sThis is probably the greatest time to be alive, if you are a patient with a rare disease. Why? Not only are there more diagnostic tools and more advances being discovered every day, but you also have the opportunity to become involved in finding solutions and new treatments.
If you are a patient with a rare disease, or a parent with a child that has a rare disease, you may or may not be aware that pharmaceutical companies, biotechnology companies, and major regulatory authorities are seeking to engage you in the drug development process. If you would like to be involved in the process of finding new treatments for your disease, how should you go about involving yourself? Your first point of call should be the organization that represents your patient group. In reality, there may be several such organizations. If the patient groups are not interested in engaging with the pharmaceutical industry and regulatory authorities, the next step would be to organize a group of patients that feel the same way that you do. This may involve forming a non profit or charity, but not necessarily. You may organize yourselves as a patient support group. This may simply involve setting up a Facebook support group.
When you are reasonably clear on your goals and objectives, obtain information about the drug development process. At Putting Rare Diseases Patients First!® our goal is to provide you with the information that you need so that you can engage effectively with pharma/biotech/regulatory authorities. Sign up to receive information about Webinars etc. at http://www.prdpf.org. Your contact information is kept in the strictest confidence, and is never shared.
When you feel reasonably well informed, the next step will be to contact the companies that are engaged in developing treatments for your disease, and the regulatory authority in your region. Ensure that they are aware of your group, and your interest in being a part of the dialogue to find solutions for patients with your rare disease.
You will be able to make a contribution in many areas, including the following:
- Setting up Patient Registries
- Design and conduct of Natural History Studies.
- Identifying the needs of patients
- Designing the ideal treatment for the disease.
The pharmaceutical industry and regulatory authorities should welcome your interest and contact. Unfortunately, individual companies are not the easiest places to contact. When you call the companies, unless you have identified a named individual, you will not be transferred to anyone. Why? There are concerns around confidentiality and security.
Becoming more patient friendly is going to be crucial if companies are going to be successful in engaging with patients. One way to move past the gatekeeper, may be to ask for the medical information department. Hopefully, the company medical information department will then be able to pass on your details to the person responsible for patient engagement.
In the Pharmcast podcast (see below), Dr. Speid speaks to pharmaceutical companies about how they can better engage with patients with rare diseases to find new treatments. Click on the following link to go to the podcast. Do spread the word among your patients, family members, and colleagues.
Lorna Speid, Ph.D.
President and Founder of Putting Rare Diseases Patients First!®