Raising the Registry Bar: Q&A with Dr. Patricia Caetano of CDA-AMC
July 23, 2024
Dr. Patricia Caetano of CDA-AMC is helping rare disease registries to get set up for success.
As of May 2024, CADTH (Canadian Agency for Drugs and Technology in Health) has been renamed the Canada’s Drug Agency / L’Agence des médicaments du Canada (CDA-AMC) and with this new identity comes with an expanded mandate. In addition to conducting health technology assessments (HTA) and providing evidence, analysis, and advice to healthcare decision-makers, the new CDA-AMC introduced initiatives to reduce inefficiencies in the drug system and support better collection and sharing of real-world data. As CDA-AMC’s new Director of Drug Data Services and Analytics, Dr. Patricia Caetano brings 15 years of experience working within Manitoba’s health ministry to the Agency’s Drugs for Rare Diseases mandate. In this conversation, she discusses how the agency is helping rare disease registries set themselves up so their data can meet the standards for healthcare decision making.
What was the impetus for CDA-AMC’s work to support registries?
In March of 2023, the federal government announced its National Strategy for Drugs for Rare Diseases (DRD), and a portion of the funding was geared toward identifying and improving real-world evidence to assist with decision-making for drugs for rare diseases, since these drugs often come with limited information and high levels of uncertainty. One of our key initiatives is to develop mechanisms for registries to improve and become ready for use in HTA and regulatory reviews - helping registries become “fit for purpose.”
What are CDA-AMC’s objectives?
One of CDA-AMC’s aims is to provide high-quality recommendations for decision-makers so that they can make timely decisions and provide better access to drugs for rare diseases. We want to create best practices and standards so that when registries collect and analyze the data and submit the results for HTA, CDA-AMC has been involved in guiding that process. We say that we want to use real-world evidence, so we need to be proactive and help registries understand how the data will be used. This way, they can prepare it in the best way possible.
“We say we want to use real-world evidence, so we need to help registries understand how the data will be used so they can prepare it in the best way possible.”
Can you describe the process for developing registry standards?
Our process started with a literature review of current best practices for international standards in the registry environment and looked at which elements had been identified as important. We then asked Canadian stakeholders to rank the importance of these components in our country’s healthcare context. We're currently conducting a final analysis so the new standards can be published within the next few months.
What will the standards include?
The standards will outline the minimum requirements registries need to adhere to so that healthcare decision-makers can be confident in the data collection practices and resulting data quality. This includes what patient populations were studied, the governance model, who the overseer of the data was, and where it is stored. Which variables were collected? What is your informed consent? Do you collect patient identifiers? What the standards won’t be is prescriptive – they won’t say you must use a certain process to adjust for missing variables or give you specific outcomes that should be reported for a particular condition.
How will the standards apply to patient-reported outcomes (PROs)?
Collecting PROs is obviously a huge endeavor, so we’ll work with patient groups, clinicians, and manufacturers to best define how PROs should be assessed within a registry. Whether or not PROs are included in a registry will not necessarily impact the quality of the registry per se, however as with any data variable that is captured, the validity and accuracy of PROs should be included in the registry’s documentation.
How will CDA-AMC ensure that registries appropriately represent real-world patient populations?
It is important to understand how patients were chosen to be in the registry – were they all from one clinician? Who is missing? Did they all have access to tests and treatments in a similar manner? Are they from similar health care systems? These are the things we need to know about the data to understand its representation and to make more informed decisions.
I also think we need to be careful about what we mean by “represent.” For more common chronic diseases like diabetes, the underlying populations can be very different across regions. For example, Manitoba has a higher proportion of Indigenous population, with higher rates of type two diabetes and different barriers or enablers to healthcare access compared to other provinces. So, we need to consider differences in population across regions and how that might influence representation in registries.
CDA-AMC is also working on inventorying the existing rare disease registries in Canada – what can you tell us about that?
The inventory is complete and will be published soon on our website. Through public searches of the published and gray literature, we've identified about 130 registries that are collecting clinical data. All contain at least some Canadian patients, and some are exclusively Canadian. We’re eager to publish it and get feedback because when I present at conferences, clinicians and patient advocates often tell me that because their disease of interest doesn’t yet have a registry, they aren’t getting represented in trials or coverage decisions. We want to hear about that.
“Clinicians and patient advocates often tell me that because their area doesn’t have a registry, they aren’t getting represented or captured. We want to hear about that.”
And it goes beyond knowing what registries exist. We also want to understand their status. What do we know about their foundational data elements and governance? Do they have the ability to link to other information sources like mortality or socio-demographic data? Do they have appropriate informed consent in place, so that the data can even be used for these kinds of purposes? Once we’ve done that assessment, we’ll look at whether existing registries align with current drugs or those emerging within the next three years or so.
CDA-AMC is also providing funding to registries to help improve processes. Where does that stand?
In March 2024, we posted a Request for Proposals from rare disease registries interested in pursuing registry-improvement projects aligned with the DRD National Strategy. After a very competitive process, approximately 18 registries were selected, and these will be announced once we sign all the contracts. But this was only the first round of funding. We'll conduct consultations with additional registry owners, institutions, and patient groups to learn about what else we can fund to see registries achieve HTA-readiness. Our goal is to fully understand the quality of rare disease registry data in Canada and help registries achieve high quality evidence where possible. We want to be constantly moving toward the point where all this data gets used and not ignored.
What do you see as the largest hurdle for registries as tools to improve decision-making?
For decision-makers, a significant barrier is access to data. With rare diseases, each province’s decision-makers have information about patients in their province but, for example, when all the provinces and territories are at the negotiating table at the pan-Canadian Pharmaceutical Alliance (pCPA), and there’s a total of 40 patients across Canada, there isn’t a system in place that allows sharing of outcomes across provinces. If data access policies and standardized registries existed, then clinicians, patients, and decision-makers could reference reports or analyses on a regular basis to understand outcomes in small populations.
From the patient side, advocacy groups often say they would love to see how the data informs decision-making. Drug plan decision-makers often have to weigh whether or not a treatment that might improve an important patient outcome is worth its additional cost. And that's rarely ever just a small incremental dollar amount over current treatments – it’s often several times more expensive. We need evidence from high-quality registries to help inform these decisions and lessen the uncertainty.
How do patient support programs (PSPs) fit into the rare disease registry work?
Once CDA-AMC starts accrediting registries, I feel that PSPs should be treated just like any other registry. Our accreditation will be like a ‘certification stamp’, to indicate that the information collected meets a certain standard. There should be no difference in that process between a PSP and any other registry developed through a clinician's office or through a university/research trial. If that data is going to be used, we all need to understand it.