Compassionate Use, Expanded Access, and Off-Trial Use of Treatments for Medulloblastoma
Upon a diagnosis of medulloblastoma, a standard treatment protocol is first carried out (typically surgery, radiation, chemotherapy). If standard treatment doesn’t work or relapse occurs, a clinical trial might be an option. Phase 2 and phase 3 trials might not offer a cure for the cancer itself, but they may help resolve or provide relief from side effects and improve quality of life.
If these options don’t work, many patients find themselves looking into other options. This could include phase 1 trials or even compassionate use opportunities, where little to nothing may be known about the compound being tried.
If you find yourself in this situation, there are several pathways to consider, including compassionate use, expanded access, and off-trial use of drugs. Below we’ll explore the differences between them, the limitations of each, and a provisional pathway that may develop in the coming years.
Compassionate Use of Medulloblastoma Treatments
Compassionate use is a way for a specific patient to get access to an experimental treatment. It is only for serious or life-threatening diseases or conditions for an investigational medical product when there are no other comparable or satisfactory therapies available.
These therapies have not been formally approved for use by the FDA, but are usually in the investigative stage through other trials. As with any therapy not formally approved, there can be serious side effects, even fatal, and the effectiveness of the therapy isn’t known.
Often the path to compassionate approval can be lengthy and difficult. In order to gain approval, the patient must accomplish the following:
- Approval from a Licensed Physician – In any compassionate use case, other than the initial request of the patient, the physician is the most integral to this process. They must have explored other treatment options and work with industry, the FDA, and an Institutional Review Board (IRB) to gain approval for compassionate use of the therapy. This is done in writing and may require updates and revisions. The physician must then oversee the administration of the therapy, general care, and reporting of responses.
- Approval of Industry – In any compassionate use case, the company producing the therapy must also approve of its use. They also may be required to participate in the reporting process.
- Approval of an IRB – The IRB’s role is to protect the rights of patients participating in any study. This is accomplished with approving the plan to administer the therapy, the consents, and communication with the patient to ensure that they understand the inherent issues of the treatment path.
- Approval of the FDA – The FDA must also review the request and determine if compassionate access is allowed.
Expanded Access of Medulloblastoma Treatments
The term expanded access is often used interchangeably with compassionate use, but it’s often used to refer to multi-patient programs of compassionate use.
Although many of the approval thresholds still must be upheld, many formal expanded access programs recognize a group of patients with the same diagnoses and set certain criteria for those who will be allowed to participate—much like a clinical trial.
In these cases, while the data still needs to be captured for patient safety, it may not be used for ultimate therapy approval with the FDA.
Expanded access programs are typically set up when:
- Clinical trials are limited,
- The type of disease is life-threatening, and
- There may be some outside support to help cover the costs of the therapy and the reporting functions.
Expanded access programs may also be used in cases where a drug may be approved for one class of patients but not another. This is often the case for certain age groups such as children when an adult version of the drug has already been approved.
Off-Trial Use of Medulloblastoma Treatments
One option that isn’t often explored is off-trial use of a therapy. Cases where this might be considered include the following:
- When a treatment has completed a phase 1 or phase 2 trial, but the patient may not qualify for an existing trial due to progression of the disease, use of another drug, or even certain functional limits.
- When a trial temporarily halts recruitment as it moves between phases. In these cases, the treating physician may seek approval for an off-trial use of the treatment. The approval may be slightly easier than compassionate use because the therapy may already be approved for limited use. Data might not be collected for drug approval, but will be reviewed for patient safety.
It is important that patients discuss this option with their doctor. Often it may be an easier, but overlooked, path to treatment.
Provisional Use of Medulloblastoma Treatments
While not a current treatment pathway, provisional use is a proposed option that is the subject of the Promising Pathway Act. This act would create a priority review for drugs intended to treat, prevent, or diagnose serious or life-threatening diseases or conditions. This is different from compassionate use in that it would provide a path to quickly get access to the drugs that are needed when time is of the essence.
The bill seeks to recognize a new status with the FDA taking compassionate access to a provisional approval for two years or less. This would allow groups of patients to participate and gain access to new therapies that are reasonably believed to be safe. These patients would then be monitored for safety, and the data would be recorded in a third-party registry for future approval processes.
If passed, this bill would provide access to more treatments, innovative drugs and biologics, and hope for the future.
To learn more about this bill, visit https://www.congress.gov/bill/117th-congress/senate-bill/1644/text. To help with the approval of this act, please contact your legislators below:
It is important to note that each of the above pathways do not deal with the financial aspect of participation and often are not covered by typical health insurance plans. Still, they seek to open up options for the patients and are of particular significance to those fighting medulloblastoma.