Participating in medical research can be a rewarding experience for adults and children. When families have special medical needs, research activities may be particularly important because families become involved in the latest diagnostic or therapeutic interventions that may be available for their medical condition. In many uncommon or rare medical conditions, no proven effective therapies may be available. In some genetic conditions, a persistent hope among both affected families and dedicated medical investigators is that the identification of the causative gene will provide insight into the mechanisms of the medical condition and thus offer new pathways for therapy for what might be untreatable complications.
We frequently receive requests from medical and dental investigators for access to our the NFED members to solicit their participation in research activities. Our Scientific Advisory Council (SAC) screens carefully each research proposal that requests access to the NFED members and its compliance with the Federal regulations for the ethical conduct of human research at the institution sponsoring the research, whether it is a company such as a pharmaceutical corporation or an academic institution such as a medical or dental school. If the SAC approves a research proposal, we will notify our families that these research programs are available for participation.
Ectodermal Dysplasia: Type Unknown
We are launching a research study to help individuals get a diagnosis for the specific type of ectodermal dysplasia that affects them. Many individuals have a diagnosis of ectodermal dysplasia but do not know which of the 180+ types they have. Right now, registration for the study is closed but will reopen in the near future.
A Personal Decision
The decision to participate in any research project is a personal one. As a research participant, you must give your consent (permission) voluntarily for yourself or permission for your child or ward to volunteer for research activities. Any decision to participate should be based on your thorough understanding of the rights and responsibilities that you and your family have as research participants. You should ask several questions of both yourself (and family) and the investigator(s) before you decide to participate, because you become, in effect, a partner in these research efforts.
- Why are you being asked to participate in a research protocol?
- What is the importance of the research to your medical condition and how does it meet your special needs?
- What are you being asked to do and how long will you be required to participate?
- Will you be able to withdraw, that is, stop your participation at any time without endangering yourself or your family?
- What are the relative benefits and the potential risks or hazards to your participation?
- What costs will you, your family, or your insurance company incur?
- Will you be reimbursed for your expenses or time or travel?
- Will you be able to ask questions at any time during the study if you do not understand the various steps in the research?
- Will you be given the results of your research-related tests, and if so, is the information something you want to know? Will you be able to understand the implications of those results, or may you need some counseling to help you to interpret them?
- What will happen to you or the medical or research material (records, documents, blood or skin or tissue samples) that you provided, if you change your mind and withdraw from the research activities?
These latter questions are particularly important if you choose to participate in genetic research studies. Genetic research studies often provide a sense of relief for families because they finally “know” the name and the cause of their medical problem. For other families, the knowledge that they have gained or the “finality” of their search may lead to psychological distress or social stigmatism, may limit their employment opportunities, or may reduce their access to health or medical insurance. All these issues are important considerations when you decide to participate in research projects.
You are the best advocate for both yourself and for your child. Medical research is an important, if not essential, component of health care for which everyone should be willing to advocate. Being informed and acting upon your best judgments offer the most promising approach to improving the health and quality of life for your families with ectodermal dysplasias.
Contact us if you have questions or are interested in any active research projects.