Challenges for patient organisations going through a Managed Access Agreement

Findings from a shared learning roundtable of patient organisations on their experiences of participating in managed access agreements.

Managed Access Agreements For Rare Conditions

In most cases, in order to access a treatment on the NHS in England and Wales, it must be approved by the National Institute for Health and Care Excellence (NICE) via a health technology appraisal (HTA) which aims to assess a treatment’s value for money. Rare conditions tend to affect small numbers of people creating uncertainties as to how many people have the condition, how the condition affects individuals and how the condition can best be managed and treated. These uncertainties in combination with the cost of rare disease treatments can make establishing these medicines’ value for money more challenging than those for common conditions. 

If a decision cannot be made at the end of an HTA because there are too many uncertainties, treatments may go into a Managed Access Agreement (MAA). An MAA allows the treatment to be provided for eligible NHS patients for up to five years while further data is collected. At the end of the MAA period the treatment will be re-evaluated taking into account this new data.

Shared learning roundtable

Patient organisations often make significant contributions to an MAA. Genetic Alliance UK hosted a roundtable of some of its members to share their experiences of participating in an MAA and offer support to members going through the process for the first time. It came to light that many of these charities faced similar challenges which broadly fit under three overarching themes:

  • Communication with the community – the group felt that the way stakeholders communicate with support organisations could be improved
  • Impact of an interim ‘no’ – seen by some as a necessary part of the committee’s drive for a cost-effective outcome, the group felt that this message was often hard on our community
  • Burden on charities and data collection issues – the group described the work they have to take on during an MAA with some calling for this to be considered more fairly

Through the discussion the group came up with a series of recommendations to help improve the process from the perspective of patient organisations involved in MAAs. 

Read the full report and recommendations here.

Next steps

These themes and recommendations have been presented to colleagues at NICE and we welcome their engagement and willingness to collaborate with us going forward on how best to implement some of these recommendations.

Read the full report and recommendations here. If you’d like to discuss this further with our policy team, please email