OIFE believes that the voice of the people with OI and their families should be heard in all contexts where decisions about their treatment and services are being made. This is to ensure that our needs, concerns and expectations are properly addressed and considered. Individual people can be represented by persons from the “patient community”, which is an overarching term covering individual patients, patient representatives (including family and carers), patient advocates and patient organisations.

Patient involvement on an individual level

This is closely linked to the term’s health literacy, education and empowerment. The concept is about helping people with OI become more knowledgeable and allowing them to take control over their bodies, their disease and treatment.

Shared decision-making is the process by which a clinician and a patient jointly make a health decision after discussing options, potential benefits and harms, and considering the person’s values and preferences.

The key is the recognition that in every medical or health decision, there are at least two sources of expertise: The clinician has had lengthy and extensive medical training; but only the person with OI knows his/her attitude to risk, how the illness is experienced in his/her particular social circumstances, and his/her values and preferences.

OIFE and our member organizations play an important role in educating the OI community about OI and its implications, to empower people to take part in decision making about their own health. We do this by organizing webinars, family meetings, developing factsheets and spreading awareness on social media and other communication channels.

Who are the patients?

It’s very important to define roles and representativeness, when talking about patient involvement. Is involvement on an individual level, a service/project level or a system/governance level?

Sometimes advice from a new and inexperienced patient, parent or a child is needed.

Other times a person, who can represent a larger group and speak on behalf of many, is needed (a patient representative). A patient representative can sometimes be a person with OI, a parent or a staff member, depending on which experience is needed.

When a person or organization is acting as a patient representative on behalf of a group of people, it is important to ask how they have gathered information:

  • From own individual experience
  • From individual patient stories, review of peer groups on social media, one to one discussions with patients, focus groups, surveys, interviews, documentation of clinic visits, published or unpublished research

It is also important to check how representative the findings are related to the larger community or minority groups, if this is relevant.

In principle, people with a collective experiential knowledge, should be preferred as patient representatives in favor of people who only have knowledge based on their own individual experience.

Sometimes a project needs the experience of a patient expert, which is a person who has undergone extensive training to learn about, their diagnosis (for instance at scientific conferences), research & development or about the healthcare system and policy work (registries, ERNs etc).

Sometimes advice from a patient organization who knows the international OI-community is needed (key opinion leaders). Patient organizations can also assist other stakeholders with a broader perspective by collecting aggregated data on unmet needs and patient priorities.

Usually, the last examples are when OIFE comes into the picture, but we can also offer assistance to find people in all the roles described above.

Patient involvement on a service/project level

OIFE is a strong supporter of involving representatives of people with OI & their families in planning and implementation of services that involve them. This includes hospitals, centers of expertise, policy making and development projects on a European or national level.

Patient involvement in clinical trials

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. It’s very important that patient organizations are involved at an early stage, to have real influence on a project or a clinical trial.

Instead of only talking about the pharmaceutical industry, OIFE prefer communicating with the different companies, preferably at a very early stage. This is to make sure that the unmet needs of people with OI have priority, that the outcome measures are relevant for people with OI and to provide input on the planning process of clinical trials.

We are transparent about our contact with different companies. All the grants we get from pharma companies are listed in our financial report, which is published on our website.

Patient involvement in regulatory affairs

OIFE is represented in different kinds of European regulatory affairs. This includes:

  • European Medicines Agency
  • EUNetHTA

To ensure impartiality/neutrality, the people who represent us in processes regarding regulatory affairs (EMA, EUNetHTA and National HTA bodies) cannot be involved in any of our advisory activities with commercial companies & commercial clinical trials. It’s therefore very important that both OIFE and the national organizations keep track of which people who represent us where. See this guideline from IMI about conflict of interest for more information.

And check out this 2,5 minute long educational video about what health technology assessment is and why it brings more challenges in rare vs. common health conditions.

Access to new and potential treatments

There are many different treatments being investigated for OI at the moment. Some are in a preclinical phase (animal research) and some are in clinical trials (where people take part in the trial): Stem cells, gene therapies and different pharmaceutical products. Decisions on market access are done on a European level.

Decisions about policy, politics and reimbursement in each and every European country, are mostly made on a national level. We therefore need to make sure that knowledge on regulatory processes, health technology assessments and advocacy are present on a national level, among our member organizations.

We also need data/documentation about the current situation for people OI, preferably for each and every country. This kind of data is called real world evidence (RWE) and patient experience data (PED).

Educational opportunities for patient experts

There are many different educational opportunities for people who want to get involved as patient representatives in policy work and/or research & development. We mention the two bigger stakeholders organizing educational activities:

Other useful resources

See also the project Reasonable Legal Agreements between Patient Advocates and Pharmaceutical Companies from WeCan.


Watch Yann Le Cam from EURORDIS explain why empowering and educating patient advocates is important below.

For more expansive information on patient involvement, download the guidelines from EMA and EURORDIS below. 

Are you interested in learning more about patient involvement or being a patient representative for OIFE? Please contact office@oife.org

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