From Research to Reality: Understanding the Clinical Trial Process
Every family in our community brings a unique perspective to the world of research. Some of you may already be familiar with the clinical trial landscape, while others might find the scientific language a bit daunting or aren’t quite sure where to begin. No matter where you are in your journey, please know that your experience is valid, and you are not alone.
We have created this page to be a welcoming home for information. Our goal is to provide a steady hand for anyone thinking about, preparing for, or currently navigating a clinical trial. We have gathered resources from the most trusted organisations to help explain the “how” and “why” of research, ensuring you have the tools you need to make the best decisions for your family.
Resources for You and Your Family
These materials are designed for you to explore whenever you feel ready. You don’t need to be an expert to start; these guides are here to provide context and comfort:
* Simple Explainer Videos: To help visualise how research works.
* Genetic Primers: Easy-to-follow articles on the science behind FOP.
* A Helpful Glossary: A guide to the terms you’ll hear during doctor visits and trial appointments.
Our hope is to take the mystery out of FOP drug development. By providing these resources, we want to help you feel more prepared for conversations with clinicians and more confident in your role as an advocate for yourself or your child.
This collection will grow over time, so if you find a specific video or article that brings you peace of mind, please share it with us so we can pass that help along to others.
Visit our Research Learning Hub
Before we start, we would like to acknowledge the hard work of so many individuals – patients, parents, friends, doctors, nurses, researchers, pharmaceutical companies and more – who, over the years, have got us where we are today.
We are ultra rare! And within the rare disease space we are most definitely punching above our weight! This is thanks to the hard work and dedication of so many people over the years, from the researchers who discovered the gene to the people working on the treatment today. We are lucky to have researchers around the world all trying to better understand FOP and ultimately to find a treatment and a cure.
One of the reasons they are able to do this, is thanks to the dedication and commitment of the families over the years. From the parents who set up the first patient organisations and pen-pal groups – both here and around the world – to our community today. We have been a loud, determined group who have raised awareness of the challenges living with FOP brings, and we have pushed to have our voices heard!
If you have taken part in the Natural History study, participated in a clinical trial, are signed up to the IFOPA’s FOP Registry, completed surveys, raised money to support the research, shared awareness day posts, told your story… then you have made a difference. You have helped us to get to where we are today.
We still have an incredibly long way to go, but today we want to thank you for getting us this far. With your continued support, we can do this. We are filled with hope for the journey ahead.
Access Approved? How Medicines are Funded and Made Available in the UK
POSTPONED: Please register your interest and we’ll let you know the new date once it’s in the diary!
Via Zoom link
Presenter: Chris Bedford-Gay, Trustee
In our second webinar, we aim to shed light on the crucial steps involved in making a new FOP medicine available to patients on the NHS in the UK. It will explain the distinct roles of key regulatory and funding bodies.
The webinar will cover:
Getting Licensed – The First Hurdle: An explanation of the Medicines and Healthcare products Regulatory Agency (MHRA), the UK’s “gatekeeper” for medicines.
The Role of NICE: A detailed look at the National Institute for Health and Care Excellence (NICE) and why it’s necessary for the NHS to assess if a new medicine offers good value for money.
The Highly Specialised Technologies (HST) Process: A special route designed for very rare diseases like FOP, its flexible criteria, and how it offers a fairer chance for these high-cost, low-patient-number treatments to gain NHS approval. The critical role of patient advocacy and input in this process will be highlighted.
Getting the Medicine to YOU: How, once a medicine is approved by NICE (especially via HST), NHS England funds and arranges its provision.
The webinar will conclude with a Q&A session, offering an opportunity for attendees to ask questions.
Please note: Programme subject to change
What’s Taking So Long? The Realities of Developing FOP Treatments
Thursday 10th July 2025 @ 8pm
Via Zoom link
Presenter: Dr Andrew Rankin, Trustee
Our first webinar aims to demystify the complex journey of developing drugs for Fibrodysplasia Ossificans Progressiva (FOP) and provide current information on treatment opportunities.
The webinar will cover:
The Journey of a Medicine: From initial ideas and understanding FOP’s biology to early lab research and crucial pre-clinical testing for safety and efficacy before human trials.
Navigating Clinical Trials: A detailed explanation of the different phases of clinical trials (Phase 1, 2, 3, and 4), what each phase entails, why they are necessary, and how they lead to regulatory approval.
Considerations for Patient Participation: What patients can expect when participating in a clinical trial
Current FOP Clinical Trials & Treatment Opportunities: An overview of the current landscape of FOP research, highlighting different therapeutic approaches and specific ongoing trials.
Attendees are invited to send in questions before the event. The webinar will also conclude with a Q&A session
Please note: Programme subject to change
The FOP Registry is independently operated by the IFOPA.
The Registry is the largest and most detailed global collection of clinical and medical information about FOP, contributed by individuals living with the condition. Its primary purpose is to help clinicians and researchers gain a deeper understanding of how FOP affects people, aiming to improve patient care and accelerate the development of new treatments and a cure. The Registry is open to anyone with FOP, with information securely provided online by the person living with FOP or their caregiver, ensuring only anonymous data is shared with researchers.
Participants also receive a $25 gift card every time they complete a survey.
Learn more here: The FOP Registry
Download the brochure: Here
