Supporting a patient organisation, 3rd Floor Heroes

September is childhood cancer awareness month. In Slovenia, one of the main patient organisations representing parents and caregivers of children that are facing cancer is 3rd Floor Heroes. This organisation is one of those that gave support to the CTGCT initiative, and this month was the right opportunity to give something back. We did this by considering the basic of why researchers and clinicians need to work with patient organisations. Their unmet needs and expectations are critical guidelines in developing new therapies. While we couldn’t yet address this need, we still heard that gifts for hospitalised children are something that could improve their wellbeing at least on their birthdays. Thanks to and people with good intentions, the CTGCT personnel provided gifts to the Division of Paediatrics, University Medical Centre Ljubljana.

The CTGCT has collaboration with various stakeholders in its core of the proposed action. Specifically, patient organisations and clinical research sponsors can identify unmet needs, and when these are addressed, we can expect an improved clinical trial experience (BMJ Open, 2020). Development and translation of novel therapies is a lengthy and costly process; thus we must do clinical trials as efficiently as possible, without worries that human factors will intervene to such extent that trials fail.

The concepts through which such collaborations exist are various, often starting with Memorandum of Understandings (or similar) that formalises expectations of all stakeholders, where terms such as “trust”, “empathy” have their purpose. This approach to development of new therapeutics is called a “Patient-centric” approach, which is essentially a feedback loop that enables to really address patients’ needs. However, this collaboration is not limited just to implementation and conduct of the clinical trial, neither dissemination of information nor clinical trial results. A collaboration between patient organisations and drug developers can also go in directions such as how the medications are packed, if patients are the one that directly administer the medicine.

It is important to understand that some patients, also described as ‘expert patients’, hold significant experiential or academic skills that carry high value for drug developers. They can have a combination of a medical condition and academic knowledge about this condition or have been involved in many events covering the specific topic. They may have also participated in similar clinical trials or had insights in one of them (ABPI, 2019).

Because of the information era and the widespread of information it is becoming increasingly important that stakeholders communicate and listen to each other throughout the course of developing new therapies. Patients or parents/caregivers of patients are increasingly becoming more involved in own medical care, and they play a role in how a new therapy, or a medical approach will be accepted.

The CTGCT has recognised that patient organisations must play a role in future of developing the Slovenian healthcare system. While this segment of stakeholders is quite underdeveloped due to lack of resources, the strong will of individual patients and parents/caregivers is enabling them to have some voice. Within the CTCGT, we plan to support them, in indirect actions such as the described one, and by directly involving them in development of new therapies. Supporting the CTGCT initiative means also supporting those with unmet medical needs.