Expanded Patient Access to BioXcel Therapeutics’ Investigational Medicines
BioXcel Therapeutics is committed to developing next wave of medicines across neuroscience and immuno-oncology. We are committed to bringing these innovative investigational medicines to patients as quickly as possible in a fair and ethical manner, and we believe in collaborating with patients, families, patient advocacy organizations, physicians, researchers, and regulatory authorities to accomplish this goal.
Evaluation of an investigational drug in clinical trials, which are designed to gain an understanding of the safety and efficacy of the drug in a specific population, is the optimal way to collect the information necessary to enable regulatory authority review and, ultimately, to bring the drug to the entire patient community. Participation in a clinical trial is also the best way for an individual patient to access an investigational drug for his/her disease. At BioXcel Therapeutics, our goal is to enroll and carry out robust, thoughtfully designed clinical trials as expeditiously as possible in order to support regulatory approvals and provide the broadest possible access to patients who might benefit from our investigational medicines.
BioXcel Therapeutics understands that in some cases there will be patients with a serious and immediately life-threatening disease for which no satisfactory treatment alternative exists, and for whom it is not possible to participate in a clinical trial. In these very specific circumstances, use of an expanded access program (known as compassionate use programs in certain jurisdictions) may be an option. Decisions about expanded patient access to our investigational medicines are made after considerable thought with the safety and well-being of the patient our foremost priority.
Decision Criteria for Considering an Expanded Access Program
An expanded access program for a specific investigational drug will be considered if BioXcel Therapeutics determines that all the following criteria are met:
- There is sufficient evidence, based on available safety and efficacy data, that the potential benefit to the patient would justify the potential risks of treatment and those potential risks are not unreasonable in the context of the disease or condition treated
- The disease or condition is serious or immediately life-threatening and there must be no other comparable or satisfactory alternative treatment options available to the patient
- The investigational drug is the subject of an active clinical development program and is not approved in any indication in the country concerned
- An adequate supply of the investigational drug exists to perform necessary clinical studies as well as to provide expanded access to patients who do not have alternative treatment options
- Providing the investigational drug will not delay, impede or otherwise compromise the ongoing clinical development of the investigational drug or the submission of any regulatory filings designed to make the investigational drug more broadly accessible
- There are definite plans to submit a marketing application and commercialize the drug in the region
- The program is compliant with local rules and laws
- The program must be discontinued as soon as feasible if and when approval of the drug is obtained in the country in which the expanded access program is being conducted
How To Request Early Access
A request for early access to a BioXcel Therapeutics investigational medicine must originate directly from a licensed physician on behalf of his or her patient and should be forwarded to BioXcel Therapeutics at firstname.lastname@example.org. The requesting physician must agree to obtain appropriate regulatory and ethics committee approvals, as applicable, and to comply with all other safety, monitoring, reporting, and patient consent requirements defined by BioXcel Therapeutics. BioXcel Therapeutics anticipates acknowledging receipt of requests for early access orally or in writing within five (5) business days. BioXcel Therapeutics is committed to evaluating all requests for expanded access in a fair and equitable manner.
All requests will be evaluated by medical professionals and decisions will be made based on scientific evidence available to the company at the time of the request.