What is a methods qualification?
PREFER's aim has always been to develop recommendations for our stakeholders on work co-created with our stakeholders. In spring 2018, the PREFER project initiated the process for receiving a qualification opinion from the European Medicines Agency (EMA), adding an external multi-stakeholder evaluation of the evidence we present. In essence, we put forward our framework along with some points to consider for method selection for a scientific advice process, where EMA’s scientific Committee for Medicinal Products for Human Use (CHMP) provides what is known as an ‘opinion’.
At the same time, we decided to explore the possibilities of having our framework evaluated from a health technology assessment (HTA) perspective trough the European EUnetHTA network in a joint process. Resulting in the first parallel qualification by EMA and Health technology bodies!
This is a two-stage process. We have answered questions from both EMA and EUnetHTA, and have received a ‘qualification advice’ from EMA on the most appropriate way to develop robust evidence. The second stage of the process is a public qualification opinion. Before that opinion is final, EMA will open the evaluation for public consultation by the scientific community. This will ensure that information is shared with the broader public, and that both our framework, with the associated points for considerations in methods selection, and the evaluation is open to scientific scrutiny and public discussion, allowing patient groups an opportunity to comment.
The framework and points for consideration on method selection that we are asking EMA and EUnetHTA to evaluate describes a structured approach to assess and use patient preferences as input in medical product decision-making. These decisions are taken by industry, regulators, HTA bodies and payers in the development, approval and post-approval phases of a medical product’s life cycle. The actual framework offers a set of principles, guidelines and tools. It provides a structure to guide those who conduct preference studies through what we have identified as key issues in preference study design, study conduct and analysis. The framework also guides decision-makers through assessing and using preference study results in their decisions. These points to consider provide specific applications of the framework across different preference study examples and methods. Which means they describe which methods work best to address specific questions in given situations. And inform decisions taken by the companies that develop medicines, the regulators that approve medicines, and health technology assessment bodies and payers who decide what becomes available to patients.
To support patient input to this process, we have described the framework in non-expert language that supports communications about our work. We have also published a high level summary in plain language about how to plan and carry out a patient preference study for patients and non-experts who want to know more about our ongoing patient preference case studies.