“Our main task is to ensure that we do not pose questions to patients that are actually unethical to ask”

Anja Schiel is a Senior Adviser & Statistician at the Unit for Health Technology Assessment (HTA) and Reimbursement, Norwegian Medicines Agency (NoMA). At the EHFG, she gave her perspective on “Making real world data real” during a workshop under the same title about improving regulatory approval of new therapies.

According to the World Health Organization, HTA is the systematic evaluation of properties, effects, and/or impacts of health technology. It is a multidisciplinary process to evaluate the social, economic, organisational and ethical issues of a health intervention or health technology. The main purpose of conducting an assessment is to inform policy decision making.

ER: What was the key message of the workshop “Making real word data real”?

AS: My message would be that drug development is complicated but there are new ways forward that are promising. In my opinion, we need to find good ways forward that ensure that we do the right thing and not fall for a hype. Sometimes we tend to think in isolated silos, but we have to listen to everybody who is involved if we want to succeed. For example, a solution that works for one stakeholder, like the regulator or the HTA agency, may not be a solution for patients or clinicians. This is the main reason why in the last ten years there has been an increased willingness in improving cross-stakeholder communication. We heard about the IMI project in this session [The Innovative Medicines Innovative (IMI) EHDEN project which aims to build a data network that allows access to data of 100 million EU citizens, standardised to a common data model].

I strongly believe in this project, because we have never witnessed such strong communication between different stakeholders. Even industry representatives have experienced it as a forum in which they can talk with each other at a very different level than ever before. And even the HTA agencies and the regulators have a much-improved understanding of each other’s work and perspectives. It is key that one doesn’t have to align everybody but one can at least understand each other’s needs better. And because of that, one might actually change one’s own behaviour and have other decision-makers in mind when giving advice or deciding on something.

AJ: Do you think that patients should have a bigger role, for example, by having an actual seat in the decision-making process at the European Medicines Agency (EMA)?

AS: Actually, the EMA currently runs a huge project on patient involvement. In the past, there was none and the EMA was criticised for that; now they are trying really hard to improve this. In particular, in the scientific advice process with companies in an early stage of product development, it is nowadays quite common that patient representatives are present at the meetings. It is very similar to NICE appraisals meetings, where there are always patients present. This also depends on the countries: for small countries, like Norway, it may be hard to find a patient organisation. In that case, you need to find someone who is willing to look beyond their own perspective and represent the patients as a group and not only their own disease-specific perspective. However, when you are patient it is extremely difficult to take on a group perspective.

The healthcare system has acknowledged that once you are diagnosed with something serious, you cannot be objective. No one would expect you to be objective in that situation. Our main task is to ensure that we do not pose questions to patients that are actually unethical to ask, like making a decision between A and B if both are unacceptable because they may be unsafe. We have a huge responsibility in protecting patients from these situations, which were common fifty years ago when patients were left alone in desperate situations. We really must prevent patients from having to make desperate decisions, because in these circumstances decisions can never be educated or objective.

AJ: Do you think there should be more room for the emotional perspective of patients? After all, it is their unique perspective on things.

AS: The unique perspective is actually quite often reflected when we talk about measures of success in a trial. Patients can explain to us what a relevant endpoint would be for them, whereas clinicians tend to measure things that are easily measurable and objective. Patients can bring in the perspective on what is relevant to their everyday quality of life. This is also a huge difference between HTA agencies and regulators, because for HTA, taking into consideration the quality of life is absolutely essential, whereas, for the regulatory side, it is more about an objective assessment of whether the risks outweigh the benefits.

This interview was conducted by the Young Gasteiners Elena Roel (ER) and Arjan van der Star (AJ).

Leave a Reply

Your email address will not be published. Required fields are marked *