Table 1.
Checklist for the characteristics of an Ideal MAP
| Accountability | ||
| Programme Goals | ||
| Is the MAP appropriate for the question at hand? | □ Yes | □ No |
| Will the patients receive earlier access to the drug? | □ Yes | □ No |
| Will the programme collect the evidence needed to find the right drug for the right patient? | □ Yes | □ No |
| Will all of the processes within the programme (eg decision making) be transparent to ensure greater buy‐in? | □ Yes | □ No |
| Governance | ||
| Programme‐Specific Committee | ||
| Will there be a programme‐specific committee established to guide the MAP? | □ Yes | □ No |
| Will there be 3 patient members on the committee? | □ Yes | □ No |
| Will the patient members meet the follow criteria: | ||
| Meet a minimum level of experience with the health‐care system | □ Yes | □ No |
| Have a meaningful role on the committee? | □ Yes | □ No |
| Are accountable back to the disease community that they represent? | □ Yes | □ No |
| Will patient organizations select the patient members? | □ Yes | □ No |
| Will there be a physician committee member? | □ Yes | □ No |
| Will they be an expert in the rare disease? | □ Yes | □ No |
| Will patient organizations select the physician member? | □ Yes | □ No |
| Will the committee meetings be open to all patients and caregivers who wish to attend? | □ Yes | □ No |
| Individual Patient Input | ||
| Will individual input from a broad range of patients be collected to develop the MAP? | □ Yes | □ No |
| Will the process be quick and efficient? | □ Yes | □ No |
| Will there be a variety of ways for patients to provide input? | □ Yes | □ No |
| Will the input processes be transparent and patients well informed of the opportunity? | □ Yes | □ No |
| International Collaboration | ||
| Will there be collaboration with other countries to learn from their experiences with MAPs? | □ Yes | □ No |
| Will there be collaboration with other countries to conduct trials (if necessary)? | □ Yes | □ No |
| Will there be collaboration with experts in other countries to educate Canadian physicians on the rare disease? | □ Yes | □ No |
| Evidence Collection | ||
| On‐going Monitoring and Registries | ||
| Will there be on‐going monitoring with an engaged physician and good documentation (eg through EMRs)? | □ Yes | □ No |
| Will the following information be collected: | □ Yes | □ No |
| Natural history data? | □ Yes | □ No |
| Qualitative data? | □ Yes | □ No |
| Clinical outcomes? | □ Yes | □ No |
| Outcome Measures and Continuation Criteria | ||
| Will the outcome measures used be meaningful to patients and adequately capture their experiences? | □ Yes | □ No |
| Will patients provide input on meaningful outcome measures? | □ Yes | □ No |
| Will decisions to continue/discontinue therapy be made between physicians and patients without the use of set continuation criteria? | □ Yes | □ No |
| Will there be follow‐through on the results of the MAP? | □ Yes | □ No |