Praxis Precision Medicines (Praxis) is dedicated to developing new therapies that have a positive impact on patient health, and to serving patients, patient families and patient communities through education, empathy, and awareness. Consistent with the Praxis mission to bring innovative medicines to patients with serious or life-threatening conditions, we are focused on enrolling and conducting the clinical trials necessary to assess the safety and efficacy of our investigational therapies, which are requirements for potential regulatory approval. We believe this is the most scientifically rigorous approach to make our medicines available broadly to patients as quickly as possible.
We are privileged to collaborate with clinical investigators and with patients who participate in our clinical studies to develop new, safe and effective therapies. At the same time, we understand that there are patients with serious or life-threatening conditions who will not be eligible for our clinical trials and may not have options for alternative therapies, including investigational therapies in trials being conducted by other sponsors. In these circumstances, Praxis will consider providing a requesting physician with pre-approval access to a specific Praxis investigational drug, for the treatment of an individual patient outside of a clinical trial, when certain conditions are met.
These conditions include the following:
- The patient has a serious or life-threatening condition and is either no longer responsive to or no longer able to tolerate any available treatment option, and no comparable or satisfactory alternative treatment is available to treat the condition;
- The investigational drug is in active clinical development with sufficient data available to and determine an appropriate dose and schedule for the patient’s specific condition;
- A benefit-risk analysis, based on both the available clinical data as well as the requesting physician’s assessment of the individual patient’s condition and history, supports making the investigational drug available;
- The patient is ineligible for participation in any ongoing clinical study of the investigational product, which includes lack of access due to geographic limitations;
- Making the investigational drug available will not negatively impact or delay the conduct of clinical trials or regulatory review or approval of the investigational drug for broader patient access; and
- There is adequate supply of the investigational drug.
We continually evaluate the benefit-risk profile of each of our investigational drugs based on evolving clinical data. Each compound under development is different and the fact that one investigational drug is made available for the treatment of a particular patient does not mean it will be made available in response to other requests on behalf of other patients whose circumstances and medical histories may be similar, or that a different investigational drug will be made available under our policy.
Requests will be considered on a case-by-case basis. Praxis is committed to evaluating all requests in a fair and timely manner. All requests must be submitted by the patient’s treating physician. Praxis may require more detailed information in order to fully evaluate a request. The requesting physician must agree to obtain appropriate regulatory and independent review board / ethics committee approvals and comply with regulatory obligations, including obtaining patient consent, patient monitoring and safety reporting. Each request will be given careful consideration by Praxis, . Physicians seeking pre-approval access for patients with no alternative treatment options should submit their requests to [email protected]. We regularly monitor this mailbox and will use our best efforts to acknowledge each submitted request within five business days after receipt.
Information about Praxis clinical trials can be found on ClinicalTrials.gov.