Rare Patient Voice: The Rare Voice: Patients, Family Caregivers, and Research 

Wednesday 1st February 2023, at 4pm GMT/ 11am ET

WEBINAR STARTSIntroduction & Housekeeping – to include welcome, agenda, house-keeping. Interactive question and answer from audience – audience will be able to ask live questions and up-vote questions from fellow participants. A very warm welcome to our THIRD in the series of RARE Rev-i-nars. Today we will be talking about how we can embed the voice of the rare disease community into research, and why, regardless of our background, we should all consider research as something we are interested and engaged in.  I am Nicola Miller, Creative Director at RARE Revolution Magazine, and in the background working on the tech today, I am joined by my colleague Becky Pender.  Today’s session is in partnership with RARE PATIENT VOICE who aim to improve the lives of those living with rare disease, by including the voice of the community into rare research. We are thrilled to have so many register for today and would encourage you to take advantage of our great panel today, by using the Q&A to ask your burning questions. And if you see a question asked that is important to you, you can upvote to identify hot topics. Any we don’t have time to get to today we will endeavour to answer after the session closes and feed back to you all.  Today’s session will be also available to revisit on demand after today’s event and we will circulate links to this in due course.

Nicola then introduces the panellists ending with Pam. Pam gives a little background on Rare Patient Voice I am delighted to be joined by another very knowledgeable and international line up today who can help guide us through what engaging in research can look like for families, and the benefits of participation. Lee ReaveyCofounder and CEO NCBRS Worldwide, an advocacy group supporting those affected by Nicolaides-Barait-ser Syndrome  Taylor KaneFounder and executive director of Remember the Girls, an international non-profit who unite, educate and empower female carriers of x-linked genetic disorders AND Pam CusickSenior vice president of Rare Patient Voice who empower patients and caregivers to share their voices with researchers and companies developing products, devices, and treatments to improve lives. Hello all and welcome Before we dive in, I would like to say thank you to everyone who took part in our poll to shape the discussion today, by telling us what you wanted to know. So let’s get straight to it and start with the basics of talking about why families should be interested in research in the first place. PAM can I come to you first.

Section 1 – Benefits of taking part in research?

Question to PamPatients and family caregivers are busy living their lives. Why should they take time to participate in research?

Question to TaylorHow does taking part in research enable patients and family caregivers to be heard?

Question to Lee from your perspectiveAs a patient and an advocacy group leader, what has your experience been with taking part in research?

Question to Pam So Pam we now know the why – but Do companies really listen to their patients and experiences when making decisions about new products, services, and approaches to rare disease?

Section 2 – Types of research Thanks all – now let’s turn to types of research.

Question to Pam can you outline for usWhat are the types of research studies that we as a community can take participate in and how easy is it to take part?

Question to Lee, can you explainWhat is the difference between a patient registry and participating in research? Can patients do both?

Question to Taylor from you experienceWhat are the advantages of signing up with a company like Rare Patient Voice?

Question to Pam-this is a question many will want to knowDo patients/family caregivers get paid for their time and can you speak to how the program can function as a passive fundraising tool for advocacy groups as well as individuals? *raised the honorarium to $120ph in celebration of our 10th anniversary

Question to Taylor and Lee can I ask you both. Lee first – TaylorLee and Taylor, both of your organizations are active in the Rare Patient Voice referral program. What has your experience been with RPV?

Round up of conversations and Q+A from attendees ROUND UP Let’s head over to the questions…

Signpost and goodbye CLOSE I would like to give a sincere thanks to all of our panellists today, Pam, Lee and Taylor, and to Rare Patient Voice for partnering with us to bring this important discussion to you all today. Thank you to you all for listening and bringing us your insightful questions, I hope you have enjoyed it as much as we have. Please feel free to reach out to us via the contacts on screen if you have any feedback or questions post event and for signposting on how to take your next steps in research participation. Many thanks and have a great rest of your day.

RARE REV-inar Episode 003 Rare Patient Voice – Part 1 – YouTube