ALS Action Canada Submission to the Canada Drug Agency

1.1 What suggestions do you have for evolving the patient input process to focus on what is needed to support deliberations on the deliberative framework domains? 

The Advocacy Committee of ALS Action Canada (ALSAC) very much appreciates the extensive efforts being made by CDA’s Engagement Group to ensure adequate inclusion of PWLE input into reimbursement decisions. This is particularly important for drugs for rare diseases such as ALS, as also is the importance of ensuring adequate input from Clinical Experts when expertise in the therapeutic area is not adequately represented in CDEC. 

ALSAC’s first concern is with CDA’s Process for Drug Reimbursement Reviews, which restricts input by PWLE and Clinical Expert to drugs that have been determined to meet the criteria for Complex Review according to Scenario One or Two, as specified in the revised Procedures. Scenario One criteria (first drug in a therapeutic area) will exclude a new drug for ALS from the Patient Input provisions in the Complex Review process. Scenario Two criteria (drug with potential to alter the treatment paradigm) are: added clinical benefit compared with the most appropriate comparator(s); AND has the potential to alter the treatment paradigm based on superior efficacy and/or safety; AND has been granted priority review by Health Canada, or accepted for review under NOC/c. These criteria are overly restrictive, because current treatment options for treatment of ALS are of limited efficacy. Any new drug with demonstrated efficacy has the potential to change the clinical treatment paradigm, but the proposed criteria require comparisons with other drugs, which does not align with the current reality of clinical trial design for ALS drug development. The search for more effective drugs for ALS does not employ comparative clinical trial designs, but rather has the objective of demonstrating efficacy relative to placebo, while often allowing concomitant treatment with riluzole and/or edaravone, since it would be ethically unacceptable to restrict their use. CDA needs to revise the criteria for Complex Review to accommodate all drugs for rare diseases, so that all reimbursement reviews of such drugs are eligible for PWLE input.

The second general area of concern for ALSAC is the way in which PWLE input is actually used during CDEC’s deliberations, and the degree to which it ever influences the final decision, given the exclusion of PWLE’s and Clinical Experts from the deliberation on proposed reimbursement criteria. The inclusion of a single patient representative on CDEC is inadequate representation for the breadth of experience across diverse therapeutic areas, especially those for rare diseases. At least one PWLE should be present in all deliberative meetings of CDEC, on a drug under review in their therapeutic area, as an advisor to the Patient Representative member of CDEC.            

2.1 What are your ideas and suggestions for methods (e.g., surveys, interviews, focus groups, videos, other) you think work well to collect patient perspectives? Please explain why.

The process currently used by CDA to collect PWLE input is overly constrained by the the very specific format of the Input Template and is very time-intensive for the Patient Groups, often requiring issuance of a survey to a large number of PWLE’s, as well as detailed analysis of the responses. While a survey issued through Patient Groups is in principle a reasonable approach, it needs to be supplemented by interviews or focus groups with individual PWLE’s in order to provide a more comprehensive picture of the therapeutic need and potential benefit of the drug under review.  A second major problem with CDA’s current process is the restriction to one submission per Patient Group. This fails to acknowledge that within Patient Groups there can be individual PWLE’s who are members of Advocacy Committees or Research Advisory Boards whose expertise extends beyond their personal experience of the disease or its treatment. Providing a mechanism for the input of such individuals would improve the credibility of CDA’s commitment to patient engagement.  

2.2 What are your ideas and suggestions for formats (e.g., written input template, verbal input, targeted input questions, open-ended questions, broad questions, a combination of these) you think work well to submit patient group input? Please explain why. 

As noted in 2.1, while CDA’s practice of inviting responses to surveys issued through Patient Groups is in itself a reasonable approach, the very specific format of the Written Input Templates currently used by CDA has constrained the type of input received, and is very time intensive for the Patient Groups. We believe that this process can be both simplified, and provide for more comprehensive input, through further consultation on the design of a new Patient Input Template.  In addition or as an alternative, broad areas of questioning and open-ended questions would provide some guidance for the input, while facilitating more comprehensive input on the unmet therapeutic need and potential benefit of the drug under review, based on the diversity of experience across a wide range of PWLE’s.

3.1 What guidance, communication, or tools would make the input process easier for you (e.g., video guide[s], learning sessions, guidance document, tool kit, other)? 

We do not see a need for extensive additional guidance. A simplified and less rigid input format is needed to allow more comprehensive Patient Group input on the unmet therapeutic need. A broad guideline on the type of PWLE input that is most helpful to the deliberative process could be useful, but this should not constrain the ability of the PWLE to engage with CDEC. This interaction needs to include the ability to participate in two-way questions and answers with the Committee, as well as comment on the proposed directions for reimbursement criteria.  

3.2 What type of feedback would you like to receive on your input submission? 

We would appreciate feedback on all aspects of patient input that have not been accommodated in the reimbursement decision. It is our recent experience that both PWLE and Clinical Expert input has had no influence on the restrictive criteria issued by CDEC for drugs recently approved for ALS by Health Canada, other than the simplistic statement in the reimbursement review decision, that reimbursement was limited to patients who meet the criteria used for inclusion in the clinical trial. Clinical experts in ALS have repeatedly stated to CDEC that this is not a clinically appropriate basis for reimbursement criteria, and feedback to the Patient Group should explicitly justify the basis for CDEC’s divergence from the opinion of Clinical Experts.

We also request feedback on the proposals made in this submission, for example: the revisions needed to the criteria for Complex Review; the need for review of all drugs for rare diseases to be Complex so that they are eligible for PWLE input; participation of at least one PWLE as an advisor to the Patient Representative on CDEC in all phases of the deliberative process; provision for input from more than one Patient Group representative; simplification and increased comprehensiveness of the Patient Group Input template.

3.3 How would you like to receive feedback on your input submission? 

We would appreciate written feedback on all aspects of patient input that have not been accommodated in the reimbursement decision, for the reasons stated in section 3.2, concerning the restrictions placed by CDEC on reimbursement of new drugs for ALS. 

We also request a written response to the specific proposals made in this submission regarding: the revisions needed to the criteria for Complex Review, as stated in section 3.2: the revisions needed to the criteria for Complex Review; the need for review of all drugs for rare diseases to be Complex so that they are eligible for PWLE input; participation of at least one PWLE as an advisor to the Patient Representative on CDEC in all phases of the deliberative process; provision for input from more than one Patient Group representative; simplification and increased comprehensiveness of the Patient Group Input template.

4.1 Do you have any comments or suggestions we should consider in evolving patient group input into drug reimbursement reviews that was not captured in the previous questions? 

No relevant feedback to submit.