When pharmaceutical companies begin the journey of developing a drug for F A, one of the first questions asked is “Where are F A patients located around the world?” The answer to this question is important because:
- Pharmaceutical companies want to know where patients are located when they plan for clinical trials. They also need this information for eventual drug distribution, so they are aware of the regulatory processes in each country where patients are treated.
- Pharmaceutical companies will need to plan for physician education about drug availability in any country where F A patients are being diagnosed and treated.
There are an estimated 15,000 individuals affected with F A worldwide. Currently, the registry includes only 1/3 of all F A patients globally. The registry provides a way to notify F A patients about upcoming clinical studies and trials and avoids delays in the drug development process.