Managed access enables patients to access promising new treatments.
Otherwise, these treatments might not be recommended due to uncertainty about their value for money.
During managed access, more evidence is collected to address the uncertainties using a managed access agreement.
NICE then uses this evidence to recommend whether the treatment should routinely be made available on the NHS.
When we make a recommendation with managed access, NHS England and your company develop a managed access agreement.
This is a time-limited agreement which sets out:
- the conditions under which patients will be able to receive NHS-funded treatment
- how data will be collected to address the uncertainties.
Managed access agreements consist of two elements:
Data Collection Agreement (DCA)
In collaboration with stakeholders, we coordinate the development of the data collection agreement. This sets out:
- roles and responsibilities of all stakeholders
- the patient population that will be eligible for treatment
- types of clinical assessments and data to be collected and analysed.
A data collection agreement will last for the shortest period of time needed to collect enough data to address any uncertainties, up to a maximum of 5 years.
Commercial Access Agreement (CAA)
The commercial access agreement is an agreement between NHS England and your company. It lays out the commercial terms on which the NHS will fund the treatment.
The agreement also ensures that, during the period of managed access, the identified uncertainty is mitigated against.
As evidence is collected, managed access oversight groups meet regularly to check the quality and quantity of the data.
Membership of oversight groups is decided on a case-by-case basis, depending on the extent of data collection in clinical practice.
Members may include:
- NICE and NHSE colleagues, clinicians and clinical experts
- representatives from patient organisations
- representatives from the pharmaceutical company that manufactures the treatment
- members of NHS data custodians that collect and analyse data as part of the managed access agreement.
During these regular oversight group meetings, members discuss:
- the progress of data collection
- plans for analysis
- any issues that may arise during clinical assessment
- any safety issues signalled by patients and clinicians.
Evidence is collected on the treatment until the end date specified within the managed access agreement. After this we will update our guidance and decide whether to recommend the treatment for routine use in the NHS.
If the evidence shows that the treatment is not a cost effective use of NHS resource, it will no longer be recommended.
Without managed access, NICE might not be able to recommend patients have access to these promising new treatments at that time. Managed access provides a vital alternative route for patients to access these treatments.
Recommendations with managed access:
- Provide patients with faster access to promising new treatments.
- Help to ensure value for money for taxpayers.
- Offer pharmaceutical companies a route to NHS funding for treatments that are plausibly cost effective.
- Allow us to use the evidence gathered to update our guidance.
NHS England has 2 dedicated sources of funding which pay for the treatments in managed access:
Each fund has a budget of £340m per year.
Your company can submit a proposal for managed access as part of its submission to NICE.
A data collection proposal details the key uncertainties that your company considers could prevent the committee from making a recommendation for use in the NHS. It also details the data that could be collected to sufficiently support the case for recommendation after a period of managed access.
We will assess the feasibility of the data collection proposals before the committee meets to make its decision. The assessment process involves engagement with a range of stakeholders, including:
- your company
- patients and their representatives
- NHS data custodians.
The extent of engagement activities will be proportionate to the complexity of the data collection proposal.
The committee will first consider whether the treatment could be made routinely available on the NHS. A managed access recommendation will only be considered if it's uncertain whether the treatment will be cost effective.
If a committee has been unable to recommend a medicine due to uncertainties that could be resolved, it can make a recommendation with managed access.
To consider a managed access recommendation, a committee will need:
- a managed access proposal
- a feasibility assessment from NICE.
The committee can consider a recommendation with managed access when:
- The medicine has not been recommended for use.
- It has the plausible potential to be cost effective at the currently agreed price, but the evidence is currently too uncertain.
- New evidence that could support the case for recommendation is expected from ongoing clinical trials. Or evidence could be collected from patients having the medicine in clinical practice.
- These data could be collected within a reasonable timeframe (up to a maximum of 5 years) without undue burden.
We evaluate how clinically-effective and cost effective those drugs are, compared to currently available treatments.
We only evaluate drugs that have a MHRA or EMA marketing authorisation or are expected to receive one during our evaluation process. We only issue final guidance on drugs that have marketing authorisation.
Managed access means patients can access clinically promising drugs before we have made a final decision on whether they are a cost effective use of NHS resources.
However, clinicians will only prescribe them if they are safe to use and clinically appropriate for the individual.
