Training patients in PE has resulted in many successes, but there have also been missed opportunities, writes Traceann Rose, Program Director at the Children’s Tumor Foundation

The Children’s Tumor Foundation established a Patient Engagement Training Program two years ago in a bid to train the neurofibromatosis (NF) patient community to become more engaged in the medicines research and development (R&D) process. 

At the time of establishing the program we were not familiar with many other rare disease groups that were players in the PE space. In fact, it has been a learning curve for CTF as well as the patients involved. It appears PE in the rare disease space was viewed rather as a trend and it was not really being practiced in a meaningful way to advance R&D.

We identified that we needed to train the NF patient community to understand the R&D process, what PE is and also the value and impact of patient involvement in research before we could begin participating in opportunities. Fortunately, we had the benefit of the EUPATI training program to benchmark and create a blend of online reading materials and educational videos, and in-person training with NF experts and other representatives. 

In reflecting on the past two years the program has evolved organically as the field of PE has grown in the USA. CTF has greater understanding about how patients can help accelerate R&D for NF and rare Diseases. We are proactively and successfully engaging stakeholders in industry, FDA, rare disease and researchers for collaboration.

Since we began, we have enjoyed many opportunities and successes, having trained 18 patient representatives in the first year of the program to become expert patient advocates.

We have participated in national and international projects such as the PARADIGM Clinical Trials working group to designing metrics for successful patient engagement in clinical trials; we were able to share that tool with the Clinical Trials Consortium working on NF.

Patient advocates have been busy. They are now speaking at NF conferences and webinars with National Organization for Rare Diseases and sharing stories about living with NF under the umbrella of the CTF PE program.

Patient advocates are also committee members, joining experts in a clinical setting developing endpoints for NF clinical trials that will support effective claims in new drug applications to the FDA for neurofibromatosis. They are also participating as co-reviewers and lay-patient representatives for the Children’s Tumor Foundation Research Programs (such as NF Registry and Research Awards). They are giving feedback on the significance of the project for the field of NF with consideration to the severity of the disease, number of individuals impacted by the disease, and advancement of scientific knowledge. 

However, it has not been without its challenges. For example, we have no other known rare disease groups with a similar programme with which to share learnings and best practices.

Despite patients being trained and empowered, there remained insufficient opportunities for involvement; we have found it challenging to engage with clinical trial teams to involve patients.

We also suffer from a lack of funding to support travel and participation in PE opportunities.

Despite this, we are continuing to raise awareness across the patient and research community about what PE is, and getting researchers to work with patients and understanding the value of their input.

In terms of evolving the program, we would have involved patients in selecting and designing training courses. We would also like to have a broader scope to the PE program beyond just training to include such things as a speaker’s bureau. This would enable pediatric, adult and a broader scope of patient experiences and representation to access varied opportunities.

We would also survey patients early in the process to help improve the program and develop metrics for successful engagement, and market the program as a rare disease program that extends beyond NF since the challenges in NF as are similar for other rare diseases.

What advocates are saying:

  • “Being a Patient Advocate empowers you and gives you some control over the disease when you can advocate for yourself and your child.”
  • “It is very important to be a Patient Advocate because I feel as though I did not have much of a voice. Now I have more of a voice and am connected to the research community.”
  • “It means everything to be able to contribute because it means that I am helping my family and not sitting back.”
  • “It’s important for people to have a voice and be a part of the research process because scientists don’t always know what the other side is experiencing.”
Editorial note:

To help you embed patient engagement across your organisation, look out for the PARADIGM Recommendations for patient engagement capabilities* that can help ensure your team is fully equipped and confident to do patient engagement.

* All PARADIGM tools will be released during the summer of 2020. Follow the information on our website. The final names of each tool might change.

This is the second article of the mini-series of six articles. Find the other articles from here.