From Research to Reality: Understanding the Clinical Trial Process
We’re thrilled to have you join our upcoming FOP webinars on ‘Making FOP Medicines Happen: From Research to Reality’.
We recognise that there’s wide range of knowledge and understanding about the ‘bench to bedside’ process within our FOP community. We have some families who are experienced, have been on this journey for many years, and perhaps have even contributed to the trial process in one way or another. We have other families who maybe feel overwhelmed by the topic, maybe science at school ‘wasn’t their thing’ and don’t know where to start. We also have very new families on board, who are maybe looking for an overview of the FOP research landscape.
In response to this we’ve created this dedicated webpage as your central hub for pre-webinar resources. We hope they will help you get the most out of the webinars – that you get what you want and need for you and your family.
There is a wealth of information out there on the internet, so we have tried to collate some of the best of what’s out there. It most certainly isn’t everything, so feel free to explore further with a quick Google search (just be sure to only use reputable sources and organisations). And if you happen to find a great resource you think others might find useful, please let us know too!
Here in our Hub, you’ll find short explainer videos, introductory articles about genetics and trials, and a glossary. These materials have been chosen to provide you with background information and context, allowing you to do some pre-reading and get familiar with key concepts at your own pace.
Our aim is to demystify the science and processes behind FOP research and drug development, empowering you with knowledge and preparing you for engaging discussions. Feel free to explore these resources before the webinars, so you can join feeling informed and ready to learn!
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
Thursday 24th July 2025 @ 8pm !!!NEW DATE!!!
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
Living Well with FOP
While we all want and live in hope of a treatment for FOP, it’s important that we don’t lose sight of living for the now. We all want to live a good life and a good life well. The Living Well Campaign is being led by Metabolic Support UK and Beacon. We are pleased to be a part of this.
Learn more about the campaign and see our poster: Here
A mutation hiding in one unique patient could save others from forming a ‘second skeleton’
An article in the Penn Medicine Journal, speaking with Professor Fred Kaplan and Andrew Davies – the unique patient with FOP.
Read online: Penn Medicine
An Introduction to Gene Therapy
An easy-to-read overview of Gene Therapy, why it’s done, Risks, and What to Expect. From the Mayo Clinic, Minnesota
Read online: Gene Therapy
Genetics 101 by National Geographic
This video provides an introduction to genetics. It explains that genes, composed of DNA within chromosomes, make up the human genome, and highlights the significance of the Human Genome Project in mapping the human genetic sequence, while also acknowledging the future opportunities and ethical considerations in genetic testing and manipulation.
Watch: Here
Beacon Resources Hub
The Hub brings together resources from across Beacon’s training programme to provide patient groups with one, central place to further their learning on topics of interest in the field of genetics and living with a rare condition.
The e-learning resources are 100% free to access, but you do need to enroll with them.
Visit their Hub here: Get Started
Some courses which may be of particular interest:
Introduction to Genetics
Medical Research Explained
Rare Disease Research and Trial Design
Drug Repurposing Explained
Navigating Health Technology Assessments
Understanding Advanced Therapies
Genetics Basics in the UK
This webpage from Gene People explains genetics in a simple way, for a UK audience. It explains how tiny instructions inside us, called genes, guide how we grow and what we’re like. It explains about chromosomes and DNA, which are like the special books where these genes are stored. There are also links to other websites and resources.
It’s a guide to understanding the amazing code that makes each of us unique – and a great place to start if you are baffled by genetics!
Learn more: Beginners Guide
The Drug Development Journey for FOP: How Medicines Become Treatments
The IFOPA have produced a video which outlines the process of developing drugs for conditions like FOP, starting with lab research to identify potential medicines. It illustrates how medicines then undergo multi-phase clinical trials to assess their effectiveness and safety in people before going to regulatory bodies to evaluate the medicine for approval.
Watch now: The Drug Development Journey
Intro to Health Technology Assessments by Beacon
This video explains Health Technology Assessments (HTA) and their application, particularly in the context of rare diseases. It highlights the concept of opportunity cost in healthcare and how HTA aims to ensure fair and consistent decisions on spending by balancing costs against patient benefits. The video also discusses the challenges of applying HTA to rare diseases due to limited information and high costs, emphasizing the growing importance of incorporating the patient voice in such evaluations.
Watch: Here
Introduction to Gene Therapy in FOP (2020)
The IFOPA hosted a webinar, led by Adam Sherman and Betsy Bogard to give an introductory overview of gene therapy, specifically in the context of FOP. It explains how gene therapy aims to correct mutations by adding or changing genetic material to produce normal proteins, using various approaches like gene silencing, addition, replacement, and editing. The video also discusses the challenges and considerations for FOP gene therapy, including safety, targeting, durability, and the difficulty of targeting muscle tissue.
Watch now: Introduction
Gene Therapy: Your Questions Answered
This video addresses common questions about gene therapy, covering its recent developments, effectiveness, treatment methods, and potential side effects, as compiled by the National Organization for Rare Disorders (NORD). It also clarifies the distinction between gene therapy and gene editing, and advises on how to find clinical trials.
Watch here: Gene Therapy
