• Rachel Galloway

What have NICE and NHS England been up to in 2021?

Tell me it’s been a busy year for the UK healthcare sector without mentioning COVID-19 or Brexit…

Here goes. Attached below is a highlight reel noting some of the key changes and milestones for the National Institute for Health and Care Excellence (NICE) and NHS England and NHS Improvement (NHSE&I) in 2021 minus the above!

Post by Rachel Galloway, Consultant


January 2021: NICE launches the Innovative Licensing and Access Pathway

The Innovative Licensing and Access Pathway (ILAP) is intended to streamline and accelerate the innovative medicine development process and result in safe, early, and financially sustainable patient access to innovative medicines.

A key aspect of the ILAP is the collaboration between major stakeholders in the regulatory and health technology assessment landscape in the UK: the Medicines and Healthcare Products Regulatory Agency (MHRA), NICE, NHS England and NHS Improvement (NHSE&I), the Scottish Medicines Consortium (SMC) and the All Wales Therapeutics and Toxicology Centre (AWTTC).

This close collaboration and planning between agencies seeks to ensure optimal data is generated for both regulatory approval and health technology appraisals. However, advice and discussions are not restricted to clinical trial designs and may also cover expedited licensing and access routes, as well as commercial and managed access issues.

The key objective of these discussions is to help companies develop and submit well-evidenced value propositions, which should increase the likelihood that they gain a marketing authorisation from the MHRA and are recommended in NICE guidance. However, NICE will apply the same evaluation framework to ILAP versus non-ILAP medicines and make fully independent recommendations on whether products should be available to NHS patients.

To enter ILAP, companies must apply for Innovation Passport designation. Their application will be assessed against three criteria by the ILAP Steering Group:

  • Criterion 1: the condition is life-threatening or seriously debilitating, or there is a significant patient or public health need

  • Criterion 2: the medicine fulfills one or more of the following areas

  1. Innovative medicine such as an ATMP or new chemical or biological entity or novel drug-device combination

  2. The medicine is being developed in a clinically significant new indication

  3. Medicine (including repurposed medicines) for rare disease and/or other special populations such as neonates and children, the elderly and pregnant women

  4. Development aligns with the objectives for public health priorities of the Chief Medical Officer, DHSC and Life Sciences Sector Deal (including those in Devolved Administrations, where appropriate)

  • Criterion 3: the medicinal product has the potential to offer benefits to patients

The first Innovation Passport was granted to belzutifan, a treatment developed by MSD for adults with von Hippel Lindau disease, a rare genetic disorder that causes cancer. As of now, no products have completed the ILAP pathway so this is a space to watch for future learnings.


February 2021: NHS patients to benefit from first full access deal in Europe for a new CAR-T therapy

NICE agreed a managed access agreement, via the Cancer Drugs Fund, with Kite for Tecartus, an autologous anti-CD19-transduced CD3+ CAR-T therapy indicated for the treatment of those with relapsed or refractory mantle cell lymphoma, who have previously had a Bruton’s tyrosine kinase (BTK) inhibitor drug. In short, it’s a personalised therapy that re-engineers a patient’s white blood cells so they can recognise and attack cancer cells.

The managed access agreement allows NHS patients to have access to the treatment while:

  1. Kite collects more clinical evidence on Tecartus’ effect on progression-free survival, overall survival, and the age when treatment starts.

  2. Kite provides Tecartus to the NHS at a confidential discounted price.

It’s anticipated that ~100 patients each year could be treated with Tecartus and the therapy will be administered from ten critical care service providers around the country, which could require some patients to travel significant distances to receive treatment.

NICE’s recommendation makes Tecartus the second cell and gene therapy that Kite have successfully made available to eligible NHS patients and currently, it’s the only company to have achieved this.


March 2021: NHS achieves key Long Term Plan commitment to roll out integrated care systems across England

This month all areas in England were formally designated into 42 integrated care systems (ICSs), effective from April 1, hitting a major milestone in the NHS Long Term Plan.

ICSs will take the lead in planning and commissioning care for their populations by bringing together NHS providers and commissioners (including hospital, community and mental health trusts, GPs, other primary care services), local authorities and other organisations including the Voluntary, Community and Social Enterprise (VCSE) sectors to work in partnership in improving health and care in their area.

Key objectives of ICSs will be to improve the health of all residents, better support people living with multiple or long-term conditions, preventing illness, tackling variation in care, and delivering seamless services while making efficient use of NHS resources and funding.

While the planning is complete, current legislative barriers to integration across health and social care bodies need to be removed and NHSE&I has asked the Government and Parliament to establish ICSs in law.


March 2021: NHS England strikes a deal on Zolgensma

Another major news item in March was the announcement that Zolgensma, a one-off gene therapy that treats Spinal Muscular Atrophy (SMA), would be made available on the NHS after NHS England struck a deal with Novartis Gene Therapies.

