Patients’ views and involvement in benefit-risk evaluation

The ultimate mission of a medicine is to benefit patients. The role of legislation and regulators is to ensure that new medicines are indeed of benefit to patients. The unavoidable risks in medicines must not outweigh the benefits.

Regulators may be accused of not considering the risks seriously enough.

  • Should they have asked for more clinical trials?
  • Could new and maybe serious ADRs have been detected earlier?
  • If so, should the medicine have been approved at all?

Perhaps for such reasons, regulators and decision-makers in industry have become more risk-averse during the past 15 to 20 years. Newly discovered ADRs may be serious and on this basis the company may decide to stop further development of the medicine, sometimes late in the development process.

After marketing a new medicine, additional serious (though perhaps rare) ADRs may be discovered. The regulators can decide on this basis to withdraw the approval of the medicine and remove it from the market or suspend the MA until more data are available for an evidence-based decision.

The package leaflet / product information must by law clearly state the benefits and risks to give the patient the chance for an informed decision. What are the patients’ views in this? In some cases, patients do not agree with the risk-aversion perspective of the regulators as found in the package leaflet.

A recent example is the medicine natalizumab for the treatment of multiple sclerosis (MS). Some rare but fatal side effects occurred after marketing. The CHMP was prepared to remove natalizumab from the market. Before doing so, the CHMP consulted the MS patients’ organisation. The patients did not agree with the CHMP. They found the medicine beneficial even though they ran the risk of some serious new ADRs. The patients’ benefit-risk assessment was different from that of the regulators and the pharmaceutical company. From a patient’s point of view, the experience of an enhanced quality of life may make it acceptable to run the risk of experiencing serious side effects.

The increasing involvement of patients in the medicines’ approval process has considerable potential. By involving patients or patients’ organisations as early as possible, better, more informed decisions can be reached.