To enable your patients to participate in our trials, please provide more information about your practice and center by filling out the interest form below.
- Expanded access refers to the use of an investigational medicinal product (i.e., one that has not been approved by the U.S. Food and Drug Administration (FDA) or other applicable regulatory authority) outside of a clinical trial. This is different from a clinical trial, where more comprehensive safety and efficacy data are collected. 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 FDA and other regulatory agencies’ guidelines should be taken into account when considering expanded access. They include:
- The illness must be serious or life-threatening with no other satisfactory 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 available safety and efficacy information.
- 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 current clinical trials or regulatory filings designed to make the therapy available to many more patients.
- At this time, Proteostasis Therapeutics believes that participation in one of our clinical trials is the most appropriate way to access our investigational therapies. If you have additional questions, please speak with your physician or contact PTIClinicalTrials@proteostasis.com. We anticipate acknowledging receipt of requests sent to this email within five business days.
- In line with the 21st Century Cures Act, Proteostasis may revise this policy at any time. This website and policy will be updated with a hyperlink or other reference to the clinical trial record containing information about expanded access after such record becomes active.