The announcement made front page news because of the therapy’s clinical promise and its price tag:

  • The therapy offers the potential for babies and young children with SMA, who would otherwise face paralysis, muscle weakness, a progressive loss of movement and often death, to reach life-changing milestones such as breathing without a ventilator, sitting up on their own, crawling and walking.

  • Zolgensma is labelled the most expensive drug in the world and has a reported list price of £1.79 million per dose.

With SMA being a leading genetic cause of death among babies and young children, NHS England had a balance to strike in providing access to a transformative medical advancement while securing a price that is fair to taxpayers.

A single intravenous infusion of Zolgensma containing a replica of the missing SMN1 gene should restore the gene allowing production of proteins essential for nerve function and controlling muscle movement. Data from the Phase 3 STR1VE and Phase 1 START follow-up trials suggests Zolgensma can provide rapid and sustained improvement in motor function in patients and prolong their lives.

It’s estimated that ~ 80 babies and young children could potentially benefit from Zolgensma each year. They’ll receive treatment at designated centres of excellence that can provide the full range services required to administer the treatment safely. These services include paediatric intensive care, as well as neuromuscular, paediatric respiratory medicine, immunology, infectious diseases, hepatology, renal, cardiology and endocrinology clinical expertise. Currently the four specialist NHS centres commissioned to administer Zolgensma are Manchester University NHS Foundation Trust, Sheffield Children’s NHS Foundation Trust, University Hospitals Bristol and Weston NHS Foundation Trust and Evelina London Children’s Hospital (part of Guy’s and St Thomas’ NHS Foundation Trust).

On May 25 2021, the NHS administered Zolgensma to the first UK patient at Evelina London Children’s Hospital.


April 2021: NICE launches ambitious strategy to provide quicker access to new treatments and innovations

On Monday 19 April 2021 NICE unveiled its 5-year strategy. A core part of this strategy aims to adapt NICE’s approach to appraisals to ensure faster patient access to new and effective treatments while maintaining robust, trusted methods. This will in part be facilitated by more proactive and earlier engagement with the life science industry during the medicine development process. Additionally, the agency will seek to expand the organisation’s skills, capacity and capabilities by forging new partnerships.

Lastly, the new approach will allow NICE to produce modular recommendations that can be rapidly updated to incorporate the latest evidence as it becomes available.


April 2021: NICE signs up to join the GetReal Institute

Another announcement from NICE in April was its plan to become a founding member of the GetReal Institute. The GetReal institute’s core mission will be to facilitate the adoption and implementation of real-world evidence in health care decision-making.

The GetReal Institute will initially have three priority focus areas:

  1. Reducing barriers to using data that is generated in routine clinical practice in health care decision making, including addressing challenges with real-world data discoverability, quality, and accessibility.

  2. Bridging the gap between RWE and conventional randomised controlled trial (RCT) approaches. This could be through the incorporation of real-world data in RCTs or the use of real-world data-based external comparator arms in RCTs.

  3. Addressing the evidence needs of "downstream" decision-makers. This includes HTA bodies, payers, clinical guideline developers, clinicians, and patients whose perspectives and decision contexts are as important as those of regulatory agencies.

Other organisations that have expressed their intention to sign up as founding members of the GetReal institute include:

  • Clinical Practice Research Datalink (CPRD) (UK)

  • Eli Lilly and Company

  • Hoffmann-La Roche

  • GlaxoSmithKline

  • International Alliance of Patients’ Organizations (IAPO)

  • Janssen, Pharmaceutical Companies of Johnson & Johnson

  • Norwegian Medicines Agency (NoMA)

  • University Medical Center Utrecht (UMCU)

May 2021: Ambulance iPads improve patient care under NHS Long Term Plan

In May NHS Chief Executive Sir Simon Stevens announced that approximately 30,000 iPads will be issued to ambulance crews across England. The objective of this initiative is to ensure patients get the right care faster.

For example, ambulance crews will be able to send photographs from the scene of an accident to clinical staff in A&E departments so they will be better prepared and can begin to make decisions on what care needs to be given when a patient arrives at hospital and how they should organise care based on the severity of the injury. The iPads will also allow paramedics to video call consultants to obtain feedback on the best course of action until they reach hospital. Thirdly, paramedics will have access to vital health records helping them assess patients’ injuries, aid their clinical decision-making and inform decisions on whether a patient should be taken to hospital or treated at the scene.


July 2021: NHS England announces new Innovative Medicines Fund to fast-track promising new drugs

The Innovative Medicines Fund (IMF) will allow NHS patients to benefit from early access to potentially life-saving new medicines indicated for any condition (other than cancer) where further data is needed to support NICE in making final recommendations around their routine use in the NHS.

