It’s time to put more POW! in PAOs.
I have a vision, I must admit I don’t know how to make it a reality but let me tell you about it, maybe we will figure it out.
Patient advocacy organisations (PAOs) are hugely important for the people they serve and society in general benefits from their work. Every PAO is unique in many ways. Some are very large organisations, others are very small, their resources range from the tens of thousands to the tens of millions of Euros and consequently, their structures and services vary greatly. So generalisations are difficult, treacherous even, but to tackle the subject and describe my vision I’m forced to talk in general terms of a heterogeneous reality.
My vision is for PAOs to have much greater influence in healthcare - more than influence, real power. I would like to see clinical trial design require input and approval from a PAO before a trial can begin.
Consider the following from Leucht, S., Helfer, B., Gartlehner, G. et al. How effective are common medications: a perspective based on meta-analyses of major drugs. BMC Med 13, 253 (2015). https://doi.org/10.1186/s12916-015-0494-1: “For an outcome affecting quality of life, ½ of a standard deviation is considered to be a minimal clinically important difference . Out of 17 common pharmacological treatments examined, only 11 met this threshold. In four of them efficacy was represented by surrogate outcomes, such as diastolic blood pressure or fasting plasma glucose, and not patient-oriented outcomes, such as pain, mortality or adverse events. Therefore, patients might not have experienced substantial benefits related to their well-being and quality of life after therapy with some of these drugs.”
Only a PAO can provide an unbiased focus on patient-oriented outcomes because they have unparalleled knowledge and empathy for patients and caregivers. Currently, PAOs have soft power; to mandate their formal inclusion in clinical trial design would give them real power and responsibility. For that to be effective, PAOs that wish to be consulted in clinical trial design will have to be specially authorised and closely regulated. PAO managers will need to have specific competencies and qualifications. Some exist, for example, Università Cattolica, in Italy, offers a Master's in “Patient Advocacy management”.
With strong credentials and impeccable credibility an authorised PAO would be an implacable reality check and a formidable partner in the development of novel treatments. With the success rate of clinical trials in the last decade barely above one in ten and the real-world success of many approved treatments below expectations, a more patient-oriented perspective might improve not only outcomes but also financial performance.