Expanded Access Program (EAP)
Ondine Biomedical is focused on improving patients’ lives by identifying, developing and developing innovative products that address clinically unmet needs. To do this, we conduct clinical trials to assess the safety and efficacy of investigational medicines, and the data generated from these trials allows us to obtain the necessary approvals from regulatory bodies such as the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA), in order to permit broad access to such medicines.
However, we also recognize that in some extreme circumstances, when participating in a clinical trial is not possible, patients facing serious or life-threatening conditions may seek special access to investigational medicines. Consistent with applicable laws and regulations, Ondine may provide patients with access to investigational medicines through Expanded/Early Access Programs (EAP’s) prior to regulatory agency approval of the product, when we believe there is sufficient evidence of the safety and effectiveness of the investigational medicine to support its use in the particular circumstance. These types of access programs can also be known as Compassionate Use, Pre-approval Access, Named Patient Access and Emergency Use. Wherever possible, we believe that it is preferable that such Expanded Access occurs within the context of a clinical study. Ondine complies with good clinical practices and takes steps to ensure the quality and integrity of our clinical trials and to minimize risks to current research participants and future patients. For a list of clinical trials currently recruiting patients, please visit www.clinicaltrials.gov .
Types of Access Programs
In general, there are two ways that Ondine can grant Early/Expanded Access to an investigational medicine. The type of program available may be dependent on local laws and regulations.
Named Patient Access (Single Patient IND in the U.S.) – where a qualified healthcare professional requests an investigational medicine for use for a single patient because, in their judgement, currently available therapies are not satisfactory. Provision of the investigational medicine by Ondine may or may not require approval by FDA as well as an Institutional Review Board.
A Cohort Type Program – where Ondine can make a promising investigational medicine available for a specific condition to a defined group of patients. Provision of the investigational medicine to the cohort program by Ondine may or may not require approval by FDA as well as an Institutional Review Board.
Ondine considers many factors when evaluating a request for named, single patient access to an investigational medicine, such as the strength of the clinical data; the benefit-risk profile; the potential impact on the clinical development program; the phase of development; the country-specific regulations; and probability and timing of regulatory approval.
In determining whether named patient access is appropriate outside of a clinical trial, the criteria that Ondine evaluates include the following:
- Ondine has sufficient evidence of the safety and effectiveness of the investigational medicine to support its use in the particular circumstance.
- The disease is serious or life threatening.
- There are no available beneficial alternative treatments or patients have exhausted all other therapies.
- A clinical study is not available, either because patients are ineligible or because they have no access to a trial.
- The efficacy and safety data available at the time are of sufficient strength to determine whether the benefit to patient would likely outweigh the potential risks.
- Access is compliant with local rules and laws.
- The request must be made by the patient’s treating physician, unsolicited by Ondine Biomedical or any other individual or organization.
- The patient’s treating physician has determined that there are sufficient safety and efficacy data, including dosing information, to support the use of the investigational medicine, that there is no comparable or satisfactory therapy available to treat the patient’s disease and that the probable risk to the patient from the investigational medicine is not greater than the probable risk from the disease itself.
Ondine will consider the evaluation criteria in determining whether to offer named patient access; however, Ondine cannot make a guarantee that named patient access will be available, and, even if a named access program is offered, Ondine cannot make a guarantee that the investigational medicine will be available to a particular patient.
Decisions regarding potential named patient access to investigational medicines can only be made, in certain circumstances, after in-depth discussion between Ondine’s clinical teams and the patient’s qualified healthcare professional and in line with required regulatory approval, if applicable.
Ondine is committed to a fair and impartial evaluation of each request for access to our investigational medicines. Therefore, all decisions are based on clinical circumstances. Whenever possible, patients will be referred to ongoing clinical trials as the primary way to access investigational medicines.
For more information from FDA about expanded access programs, click here.
For individual patients in the United States, treating physicians can submit requests to: firstname.lastname@example.org
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