impact Test, which formalised the affordability checks NHS England wanted to apply to recommended medicines and applied a funding envelope. Medicines recommended by NICE need to have projected spending of less than £20 million in each of first three years they are available on the NHS to be automatically recommended. Manufacturers of medicines that exceed this have to enter into commercial negotiations with NHS England to manage affordability. If these are unsuccessful, NHS England can request NICE extends the time it has to make a cost-effective medicine available from the 90-day statutory requirement to up to three years from approval.

Apart from objections to perceived delays in making medicines available, there have been concerns behind the fairness of £20 million threshold. As the example of Sofosbuvir demonstrates, even products that are clinically and cost effective per patient can dent the threshold if there is a large population of eligible patients.

There was hostile reaction to the Budget Impact Test from the pharmaceutical industry, which brought a legal challenge on the grounds the test was incompatible with 90-day availability pledge, a challenge which ultimately failed. It is early days for the test so it is hard to measure success: to date only one drug scrutinised by NICE– Pembrolizumab (a lung cancer treatment) – has failed the Budget Impact Test and in the end price negotiations between NHS England and its manufacturer were fruitful enough for NHS England to deem it affordable for routine availability.

The Office for Life Science competitiveness indicators compares UK performance in a series of life science metrics against a range of comparator countries. The most recent indicators suggests the UK lags behind its peers in how quickly recommended medicines are made available, which is why many think streamlining the medicines access pathway should be a priority. The government has attempted to speed up access through a set of reforms recommended by the Accelerated Access Review report. One being the accelerated access pathway, which aims to bring breakthrough medicines to market and then to patients as quickly as possible.

Whether the scheme can actually improve outcomes is another question. The US Food and Drugs Administration has had a number of similar expedited review schemes for decades; however, evidence on the effectiveness and safety of medicines on these schemes compared to non-expedited medicines is mixed, with signs their introduction has incentivised manufacturers to weaken the evidence they submit to regulators to demonstrate clinical safety and efficacy for fast-track medicines. The accelerated access pathway needs to show coherence to differentiate itself in an already crowded marketplace of similar levers available to NHS England/NICE. Furthermore, it needs to ensure only medicines that clearly demonstrate innovative potential are chosen to be fast-tracked.

Recent policies from NHS England indicate that it has clear ideas about what should be routinely provided on the NHS. However, there is concern that its decisions are based on the wider funding issues in the NHS rather than on what NICE believes may be cost effective in the long term and that some NHS England decisions may be detrimental to patient access.

This is all happening as NHS England plans how increased funding settlement is spent. If money can be earmarked to transform medicine spending, the current affordability and access trade-offs NICE and NHS England are implementing for new medicines may be temporary and perhaps NICE guidance can be more strictly adhered to.

What does the future hold for integrated care systems?

Chris Ham writing for The King’s Fund: NHS England’s belated decision to change the name of accountable care systems to integrated care systems has the virtue of describing more accurately the work being done in the 10 areas of England operating in this way. Despite this, there is understandable confusion in many quarters about NHS England’s plans, and uncertainty about what they mean for the NHS in the medium term.

Integrated care systems, as they were re-branded in updated planning guidance published recently by NHS England, bring together NHS commissioners, providers and local authorities to plan how to improve health and care for the populations they serve. For the organisations involved in these systems, lines of accountability remain unchanged. NHS organisations continue to look upwards to NHS England and NHS Improvement and local authorities look outwards to their communities. Many systems are also working to establish a form of mutual accountability in which the organisations involved hold each other to account for the delivery of their plans.

Mutual accountability hinges on the existence of a high degree of trust and respect between organisations and their leaders. It also requires the establishment of governance arrangements that support collaboration while respecting the statutory responsibilities of these organisations. Integrated care systems have no basis in law and they depend on the willingness of the organisations involved to think and act as part of a wider system even when it may not be in their interests to do so.

Integrated Care

These systems also require NHS England and NHS Improvement to work together to oversee how areas working in this way are performing. This is beginning to happen in some areas through joint working by teams drawn from national bodies and the development of single regulatory oversight. NHS England’s expectation is that integrated care systems will become self-governing as they mature and that regulatory intervention will then be the exception rather than the rule, but this is some way off in most areas.

The updated planning guidance makes clear that integrated systems will become increasingly important in planning services and managing resources in future.

The updated planning guidance makes clear that integrated systems will become increasingly important in planning services and managing resources in future. The 10 areas already operating in this way will prepare a single system operating plan and take responsibility for a system control in 2018/19. The guidance also states that other systems will join the programme where they can demonstrate strong leadership, a track record of delivery, strong financial management, a coherent and defined population, and compelling plans to integrate care. The message could not be clearer: system working is here to stay.

Integrated care systems, like the sustainability and transformation partnerships from which they have evolved, are conscious workarounds which seek to make sense of the complex and fragmented organisational arrangements resulting from the Health and Social Care Act 2012. Changes to the law to regularise what is happening, included in the Conservative Party’s 2017 election manifesto, are off the agenda for the time being. This is because the government lacks a working majority and also because Brexit is dominating the parliamentary timetable.

The difficulty this creates is that workarounds are inherently unstable, even in the most favourable circumstances, and can only be sustained for so long. Informal mechanisms such as memoranda of understanding and partnership boards to underpin decisions about the use of NHS resources have a part to play but may break down when difficult decisions arise. There are worries too that decisions will be taken behind closed doors in forums that have no statutory basis and are not open to public scrutiny.

There are worries that decisions will be taken behind closed doors in forums that have no statutory basis and are not open to public scrutiny.

Changing the name of accountable care systems to integrated care systems will not distract attention from concerns by campaigners about a proposed national contract for accountable care organisations which they fear will result in ‘backdoor privatisation’. The contract is intended to be used by NHS commissioners who wish to undertake a competitive procurement process to integrate the delivery of care for their populations. NHS England has responded to the concerns raised by delaying the contract’s introduction and agreeing to consult on how it expects it to be used.

The puzzle is why the proposed contract is needed when many areas of England have already made moves to integrate care making use of existing legislative flexibilities. These areas are working with NHS providers to align hospital, community and mental health services, with GPs and adult social care services also involved in some places. They are doing so in a variety of ways, including through the use of alliance contracts and lead providers who subcontract with other providers to join up care in public sector partnerships..

Where this is happening, the emphasis on collaboration between commissioners and providers appears to run counter to the intention that the proposed contract should be used in a competitive procurement process. Clinical commissioning groups are also often uncertain on when they are required to go out to tender for the services they commission and are alert to the possibility of legal challenges from private companies who may feel excluded from decisions to keep contracts within the NHS family. The NHS market may be dying but is not yet dead, underlining the tension at the heart of recent developments.

For the foreseeable future, the NHS and its partners will have to live with this tension and the ambiguity it creates. They will also hope that the political will can be found before too long to align the law with the priority now being given to integrated care and partnership working. Is it too much to expect a cross party consensus to emerge that will make this possible?

Source: The King’s Fund