Phentolamine opthalmic solution
Opus Genetics development resources are focused on conducting clinical studies required by regulatory authorities to fully answer important scientific questions about the potential risks and benefits of our investigational products, and to obtain regulatory approval.
Opus Genetics is committed to making investigational products available to seriously ill patients who have exhausted other treatment options. A treating physician, who is able to comply with the requirements that are stated in this document, may request information about how to apply for access to Opus Genetics investigational products by contacting Opus Genetics.
Patients with serious or life-threatening diseases or conditions seek access to promising investigational medicines outside of a clinical study setting. The associated provision for requesting access to an investigational medicine is called ‘Expanded Access’ but is also known by other terms such as compassionate use” or “hospital exemptions”, “early access”, “emergency use”, “right to try access”, patients who seek access to investigational medicines outside of an established clinical study and prior to Health Authority marketing authorization may wish to do so because standard treatments have failed, they cannot tolerate already approved medicines, or because they are unable to participate in a clinical study.
The policy below sets forth the general guidelines that Opus Genetics will apply to its Expanded Access Program to ensure that requests for access to our investigational medicines are considered and processed in accordance with applicable legal and regulatory requirements, as well as in alignment with principles consistent with the Opus Genetics’ policies and procedures. The Policy is applicable globally and across all therapeutic areas. Further information about Opus Genetics’ clinical trials is available at Clinicaltrials.gov.
This Policy is designed to guide fair and equitable access decisions while maintaining Opus Genetics’ obligation to carry out the clinical development programs necessary to assess safety and efficacy of our investigational products. The guiding principles for granting access to individuals outside the clinical development programs build on our Guiding Principles for Expanded Access Programs and should consider:
•What actions by Opus Genetics will help ensure all patients are treated fairly and equally;
•Whether sufficient clinical data exists for a particular disease or condition to anticipate that the potential benefits outweigh the potential risks of the investigational medicine for that patient;
•Are the potential risks of treatment with the investigational drug reasonable in the context of the patient’s disease or condition being treated;
•Is there another investigational drug available with Opus Genetics or at another company that is more suitable;
•How will patients be selected for treatment;
•Whether a clinical trial participant who has had clinical success during enrollment in a clinical trial of investigative medicine will have continued access to the investigational medicine after completion of, or withdrawal from, the trial (i.e., bridging to commercial availability);
•Whether the granting of an individual request, given the current epidemiologic understanding of the number of patients who would have a similar clinical profile, would unduly burden the company with a magnitude of similar requests which should, by principles of justice and fairness, be provided to these other patients;
•How will patients be taken off treatment if the medication makes it to market or does not make it to market; and
•Whether it is feasible for the company to provide the required access for the duration of the patient’s necessity once any access is provided.
•Opus Genetics should evaluate, prior to the initiation of a program and periodically throughout a program, given the number of individual requests, whether a group or protocol-driven cohort program should be initiated.
1.1 Any use of a Opus Genetics investigational product outside a clinical study in a country must be in accordance with local laws and regulations governing such programs, including Opus Genetics policies and procedures.
1.2 In general, where permitted by local regulation, the investigational product supplied via Expanded Access may no longer be provided by Opus Genetics when it becomes available via the local healthcare system.
1.3 Opus Genetics may decide not to provide an investigational product under this policy if the Company does not intend to market the product in the country.
1.4 To qualify for expanded access under this procedure, the product must be under investigation in one or more clinical studies, have sufficient evidence to expect that the investigational product will have an acceptable safety profile for the intended patient population, and assurance that the provision of the investigational product under this procedure will not interfere with or compromise the clinical development of the product.
1.5 Patients must be eligible for access to an investigational product, and must therefore meet the following criteria:
• Suffer from a serious or immediately life-threatening disease or condition;
• Have undergone appropriate standard treatments without success and no comparable or satisfactory alternative treatment is available or exists to treat the disease or condition;
• Are ineligible for participation in any ongoing clinical study of the investigational product, which includes lack of access due to geographic limitations;
• The patient has a disease for which there is sufficient evidence of a projected benefit from the use of the investigational product and the benefit outweighs the known or anticipated risks;
• There is adequate information to support appropriate dosing for a special population patients such as pediatric, elderly, renal or hepatic disease, etc; and
• Any other pertinent medical criteria for access to the investigational product, as established by the Opus Genetics clinically or medically responsible individual.
1.6 This policy is posted on Reagan-Udall’s public website (Expanded Access Navigator) and apply to all Opus Genetics employees.
Lastly, Opus Genetics may consider closing a program for various reasons, including but not limited to:
•Commercial availability of the medicine for a particular need or condition, at which point it would be more broadly available to these patients in need;
•A definitive negative regulatory decision or a company decision to not proceed with clinical trials or commercialization for a particular disease or condition. In this instance, patients on expanded access treatment at the time of such a decision may continue to access therapy until disease progression;
•New information about the activity or safety of a medicine that could substantially change its benefit/risk profile; or
•Unexpected, limited product supply or other manufacturing issues.