Using patient data to improve patient care: Are we there yet?
October 17, 2020
Specialty medications continue to conquer new ground, becoming more sophisticated and widespread with each passing year.
Between 2018 and 2019, use of these medications among Canadians grew by 7.8%,1 and growth continues apace. These drugs take a lot of effort to develop, but they save and transform lives.
And yet.
These “modern miracles” aren’t consistently reaching the Canadians who need them. In a stakeholder survey conducted in 2018-19, both Canadian cancer patients and their physicians identified “long waits and inaccuracies in diagnoses” as top issues in oncology treatment.2 The next hurdle is access. One in five Canadians struggles to pay for prescription medications,3 and the path to reimbursement for specialty drugs has become so tangled that few patients can travel it on their own.
On the plus side, stakeholders are getting serious about using patient data to tackle these problems. A white paper called Mission: Patient Centricity explains how outcomes data can help demonstrate treatment efficacy, support negotiations with payers, and keep patients with rare diseases motivated. 4 Patients themselves have joined forces to identify and communicate the health outcomes that matter to them. The initiatives detailed below suggest we’re on the right track; we just need to connect the guideposts.
Patients seeking value
No longer content to sit on the sidelines, patient organizations are setting the agenda for the selection and use of real-world data. After the concept of value-based healthcare (VBHC) gained traction in a 2018 summit, the patient group Coalition Priorité Cancer au Québec saw an opportunity to mobilize VBHC in the province. Partnering with VBHC Canada, the Coalition launched a patient-led demonstration project to review patient-reported outcome measures (PROMs) for breast, lung and colorectal cancer.5 The initiative entrusted patients in four cancer care centres to validate the outcomes and identify obstacles to achieving them, while keeping all levels of government abreast of their progress.5 If all goes according to plan, the learning will give rise to policies and procedures that better support the PROMs.
Along similar lines, Colorectal Cancer Canada (CCC) has launched the Patient Values Project to assign weights to various patient priorities in cancer drug treatment.6 These weights will give other patient groups a new vocabulary for liaising with governments and increasing timely access to the cancer drugs that best meet their needs.6 At the same time, CCC is pushing for the collection of patient-centric data in clinical trials.6
As a shining example of the narrowing gap between ivory tower and ground-level reality, the Canadian Myeloma Research Network has set up a database of real-world outcomes that researchers and governments can use to explore therapeutic gaps and solutions.7 The network’s goals also include dialing up the patient voice in clinical trial steering committees and facilitating access to new treatments that are not yet reimbursed.
First-hand experience
In tandem with these developments, patient experience is steadily gaining ground as a metric to consider in drug development. To this end, various patient groups are polling their members to find out exactly what they like and dislike about their condition and its treatment.
In a recent virtual symposium, the Canadian Skin Patient Alliance presented results of its survey of patients with hidradenitis suppurativa, a painful skin condition affecting up to 4% of Canadians.8 Attesting to the enormous challenge posed by this disease, survey respondents had tried an average of 15 treatments, surgeries and lifestyle modifications.9 And while 36% used biologics to manage the condition, only 20% reported satisfaction with currently available treatments, reflecting a considerable unmet need.8 Similarly, Migraine Canada is developing surveys to capture the experience of living with migraines and gathering testimonials from people who have tried different treatments for the disorder.10
The Canadian Agency for Drugs and Technologies in Health (CADTH) takes such data points seriously. At the outset of a project, CADTH solicits feedback from patients—typically centred on disease impact, treatments, and presentation of information—and folds the input into its drug reviews.11 In an analysis of this process, CADTH concluded that “patients offer unique insight into what it is like to live with a specific disease or medical condition; they also help by sharing their hopes for new treatments.”11
Patients get a hearing: the case of Aimivog
In July of this year, CADTH’s Canadian Drug Expert Committee (CDEC) recommended that the specialty migraine drug Aimovig (erenumab) be reimbursed for patients meeting specific criteria.12 The recommendation represented the culmination of an arduous review process that included two reconsideration meetings.12 Patient groups can take at least part of the credit for this outcome, having identified an unmet need for safe and effective medications to prevent chronic migraines. When making its final recommendation, CDEC considered input from Migraine Canada and Migraine Quebec, including a national patient survey affirming that migraine impacted intimate and family relationships for 97% of respondents.12,13
Distinct and bold
Sharing data with regulators is one thing, but what about industry? The idea made sense to Pierre Fitzgibbon, Quebec’s Minister of Economy, who recently suggested giving the pharma industry access—at a cost—to patient data held by the Régie de l'assurance maladie du Québec (RAMQ).14 Stating that the future of medicine lies in using data to customize treatment, he maintained it was possible to share the data with industry without breaching patient confidentiality.14
As expected, not everyone cheered. Solidarity deputy Vincent Marissal, for one, likened the move to giving “the keys to the bloodbank to the vampires.”14 The Alliance des patients pour la santé took a more nuanced position. In a social media post, the group affirmed that “the road turns” and “there is no point in sticking your head in the sand.”15 Noting the theoretical benefits of the idea to research and patient care, they deemed it worthwhile to explore the feasibility of the proposal.15
Measures that matter
Gone are the days when a drug’s efficacy in clinical trials trumped all other considerations. The success of today’s specialty medications depends increasingly on data from the real world. No longer an afterthought, such real-world data is helping to shape everything from drug development and evaluation to PSP design and agreements with payers.
