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Rare Diseases February 2019

Collecting data from patients to improve access to treatment


Sheela Upadhyaya

Associate Director, Highly Specialised Technology (HST) Programme, National Institute for Health and Care Excellence (NICE)

People with rare diseases can face many challenges on the – often long – road to accessing treatments. These challenges can make informed funding decisions very tough; in part due to limitations in the evidence for the treatment’s effectiveness. 

These evidence gaps are often difficult to quantify and decipher in world that values scientific data over real-life, experiences. Managed access arrangements (MAAs) represent a way of enabling patient access to promising drugs while collecting data from their use in the real world. The data, collected from patients, helps to address clinical uncertainties and mitigate some of the financial risk to the NHS. The more insight, the higher the chances of better long-term commissioning decisions. 

Multi-stakeholder approach across patients and healthcare professionals

In order for an MAA to collect meaningful and robust data, a multi-stakeholder collaborative approach is necessary. All stakeholders play a vital role in developing and delivering the MAA by helping to define which data to collect, how to collect it and from whom. 

Industry and the NHS are required to facilitate the collection of data agreed in the MAA, while clinicians and patient groups help to identify and overcome personal and practical challenges to data collection. 

Practicalities of a data collection group

In England, the National Institute for Health and Care Excellence (NICE) plays a role in overseeing the delivery of the agreement by ensuring the data outputs meet the needs of a future evaluation by NICE. NICE does this by assembling a Managed Access Oversight Committee (MAOC) for each MAA, consisting of representatives from the company, patient organisations, commissioning bodies and treatment centres. 

This group operates under the chairmanship of NICE with the primary function of reviewing the progress of data collection and identifying operational challenges in implementing the terms of the arrangement, ensuring implementation issues are promptly resolved. 

Who benefits from a managed access agreement?

These agreements offer an opportunity for patients with rare diseases to access new and innovative drugs that may deliver benefits to them and their families, in addition to collecting real-world data to address gaps in the original evidence base. This set-up provides a win-win scenario for all stakeholders: 

  • Patients who meet appropriate eligibility criterion can secure access to a treatment that would not routinely be available outside of managed access in England
  • Industry is able to collect data in a real-world setting that is applicable to a UK population
  • The NHS has some protection of the financial risk, as the agreement is time limited 
  • NICE can reassess the technology with a fuller evidence base in the future

These opportunities can only be explored when the NICE committee feel it is possible to collect further data to help close gaps in the presented evidence. The committee will ask itself: ‘Which data is missing?’; ‘Can it be collected?’ and ‘How much time will it take to achieve adequate data before it will consider if an MAA will be a good option to consider?’

A questionnaire to collect patient data

The data is collected mostly in the clinical setting but can be gathered in other ways, e.g. by asking patients and/or their families to complete questionnaires about the impact the treatment is having on their quality of life. 

At the end of the agreement, the collected data are incorporated into a new submission to NICE for re-evaluation to determine if the technology should be recommended to be provided routinely to eligible patients by the NHS. 

All stakeholders are made fully aware that the decision made by NICE at re-evaluation is the one the NHS will be mandated to implement.   

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