Expanded Access
At Cellphire, our goal is to improve and save the lives of patients by developing next generation therapies. All investigational therapies must first go through clinical trials and be approved by the U.S. Food and Drug Administration (FDA) to ensure the therapy benefits outweigh any risks for the intended patient population before being made commercially available. In most cases the only way patients can access investigational therapies or drugs (INDs) is to participate in a clinical trial. We understand there may be situations where seriously ill patients may not be eligible or able to participate in clinical trials. In these circumstances, Cellphire will consider providing a requesting physician with expanded access (“compassionate use”) to an IND outside of a clinical trial for the treatment of an individual patient when certain conditions are met. These conditions include the following:
- The patient has a serious or life-threatening illness or condition and is either no longer responsive to or no longer able to tolerate any available treatment option and does not qualify for the current clinical trial;
- The investigational drug is in active clinical development with sufficient data available to 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;
- 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
- Adequate supply of the investigational drug is available.
Cellphire Policy: Expanded Access Use of Thrombosomes®
Overview
This policy regarding Expanded Access to Cellphire’s Investigational New Drug Thrombosomes (TBX), an activated freeze-dried platelet, is aimed at addressing the limited availability of platelets that are the result of the COVID-19 pandemic. Blood products are in critical need during this time frame and blood banks are faced with extremely limited supplies. In the situation where a patient presents with WHO Grade 3 bleeding or higher and is in critical need of platelet/blood products, this Policy facilitates the use of TBX to treat the acute bleed and possibly mitigate the risk of additional critical bleeding. The benefit of the use of TBX in this critical set of circumstances is believed to outweigh the risks of the use of this investigational product.
Policy Statements
In cases where a clinical trial with Thrombosomes is not an option, Cellphire may elect to provide the Sponsor/Investigator expanded access. Treating Sponsor/Investigators and patients should note that clinical safety and efficacy of investigational products has not been fully established, so all potential risks and benefits should be carefully evaluated before seeking expanded access to this product. It is envisioned that requests could be made by Sponsor/Investigators as either an Individual Patient IND or Emergency Use Individual Patient IND. For more information on submitting requests to the FDA, click here. This policy is aimed at addressing both types of requests. Cellphire will consider requests for access to TBX, according to internal Cellphire SOPs and as permitted by applicable law, in very specific circumstances, when certain criteria are met. For more detailed information, see Cellphire’s full policy here.
Requesting Access
Sponsor/Investigators seeking single patient expanded access (either emergency or non-emergency use) to TBX on behalf of their patient should call Cellphire at 301-545-2528 or submit an inquiry to expandedaccess@cellphire.com. The request will promptly be addressed, generally within 24 hours of receipt by a member of either the Cellphire Clinical Affairs or Clinical Study Teams. Sponsor/Investigators should provide a written summary of the specific medical circumstances that require single patient expanded access and the need for treatment with TBX, which briefly includes responses to each of the eligibility requirements listed above. It should be noted that there is no guarantee that an expanded access request will be granted by FDA. Sponsor/Investigators who receive a TBX dose for their patients through the expanded access program must comply with all applicable FDA regulations, contractual conditions, safety reporting required by FDA, and protection of intellectual property. This policy is subject to change. Cellphire will revisit the policy periodically and amend it as appropriate. This policy is not a guarantee of access to any of Cellphire’s investigational products.
