Webinar on Drug Development For Patients with Rare Diseases

December 10, 2014

The Seven Things You Need to Know about the Drug Development Process For Rare Diseases

You are cordially invited to a free WEBEX (seminar) on the rare diseases drug development process. This seminar is being organized by Rare Diseases Patients First!, a non-profit that provides unbiased information to patients with rare diseases. We know that many of you are very interested in this topic because it directly impacts the treatments that you have access to, or the treatments that your sick child has access to.

We know that patients with rare diseases are very busy. This is one hour that will be an effective use of your time.

Date: 10th December 2014

Time: 8 am PST (US West Coast); 11 am EST (New York Time); 4 pm Local Time in the United Kingdom; 5 pm Local Time in Europe

Duration: 1 hour

Format: A presentation will be followed by an interactive question and answer session. This is your opportunity to have your Questions about how new drugs are developed answered.

Registration: Please email Dr. Speid at [email protected] with RDPF! In the subject line. She will send you the WEBEX login information.

Cost: FREE

The development of new treatments for rare diseases is governed by regulatory processes. This process is complex, and has been put in place to protect patients. You will find out what it the process consists of, and how you can interact effectively with it.

During this first WEBEX seminar, you will find out:

  • How drugs are developed for rare diseases
  • Some typical designs for rare disease clinical trials
  • The type of safety information that is collected during clinical trials
  • How you can contribute to the clinical trial process
  • The informed consent process and what it involves
  • How to identify conflicts of interest as you interact with the clinical trial process
  • Some considerations for clinical trials that involve children
  • A list of questions to ask your clinical trial investigator at different stages of the clinical trial process




I’ve been diagnosed with a rare disease. What do I do now?

When a patient or loved one is diagnosed with a rare disease, there is sometimes a sense of relief that at last a name has been given to the symptoms that have been experienced, often for years. This is usually quickly followed by a hunger for information and direction on what to do next. There are approximately 7000 rare diseases. There are only about 200-400 treatments for these rare diseases. Many of these treatments are suboptimal. This means that most patients with a rare disease are being treated symptomatically or are without any real treatment at all.

In order to find new treatments for rare diseases it is necessary to conduct research and clinical trials. The process of new drug development is a complicated one. The pharmaceutical industry is known to have a relatively poor rate of successful new drug discovery and commercialization. This rate of success has been getting worse over the years. The fact is that although our knowledge about diseases is increasing exponentially, the process by which drugs are developed is becoming more, not less challenging. There are many reasons for this.

Patients with rare diseases are often desperate for new and effective treatments. There has been a recent trend for vocal patient groups to demand that the standards of drug development are relaxed to allow access to effectively unproven treatments. This demand for new treatments is especially vociferous when rare diseases lead to shortened lives in children. Is this a good trend? What can patients with rare diseases do to make the drug development process more productive and effective?

Rare Diseases Patients First! is an organization that seeks to provide free and unbiased educational content about the drug development process to patients with rare diseases, and parents of sick children with rare diseases.

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