Patient Advocacy Basics

In this lesson, you'll learn about what patient advocacy really involves, and how to do it effectively and strategically. 

We'll unpack what "the patient" actually means, from individual patients and carers to patient advocates, organisation representatives and patient experts; and look at the three levels advocacy operates on: patient support, research, and health policy. You'll explore common pitfalls (like advocating at the wrong level or attending meetings without real impact), map out the key stakeholders you'll engage with, and see real examples of evidence-based advocacy in PNH and other disease areas. 

By the end, you'll understand how to focus your efforts where they'll have the most impact, and you'll know which networks, resources and PNH Global Alliance programmes are there to support you along the way. 

In this lesson, you'll learn about what research actually is, and why you don't need a research department or a PhD to start doing it. 

We'll cover how the healthcare ecosystem works and where patient organisations fit within it, what separates an individual anecdote from real evidence (and why that distinction matters to clinicians, payors and pharma alike), and what patient experience data (PED) is and how it complements clinical trial and real-world data. You'll also get a simple five-step framework for planning your own project, from defining the problem to sharing the results, along with guidance on matching your method to your audience and the basic ethics, consent and GDPR considerations to keep in mind. 

By the end, you'll have the confidence and a practical toolkit to start generating your own evidence, however small the first step. 

In this lesson, you'll learn about how to read and critically assess scientific evidence, without needing to become a statistician. 

We'll cover the basic vocabulary of statistics, including the difference between descriptive and inferential statistics, what a p-value and a confidence interval actually tell you (and don't), why sample size and representativeness matter (especially in rare diseases) and how to spot the difference between correlation and causation. You'll also look at how data visualisation can shape (or distort) the story a graph seems to tell. 

By the end, you'll have a practical framework for reading a scientific paper or conference poster critically: what question was being asked, who was studied, how big and meaningful the results are, and what's missing; so you can engage with evidence confidently in discussions with researchers, clinicians and regulators. 

This session is divided into four parts – watch them at your own time! 

In this lesson, you'll learn about why and how patient organisations are shifting from relying mainly on personal stories to combining those stories with structured evidence. 

We'll look at why the traditional advocacy approach is no longer enough, exploring how HTA bodies, industry, clinicians and regulators each ask different questions about a treatment's real value; and introduce the Evidence-Based Advocacy Model as a way to bring patient experience and data together. You'll see real examples, including patient preference studies and the European Atlas on Clinical Trials, and walk through the process of generating your own evidence, from defining a research question to presenting the findings. 

By the end, you'll understand how to strengthen your organisation's influence by pairing lived experience with credible, methodologically sound evidence, and the common barriers patient organisations face in doing this well.