The evaluation process provides many opportunities for NICE, your company and other stakeholders to identify if a medicine may be suitable for managed access.
- At scoping - the decision-problem stage where your company is considering making a managed access proposal.
- At the submission stage - a managed access proposal as part of your company's submission.
- At technical engagement (if held), which is when significant uncertainties are highlighted, and a managed access proposal could be submitted.
Before an evaluation has started, NICE offer several opportunities to work with companies to explore whether to make a managed access proposal.
Early discussions about managed access issues primarily happen through our Office for Market Access. They can also take place during the Innovative Licensing and Access Pathway (ILAP). NICE Scientific Advice can also provide input. These services are independent of our guidance producing programmes.
Technologies in the Cancer Drugs Fund (CDF)
- TA505 Ixazomib citrate for treating relapsed or refractory multiple myeloma
- TA529 Crizotinib for treating ROS1-positive advanced non-small-cell lung cancer
- TA540 Pembrolizumab for treating relapsed or refractory classical Hodgkin's lymphoma
- TA554 Tisagenlecleucel-T for previously treated B-cell acute lymphoblastic leukaemia in people aged 3 to 21 at initial diagnosis
- TA559 Axicabtagene ciloleucel for treating diffuse large B-cell lymphoma, mediastinal B-cell lymphoma and follicular lymphoma
- TA567 Tisagenlecleucel-T for treating diffuse large B-cell lymphoma after 2 therapies
- TA573 Daratumumab in combination with bortezomib for treating relapsed or refractory multiple myeloma
- TA576 Fedratinib for treating disease-related splenomegaly or symptoms in myelofibrosis
- TA598 Olaparib for maintenance treatment of BRCA-mutated ovarian, fallopian tube and peritoneal cancer after response to initial platinum-based chemotherapy
- TA611 Rucaparib for maintenance treatment of epithelial ovarian, fallopian tube or peritoneal cancer
- TA619 Palbociclib in combination with fulvestrant for treating metastatic, hormone-receptor positive, HER2-negative breast cancer after endocrine therapy
- TA620 Olaparib for maintenance treatment of recurrent, platinum-sensitive ovarian, fallopian tube and peritoneal cancer that has responded to platinum-based chemotherapy
- TA630 Larotrectinib for treating NTRK fusion-positive advanced solid tumours
- TA644 Entrectinib for treating NTRK fusion-positive solid tumours
- TA645 Avelumab with axitinib for untreated advanced or metastatic renal cell carcinoma
- TA658 Isatuximab with pomalidomide and dexamethasone for treating relapsed and refractory multiple myeloma
- TA663 Venetoclax with obinutuzumab for untreated chronic lymphocytic leukaemia
- TA673 Niraparib for maintenance treatment of advanced ovarian, fallopian tube and peritoneal cancer after response to first-line platinum-based chemotherapy
- TA677 KTE-X19 for treating relapsed or refractory mantle cell lymphoma
- TA693 Olaparib in combination with bevacizumab for maintenance treatment of advanced ovarian, fallopian tube and peritoneal cancer after response to first-line platinum-based chemotherapy with bevacizumab
- TA704 Trastuzumab deruxtecan for treating HER2-positive unresectable or metastatic breast cancer after 2 or more anti-HER2 therapies
- TA742 Selpercatinib for treating advanced thyroid cancer with RET alterations
Other technologies in managed access
- TA588 Nusinersen for treating spinal muscular atrophy
- TA743 Crizanlizumab for preventing sickle cell crises in sickle cell disease
- TA755 Risdiplam for treating spinal muscular atrophy in children and adults
Highly specialised technologies
- HST3 Ataluren for treating Duchenne muscular dystrophy with a nonsense mutation in the dystrophin gene
- HST6 Asfotase alfa for treating paediatric-onset hypophosphatasia
- HST12 Cerliponase alfa for treating neuronal ceroid lipofuscinosis type 2
- HST15 Onasemnogene abeparvovec for treating spinal muscular atrophy
Our health technology evaluations manual (2022) sets out a uniform approach to guidance updates following a period of managed access.
This is known as a managed access review.
We are currently undertaking further engagement with companies, industry bodies and patient groups who have an interest in those treatments which are currently in managed access in the Cancer Drugs Fund (CDF)