Patients & Families
Rare Diseases
It’s estimated there are more than 7,000 different types of rare diseases. In the U.S., a rare disease is defined as a condition that affects fewer than 200,000 people. The exact cause of many rare diseases is unknown, however the majority are thought to be genetic, directly caused by changes in genes or chromosomes, while others may occur randomly. Rare diseases are estimated to affect 25 to 30 million Americans. While individual patient numbers of specific diseases may be small, the community as a whole is large, and MeiraGTx is proud to be a part of the millions of people working towards finding cures to these currently untreatable diseases.
Rare Disease Patient Resources
For additional information about rare diseases, please visit the following organizations to find resources and support:
MeiraGTx is focused on developing potentially curative treatments for patients living with serious diseases. Our goal is to provide access to our product candidates at the appropriate time and in the correct manner for patients.
Expanded access refers to the use of a product candidate outside the auspices of a clinical trial when the primary purpose is to diagnose, prevent, or treat a serious condition in a patient for which no alternative treatments are available. This is different from a clinical trial, where more comprehensive safety and efficacy data are collected. Expanded access use is usually requested by physicians on behalf of their patients for whom participating in a clinical trial is difficult or impracticable. We understand the need for expanded access programs, and we recognize the importance of having an appropriate policy.
A number of factors consistent with the policies of the U.S. Food and Drug Administration (FDA) and guidelines of international regulatory agencies should be taken into account when considering expanded access. They include:
- The illness must be serious or life-threatening
- There are no other viable treatment options (such as approved products or enrolling clinical trials)
- There is sufficient evidence that the potential benefit to the patient would likely outweigh the potential risks based on safety and efficacy information available at the time
- Ability to provide a product in a fair and equitable manner, so that there is adequate manufacturing capacity for ongoing programs
- Whether granting expanded access would potentially compromise the scientific validity of broader development programs, or interfere with or delay the conduct of current clinical trials or regulatory submissions designed to make the product candidate available to many more patients
At this time, MeiraGTx believes that participation in one of our clinical trials is the most appropriate way to access our product candidates. If you have additional questions, please speak with your physician or contact patients@meiragtx.com. We anticipate acknowledging receipt of requests sent to this e-mail within five business days.
Pursuant to the 21st Century Cures Act, MeiraGTx may revise this policy at any time.
Dated: February 2021
