Evaluating medicines using 'real-world data'

Data obtained from day-to-day clinical practice and patients’ experiences should exert more influence on the decision on whether or not to reimburse the costs of medications. That is the conclusion Utrecht University PhD Candidate Amr Makady reached during his research. Makady defended his dissertation on Wednesday, 9 May.

In order to support their decision-making process, policymakers usually use the results of health technology assessments (HTAs), which are primarily informed by randomised clinical studies using pre-selected, homogeneous groups of patients and controlled protocols to monitor the patients. However, in order to better understand how a medication works, policymakers should also have access to ‘real-world data’.

Admission to basic healthcare package

Information from the real world can be collected from a variety of sources, through which patients can share their experiences with a medication, including patient registrations and even social media. Pharmaceutical researcher Amr Makady works at the National Health Care Institute, the Dutch government agency that evaluates the therapeutic value and cost-effectiveness of pharmaceuticals. The Institute then advises the minister on the admission of medications to the basic healthcare package. “Reactions in a patient forum, for example, can show that a medication doesn’t work as well in practice as it did in the clinical studies. Or that a medication has unpleasant side effects, but the patients feel that the benefits justify the side effects.”

Patient forum

Makady: “At the National Health Care Institute, we try to estimate the cost effectiveness of pharmaceuticals over longer periods, such as 30 years. However, the clinical studies upon which we base our conclusions often only offer insights over shorter periods. In my dissertation, I studied whether, and how, we should draw conclusions from current, real-world data for HTA and decision-making. That offers us much more relevant, and perhaps even more reliable insights about the experiences that large groups of people have with specific medications.”

Conditions for use

Makady: “In my dissertation, I examined the conditions for using these types of data in drawing up recommendations and developing policy at the National Health Care Institute. First of all, a wide range of stakeholders must agree on what real-world data actually entails. Furthermore, it is important that the parties discuss how the data should be analysed and interpreted. Obviously, we should also consider the privacy of the individuals referred to in these types of data. Only patient information that has been made anonymous should be used for this purpose.”

Social insights

A next step involves considering what to do with the information obtained from real-world data. “If day-to-day practice shows that a medication doesn’t work as well as originally thought, then should the National Health Care Institute change its reimbursement advice so that health insurers no longer have to finance it?” Makady’s dissertation also provides starting points for further research into the digitisation of patient information in general. “The question is: which conclusions can we possibly draw from sources such as big data and social media? We could obtain more insight into issues that affect the social arena than is currently the case. Perhaps, we may be able to use that knowledge to develop quality certificates for new sources of data”, according to Makady.

Collaborative effort

Amr Makady defended his dissertation at Utrecht University on Wednesday, 9 May. The research project is a collaborative effort between Utrecht University and the National Health Care Institute. Makady’s PhD promoters were Ton de Boer (UU), Hans Hillege (UMC Groningen) and Olaf Klungel (UU), along with co-promotor Wim Goettsch (ZIN & UU). The National Health Care Institute advises the Ministry of Public Health, Welfare and Sport on the effectiveness of medications, and on admitting those medications that are at least as effective as existing medications to the basic insurance package.