How are managed access agreements used in the context of the NICE highly specialised technologies process?
The HST process
The highly specialised technologies (HST) process was introduced in 2013 for the purpose of evaluating very rare conditions. The HST process is based upon the existing technology appraisal process but is specifically designed to take into account the difficulties of designing and running clinical trials in small patient populations that will undoubtedly result in greater uncertainties in estimates of cost-effectiveness than would be acceptable in the standard STA process. Consequently, a more generous cost-effectiveness threshold of £100,000 per QALY is applied, rising to £300,000 per QALY for treatments that deliver more than 30 additional QALYs to the patient over their lifetime1. However, when uncertainties in the data have a substantial impact on the cost-effectiveness result, NICE may wish to recommend the technology in the context of a Managed Access Agreement (MAA); that is, that the technology is used only in the context of research or that the technology is recommended as an option, but that formal data collection is conducted alongside routine use. The ideology behind this is that patients are not denied access to a potentially life changing treatment while allowing further data to be collected to provide increased certainty around the cost-effectiveness of the treatment. To date, out of 14 published HST appraisals, whilst all have been approved, four have required a MAA2-5.
Managed access agreements
The HST process includes reconsideration steps at specific points in the timelines that enable the company to develop new, or enhance existing, MAA proposals. Such a step can be expected to add 4-12 weeks to the appraisal timelines. When considering whether to make a recommendation in the context of a MAA, NICE will consider the following1:
- The need for and potential value to the NHS of additional evidence that can inform the future development of NICE guidance and clinical practice on the use of the technology;
- The uncertainty in the analysis and what is needed to reconsider the decision in the light of research findings;
- Whether the data collection is feasible;
- The extent of irrecoverable costs incurred from introducing the technology and plans to mitigate this risk;
- The time it is likely to take for research findings to be available to inform subsequent NICE guidance and clinical practice;
- Other factors that may impact on the data generation, such as other research that is underway or likely to be commissioned and completed
Should a MAA be required, a Managed Access Oversight Committee will be convened to oversee the process. This committee includes representatives from NICE, the company, NHS England, treatment centres, patient groups and other research partners involved in data collection. The committee will specify the required elements of the MAA, which according to NICE’s HST process guide may include1:
- A proposal that addresses a significant uncertainty in the evidence base identified by the evaluation Committee (for example, a plan for generating further evidence for a patient population that is covered by the marketing authorisation but not represented in the clinical trials);
- A duration of the arrangement, with a rationale, that is agreed by the key stakeholders: the company, NHS England and patient groups;
- Clearly defined starting and stopping criteria with identified entry and exit points throughout the treatment pathway;
- Treatment continuation criteria;
- A list of outcomes for which data will be collected;
- How data will be collected and analysed;
- An agreement on how regular the outcomes in the MAA will be reviewed;
- The funding arrangements;
- A statement that describes what will happen to patients receiving treatment who are no longer eligible for treatment if a more restricted or negative recommendation is issued after the guidance has been reviewed following data collection;
- Financial risk management plans agreed between NHS England and the company that undertake risk-sharing for the duration of the agreement;
- An acknowledgement by patient groups of the role and responsibilities they hold within the arrangement.
Once the required elements have been agreed upon, the company is responsible for developing a data analysis plan and for data collection and data entry, as well as database management and data analysis. A date will be set for NICE to review the original recommendation at which point NICE will review the additional data collected and issue revised recommendations.
MAAs – a win for all?
MAAs undoubtedly play a role in ensuring patient access to potentially life changing treatments in therapy areas where there are currently little or no treatment options and where the rarity of the disease makes running clinical trials very challenging. Many of the conditions treated by highly specialised technologies are life limiting and the therapies have the potential to significantly improve patients’ quality of life. However, NICE still has a responsibility to ensure the level of supportive data is adequate to justify NHS spending on what are usually very costly treatments. Of the MAAs currently in place, only one has completed data collection (elosulfase alpha for treating mucopolysaccharidosis type IVa) and final NICE guidance is expected at the end of 2021. It is therefore too early to draw any conclusions on how effectively MAAs are bridging the data gap, but it is certainly an area to watch.
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