Preliminary criteria and discussion points (from highest to lowest support)
| Criterion (%) . | Elaboration . | Discussion/decision . |
|---|---|---|
| Medical need/acuity (21) | Urgency will look different according to each disease site Each disease site will need to identify its own disease parameters What clinical conditions are minimum indications for CAR-T to be offered? Does the patient meet these indicators? Will those who are most ill be able to wait for manufacturing of cells? What are the clinical risks to the patient of delaying treatment (eg, requiring more chemotherapy to stay in remission and impact on prognosis)? How to determine reliably and consistently who is more ill? May include considerations of medical gestalt, including frailty, refractory disease, chemotherapy sensitivity, and presence of extramedullary disease Urgency does not stand alone as a determining factor; it will need to be considered along with other criteria (eg, medical benefit and safety) | “Medical need/acuity” was refined to “medical urgency” to reflect the relevant variable. We elaborated on this criterion to reflect the discussion about urgency being 1 consideration among others, and that it should not be the sole criterion by which to allocate spots. |
| Likelihood of benefit/tolerate therapy (19) | Medical benefit may look different for each disease site How is “benefit” determined (eg, length of life and quality of life)? Consider the likely duration of benefit for the patient and whether alternative treatment options exist (ie, life expectancy with CAR-T vs other treatment options/no other options) Curative potential Consider the “window of opportunity” for benefit: the longer a patient waits, he/she may be less likely to benefit and may miss the window for a good outcome | Refined likelihood of benefit to “medical benefit.” Rationale: removing quality of life from an understanding of benefit was believed justified because quality of life is subjective and would not be reliably externally assessed. “Medical benefit” should reflect considerations of curative and noncurative potential. Partners agreed that it will be difficult to decide between these groups of patients, but that those patients who are curative but can wait longer for CAR-T without compromising a good outcome could perhaps wait longer for a spot. |
| Safety/risk of complications (19) | Consider the clinical risks of receiving CAR-T (eg, death and major toxicities) Is the patient healthy enough overall to withstand treatment (eg, organ function, comorbidities, age, frailty, and performance status)? Consider the clinical risks to delaying treatment to the patient (eg, requiring more chemotherapy to stay in remission and impact on prognosis) | This was deemed an important criterion as is |
| Adherence to treatment regimen/compliance (11) | What is the likelihood that the patient will be able to comply with the requirements of treatment (eg, protocols and appointments and ability to travel)? | Comments were that this represents another potential to create disadvantage depending on reasons for nonadherence. Similarly, others thought this should not be a deciding criterion. |
| Social/caregiver support (8) | Does the patient have access to sufficient social/caregiver resources to optimize outcomes? | Comments were that this criterion is unacceptable, is potentially discriminatory, and will create disadvantages. The health system should support such patients. |
| Impact on other resources (8) | What is the impact on resource use (eg, having to use additional chemotherapy because of delayed CAR-T)? | There was concern that this disadvantages people who may require more treatment to get them to CAR-T |
| Length of wait (6) | Where all else is equal (ie, likelihood to benefit and likelihood to harm), a patient waiting longer should receive access before a patient who has waited for a shorter period | This criterion could potentially be a tiebreaker |
| First come, first serve (5) | How would we know who was first come so that he/she would be first served (eg, time of consult and time of the patient’s decision to proceed)? Potential to penalize patients who take time reaching a decision. | There was agreement that first come, first served was too arbitrary of a criterion for such an important decision as to whether or not to prioritize a patient to CAR-T. This criterion was subsequently removed from the list. This treatment option is so valuable, the selection process needs to be fine-tuned. |
| Exhausted all other treatment options (2) | CAR-T should be offered in the context of a range of treatment possibilities. What are the other options? How do their risks/benefits compare? | This criterion only had 1 vote in the poll, the lowest of any other criterion. Patients had concerns about this. Uncertainty regarding “What does no other treatment options mean? Available? Working?” “What if an Indigenous person chose to pursue non-Western healing, and did not exhaust all Western options?” |
