The OIFE does not fundraise for research, but we promote research by doing the following:
- Encouraging more research in OI on different aspects
- Networking with researchers & clinicians worldwide
- Being informed about ongoing research in OI on a global level
- Creating arenas for OI researchers to meet
- Documenting unmet needs and preferences of people with OI through initiatives like the IMPACT survey or the Pain survey
- Disseminating results from research through events and communication channels like OIFE Magazine and our website
- Assisting with recruitment through announcements
- Endorsing/supporting specific studies in their application process
- Partnering with specific projects which has a broad international scope
- Providing advice from the patient perspective (patient involvement)
Some topics are common for both OI and other rare bone conditions. Each and one of us also have limited resources. This is why we have developed the project A stronger BOND between us, to collaborate where it makes sense.
OIFE believes that all clinical trials and research projects affecting OI directly should have some kind of patient involvement. Patient involvement can happen in different ways and in different phases of a project.
OIFE Investigator Meeting and other events
In November 2022 OIFE hosted our very first OIFE Investigator Meeting. More than 140 participants from Europe and beyond took part in the online event which mainly included researchers and clinicians, but also some industry representatives, patient representatives and students. The programme both included talks about what is going on in OI research (basic and clinical) as well as short workshops on how we can collaborate more closely within European OI research. The event had very positive feedback and a large majority wanted the event repeated as an annual online event.
The OIFE also attends scientific conferences on OI to network and interact with researchers and keep up to date. The most important ones being the scientific conferences– like the International Conference on Osteogenesis Imperfecta that is held every third year, the last one being OI2022 in Sheffield and the ICCBH-conferences held every two years.
The OIFE also attends scientific conferences on OI to network and interact with researchers and keep up to date. The most important ones being the scientific conferences taking place every 3rd year – like OI2022 in Sheffield.
Basic, Applied and Clinical Research
OIFE collaborates with both basic researchers, clinical researchers and industry.
Basic research, is a type of scientific research with the aim of improving scientific theories for better understanding and prediction of natural or other phenomena. An example from OI, can be achieving a better understanding of how bone cells behave in different environments by using mouse models or zebrafish models.
In contrast, applied research uses scientific theories to develop technology or techniques which can be used to intervene and alter natural or other phenomena. This includes clinical research and studies to investigate new treatments or drugs for OI. Basic research often fuels the technological innovations of applied science. The two aims are often practiced simultaneously in coordinated research and development.
Clinical research determines the safety and effectiveness of medications, devices, diagnostic products and treatment regimens intended for human use. These may be used for prevention, treatment, diagnosis or for relieving symptoms of a disease.
In addition to more traditional medicinal therapies and products (medications), there are also advanced therapies being investigated, which refers to medicinal products that use gene therapy, cell therapy, and tissue engineering. These are called Advanced Therapy Medicinal Products (ATMPs)
Clinical research is different from clinical practice. In clinical practice established treatments are used, while in clinical research evidence is collected to establish a treatment. When you visit your doctor at the hospital for a medical problem or your family doctor for an annual checkup – this is part of clinical practice.
Clinical trials are an essential part of developing new treatments and test whether a specific treatment is effective and safe. Trials start with a small number of volunteer test subjects, and can grow to include larger number of participants.
There are different kinds of clinical trials. The gold standard is the controlled, randomized, and double-blind studies. See this educational video which explains some of the most important expressions from clinical trials.
While clinical studies of treatments for OI and other rare diseases have some differences, their basic structure is the same. All enroll volunteers at each phase, following protocols set out at the beginning of the study.
The phases of a clinical trial
The phases of a clinical trial include:
- Preclinical or Non-clinical testing: scientists give the treatment to animals (like mice or zebrafish) to see if it produces a response and is safe
- Phase 1: a small group of human participants are examined; looking for safety, proper dosage amounts and to confirm it has some response in humans. Phase 1 may be done in healthy volunteers or in patients with the disease, or at-risk of the disease, under study.
- Phase 2: further tests safety and if it stimulates response in different types of people
- Phase 3: determine if treatment is effective. These studies should be large enough to find relatively rare side effects that might be missed in earlier studies.
- Phase 4: sometimes used after a treatment has been approved, this phase is used to find any long term effects of the treatment
Individuals with OI play an especially important role in the outcome of clinical trials because they have a rare condition. Compared to more common conditions, rare disease based clinical trials usually have far fewer participants, making every volunteer even more valuable to the research process. Progress really depends on us being willing to participate.
A clinical trial can be sponsored (financed) by both noncommercial stakeholders (like hospitals, universities, non profits etc) and commercial companies (industry).
OIFE provides advice from the patient perspective to both commercial and non commercial stakeholders who are planning clinical trials in OI.
Where can I find ongoing clinical trials?
The best way to find out about studies in Europe, is to subscribe to the OIFE Magazine, where we include announcement from trials that are recruiting. To find out about more information on specific clinical studies, you can also go to euclinicaltrials.eu and search for “osteogenesis imperfecta” in the “Condition or Disease” field.
The best way to hear more about potential clinical studies, if you live in the USA, is by enrolling at the OI Registry. People from outside the USA can also join the OI Registry, but most of the studies announced are recruiting in North America. To find out about more information on specific clinical studies, you can also go to Clinicaltrials.gov.
OIFE collaborates with researchers
OIFE does not provide research grants, but we support researchers and international research projects in various ways. Click here to see what we can help with and important things to know before you contact OIFE and ask for our support.
OIFE collaborates with industry
OIFE collaborates with different pharmaceutical companies and companies producing orthopaedic equipment. Click here to see what the OIFE can offer to industry.