The IMF will ringfence £340m of NHS England funding annually - the same amount as the Cancer Drugs Fund – and allow patients to benefit from early access to the most promising and innovative treatments. It will operate in the same way as the Cancer Drugs Fund with NICE thereby giving manufacturers an alternative access route where there are challenges for NICE in making an immediate decision about routine availability on the NHS. (New cancer therapies with significant uncertainty around their clinical and cost-effectiveness will remain funded via the Cancer Drugs Fund).

It is anticipated that the IMF will become a commonly used option for gene-therapies that claim long-term or lifesaving benefits but lack sufficient long-term evidence at the point of marketing authorisation for NICE to make a final decision on their clinical and cost-effectiveness. Other scenarios where similar challenges may be faced are for innovative orphan drugs where again there are uncertainties simply because data collection takes longer to obtain due to the smaller patient cohort.


August 2021: NICE approves first treatment in a pilot of the new approach to cost-comparison fast-track appraisals

This summer NICE piloted a new approach to the cost-comparison fast-track appraisal process for the review of selected low-risk appraisals. The new approach involves using a subset of the appraisal committee to assess low-risk treatments that can be compared to similar therapies that have already been appraised by NICE. The sub-committee then makes a recommendation after which a full committee considers the guidance prior to its finalisation and publication. If need be, a full meeting can occur prior to this in case there are any concerns the committee need to discuss and resolve.

Bimekizumab, which is indicated for the treatment of severe plaque psoriasis, was the first therapy to go through this new process. The sub-committee determined that the clinical trial evidence showed bimekizumab to be more effective than three comparators recommended by NICE and that the cost-effectiveness estimates were in line with what is considered an acceptable use of NHS resources, so the new therapy was recommended.

This fast-track process should help NICE address some of the guidance backlog, appraisal priorities and capacity challenges created because of the impact of a-virus-that-shall-not-be-named.


September 2021: NHS patients are the first in Europe to be offered sotorasib

Lumykras (sotorasib) was fast-tracked to NHS patients after clinical trials demonstrated it stopped lung cancer growth for seven months. The therapy targets a mutation on the KRAS gene by binding to and inactivating it, thereby preventing cell division and cancer growth.

The treatment is anticipated to be a major breakthrough because the KRAS gene mutation is present in a quarter of all tumours. The research involved in developing sotorasib could represent a major step towards targeted therapies for many other deadly cancers including pancreatic and colorectal cancers.

~600 non-small cell lung cancer patients will have access to the drug each year via an early-access deal in which Amgen will facilitate quick access to the drug on a budget-neutral basis to the NHS.


October 2021: NHS strikes a deal on the first sickle cell treatment for over 20 years

Adakveo® (crizanlizumab-tmca) is now available to patients in England with sickle cell disease to prevent episodes of pain and other complications.

The treatment will be made available to 5,000 people over the next three years via a managed access agreement that requires the manufacturer, Novartis, to provide the therapy to the NHS at a confidential discount and collect further data on treatment outcomes.

The drug is anticipated to improve patient QoL by reducing the number of sickle cell crises experienced, which may also reduce the number of A&E visits needed by the patient. (Sickle cell crises often require hospital admission to control the pain and prevent potentially fatal organ failure).


November 2021: the NHS opens the first specialist clinics for severely obese children

A pilot scheme involving 15 new specialist clinics will allow severely obese children (ages 2-18) in England to access weight-loss support programmes, involving diet plans, mental health treatment and coaching. Support will be provided from a range of medical disciplines including dietitians, psychologists, specialist nurses, social workers, youth workers and paediatricians.

The aim of the programme is to prevent long term health problems associated with obesity such as Type 2 diabetes, heart attacks, strokes and cancer, while also trying to uncover the common factors causing obesity in children.

The scale of the childhood obesity problem has grown dramatically over the last couple of decades and isn’t improving – in fact the problem is thought to has worsened during the last year and half with lockdowns and restrictions leaving many young people struggling with weight gain.

The latest data indicates that a fifth of children leaving primary school in England are obese and that children living in the most deprived areas were twice as likely to be obese compared to those living in the least deprived areas.


November 2021: NHS strikes a deal on a third SMA treatment

The commercial deal struck between NHS England and Roche for Risdiplam means there are now treatment options accessible to patients with SMA types 1, 2 and 3 in England.

Risdiplam is the first non-injectable treatment for patients with SMA, which works by modifying the SMN2 gene to increase the amount of SMN protein produced. Unlike Zolgensma, a potential one-off gene therapy treatment, Risdiplam needs to be taken once a day but can be taken at home and provides a treatment option for patients for whom Zolgensma or Spinraza may not be suitable.

Several hundred patients with SMA in England, who were not able to receive treatment with other SMA treatments, have already received treatment with Risdiplan via an early access to medicines scheme. The scheme, which commenced in September 2020, was funded by Roche and allowed patients to receive treatment with Risdiplan prior to the product receiving marketing authorisation from the Medicines and Healthcare products Regulatory Agency (MHRA).


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