The timing of this award is aligned with efforts to improve the clinical trial readiness of the Alpha-1 community worldwide since the pipeline of therapies being applied to AATD is robust. Access to AlphaNet and Alpha-1 Foundation infrastructure is available to help the successful applicant, although letters of support from these programs should be part of the submission package. Current programs in the North American Alpha-1 community include the Alpha-1 Foundation Research Registry, the Alpha-1 Canada Registry, Databases of AlphaNet Canada and AlphaNet, Inc., and the NIH Alpha-1 Biomarkers Consortium (A1BC) program that is linked to the Alpha-1 Foundation Registry. The A1BC program is designed to support a wide range of meritorious biomarker research related to AATD including basic laboratory investigations, epidemiology, clinical genetics and imaging research.
Successful applicants will be required to use the A1BC cohort to validate successful PRO tools that are developed outside of this population.
The deadline for the receipt of grant applications is January 15, 2023 by 11:59 PM ET.
Application instructions can be found here. To download the application click here.
**ONLY qualified donees, who are currently registered with the Government of Canada, are eligible for this RFA. To check donee qualification status, see List of charities and certain other qualified donees – basic search (cra-arc.gc.ca) for donees located in Canada and List of universities outside Canada registered as qualified donees – Canada.ca for donees outside of Canada.
GOALS OF THE ALPHANET CANADA PATIENT-REPORTED OUTCOME (PRO) GRANTS AWARD PROGRAM
PROs have been collected in the Alpha-1 community on health-related quality of life (e.g., short form 36, SF-36) and lung disease specific quality of life (e.g., St. George’s Respiratory Questionnaire, SGRQ). These tools are useful in reporting the burden of disease when comparing AATD to the general population.
However, what is missing in the Alpha-1 community is a targeted PRO that will allow investigators to demonstrate responsiveness to treatment(s) in a clinical trial. The development of an Alpha-1 specific PRO should provide additional complementary information about a patient’s health related quality of life (HRQL). This RFA recognizes that more than one Alpha-1 PRO tool may be required to capture the heterogeneity in AATD. It is generally believed that a carefully developed, patient-tailored, and disease-specific instrument(s) is likely to be more sensitive to underlying change than generic instruments.
There are many types of PRO tools that measure functional status (physical and cognitive), symptoms and symptom burden (e.g., fatigue and dyspnea), impact on health behaviors, and the patient experience of care. The vision for this RFA is focused on development and validity analyses for a disease-specific tool that is freely available and supported by Alpha-1 community resources.