| Criterion (%) . | Elaboration . | Discussion/decision . |
|---|---|---|
| Medical need/acuity (21) | Urgency will look different according to each disease site Each disease site will need to identify its own disease parameters What clinical conditions are minimum indications for CAR-T to be offered? Does the patient meet these indicators? Will those who are most ill be able to wait for manufacturing of cells? What are the clinical risks to the patient of delaying treatment (eg, requiring more chemotherapy to stay in remission and impact on prognosis)? How to determine reliably and consistently who is more ill? May include considerations of medical gestalt, including frailty, refractory disease, chemotherapy sensitivity, and presence of extramedullary disease Urgency does not stand alone as a determining factor; it will need to be considered along with other criteria (eg, medical benefit and safety) | “Medical need/acuity” was refined to “medical urgency” to reflect the relevant variable. We elaborated on this criterion to reflect the discussion about urgency being 1 consideration among others, and that it should not be the sole criterion by which to allocate spots. |
| Likelihood of benefit/tolerate therapy (19) | Medical benefit may look different for each disease site How is “benefit” determined (eg, length of life and quality of life)? Consider the likely duration of benefit for the patient and whether alternative treatment options exist (ie, life expectancy with CAR-T vs other treatment options/no other options) Curative potential Consider the “window of opportunity” for benefit: the longer a patient waits, he/she may be less likely to benefit and may miss the window for a good outcome | Refined likelihood of benefit to “medical benefit.” Rationale: removing quality of life from an understanding of benefit was believed justified because quality of life is subjective and would not be reliably externally assessed. “Medical benefit” should reflect considerations of curative and noncurative potential. Partners agreed that it will be difficult to decide between these groups of patients, but that those patients who are curative but can wait longer for CAR-T without compromising a good outcome could perhaps wait longer for a spot. |
| Safety/risk of complications (19) | Consider the clinical risks of receiving CAR-T (eg, death and major toxicities) Is the patient healthy enough overall to withstand treatment (eg, organ function, comorbidities, age, frailty, and performance status)? Consider the clinical risks to delaying treatment to the patient (eg, requiring more chemotherapy to stay in remission and impact on prognosis) | This was deemed an important criterion as is |
| Adherence to treatment regimen/compliance (11) | What is the likelihood that the patient will be able to comply with the requirements of treatment (eg, protocols and appointments and ability to travel)? | Comments were that this represents another potential to create disadvantage depending on reasons for nonadherence. Similarly, others thought this should not be a deciding criterion. |
| Social/caregiver support (8) | Does the patient have access to sufficient social/caregiver resources to optimize outcomes? | Comments were that this criterion is unacceptable, is potentially discriminatory, and will create disadvantages. The health system should support such patients. |
| Impact on other resources (8) | What is the impact on resource use (eg, having to use additional chemotherapy because of delayed CAR-T)? | There was concern that this disadvantages people who may require more treatment to get them to CAR-T |
| Length of wait (6) | Where all else is equal (ie, likelihood to benefit and likelihood to harm), a patient waiting longer should receive access before a patient who has waited for a shorter period | This criterion could potentially be a tiebreaker |
| First come, first serve (5) | How would we know who was first come so that he/she would be first served (eg, time of consult and time of the patient’s decision to proceed)? Potential to penalize patients who take time reaching a decision. | There was agreement that first come, first served was too arbitrary of a criterion for such an important decision as to whether or not to prioritize a patient to CAR-T. This criterion was subsequently removed from the list. This treatment option is so valuable, the selection process needs to be fine-tuned. |
| Exhausted all other treatment options (2) | CAR-T should be offered in the context of a range of treatment possibilities. What are the other options? How do their risks/benefits compare? | This criterion only had 1 vote in the poll, the lowest of any other criterion. Patients had concerns about this. Uncertainty regarding “What does no other treatment options mean? Available? Working?” “What if an Indigenous person chose to pursue non-Western healing, and did not exhaust all Western options?” |