Pharmaceutical Accountability Foundation

Martina Liccardo’s take on the role of the European Medicine Agency in Leadiant’s price misuse with CDCA

Highlighting some Master Thesis discussion points from Martina Liccardo's research project for PAF

As an international student I have always sought out opportunities to meet local people and to broaden my personal and professional network.

When provided with the occasion, I did not hesitate to write my master’s thesis in partnership with the Pharmaceutical Accountability Foundation as I was sure it would be a great chance to learn more about the pharmaceutical industry in general, and to meet experts of the sector and beyond, who could only further my education and enrich my cultural repertoire.

The best way to honour the Foundation’s mission was to make their efforts and commitments known to others. Hence, I decided to incorporate in my research their first advocacy case concerning the CDCA medicine, an orphan drug intended for the cure of a life-threatening or chronically incapacitating condition affecting not more than five in 10,000 persons.

After the authorisation, this medicine received ten years market exclusivity, which is not unusual per se (such scenario is indeed allowed by EU legislations), however, once obtained, the price progressively increased until reaching 500x the original value.

My research focused on trying to grasp the extent to which this possible situation may have been contingent upon the European Medicines Agency’s lack of autonomy and transparency (key characteristics of the theory of Regulatory Governance) in evaluating medicines and providing marketing authorisation recommendation to the Commission.

My research findings (retrieved from primary documents as well as expert interviews), have shown that:

  • The European Medicines Agency is highly dependent on the regulations dictating tasks and procedures, from which the agency has no autonomy to diverge. It is reliant on Member States’ provision of experts to be appointed on their boards. Finally, industries provide up to 90% of the agency’s financial resources, making it particularly susceptible to their requests.
  • Regarding the characteristic of transparency, the agency’s apparent promises fall short. It seems that through its operations, EMA favours industry’ interests by safeguarding potentially sensitive information present in certain documents.

Expert interviews have revealed that the problem of “price misuse” such as the one for the CDCA is not to be attributed to the European Medicines Agency, regardless of the shortcomings it may have in its organisation. Rather, to the way the system has been structured and the way legislations were written. The agency does not regulate nor control in any way how medicines are purchased and distributed.

In my opinion, even though the European Medicines Agency does not hold responsibility for this specific case, a possible solution could be found in a European pricing scheme that -at least- obliges companies holding patents of certain medicines to respect a price range. Hence, avoiding those to generate huge profits harming those patients who may not be able to afford the treatments (such as for the CDCA case).

I strongly invite other people who may be interested in this subject to research and ask questions when curious. It is the best possible way to draw attention and make other people aware of certain problematics.
Moreover, my research could be expanded on a scientific point of view, and I leave this as a suggestion for further studies.