As a growing number of studies have shown, clinical trial data does not reliably translate to broader populations. A case in point: an analysis of 15 studies published this year, researchers compared the effect of riluzole, a drug for amyotrophic lateral sclerosis (ALS), in population studies versus clinical trials.16 As it turned out, the drug extended lives by 6 to 19 months longer in real-world situations than in clinical trials, giving hope to ALS patients and strengthening the rationale for the medication.16
Real-world data can inform not just treatment, but diagnosis. In one instance, Quebec and Ontario researchers analyzed tissue samples from 483 prostate tumors, identified the “molecular signature” of each sample, and used the information to develop algorithms for recognizing different forms of prostate cancer.18
Industry, for its part, has been harnessing the PSP infrastructure to collect real-world evidence (RWE) to support research and improve services in these programs. To get a pulse on this trend, the RWE & OBA Working Group surveyed Canadian pharmaceutical companies with PSPs and PSP vendors in July 2020.17 Questions focused on the current status of PSP data collection and the potential for RWE to support outcomes-based agreements (OBAs) with payers. Results will be published late this year.
We’re getting close
The pieces are all in place. Canada has shown itself capable of using data as a diagnostic and treatment tool, to identify outcomes that mean the most to patients, and to amalgamate the patient perspective into decisions about specialty drugs. Just a few more bold moves will take us to the finish line: using patient data to assess specialty drug performance and get the right drugs to the patients who need them. If we continue to put patients first, we’ll get there.
References:
2020 Telus health drug data trends & national benchmarks. https://plus.telushealth.co/blogs/health-benefits/en/2020-drug-data-trends-national-benchmark-report/
All.Can Canada: Consolidated report on waste and inefficiency in Canadian cancer care. https://saveyourskin.ca/wp-content/uploads/Consolidated-Report-of-Waste-and-Inefficiency-in-Canadian-Cancer-Care_All.Can-Canada-Report.pdf
A prescription for Canada: achieving pharmacare for all. Government of Canada. https://www.canada.ca/en/health-canada/corporate/about-health-canada/public-engagement/external-advisory-bodies/implementation-national-pharmacare/final-report.html
Mission: patient centricity. Takeda, 2020. https://www.takeda.com/4a4d5e/siteassets/system/what-we-do/areas-of-focus/rare-diseases/personalized-care-for-patients-living-with-rare-diseases/mission-patient-centricity_co-branded-takeda_pwc_en_final.pdf
A dose of reality: patients redefining the future of healthcare in Canada. Summary report of the 4th annual summit, 2019. https://saveyourskin.ca/wp-content/uploads/Patients-Redefining-Healthcare-Summit-2019-Report-EN-Final.pdf
Colorectal Cancer Canada. Ongoing initiatives. https://www.colorectalcancercanada.com/about/current-initiatives/
Myeloma Canada Research Network. About Us. http://www.mcrn.ca/about-us/
CADTH 2020 Symposium. Synopsis of presentation by Rachel Manion of the Canadian Skin Patient Alliance. https://symposium.cadth.ca/2020/07/27/effectiveness-and-patient-satisfaction-with-treatment-options-for-hidradenitis-suppurativa-in-canada-results-from-the-hidradenitis-suppurativa-patient-experience-survey/
Rachel Manion et al. Canadian Skin Patient Alliance poster. https://symposium.cadth.ca/wp-content/uploads/2020/07/Poster_1906.pdf
Migraine Canada. Coming up: Migraine Canada will take action in 2020. https://migrainecanada.org/coming-up/
Boucher J et al. How patient feedback is used by CADTH: insights from patient letters. https://symposium.cadth.ca/wp-content/uploads/2020/07/Poster_1669.pdf
CADTH Canadian Drug Expert Committee recommendation for erenumab. July 2020. https://www.cadth.ca/sites/default/files/cdr/complete/SR0578%20Aimovig%20-%20CDEC%20Final%20Recommendation%20July%2024%2C%202020%20%28redacted%29_For%20Posting.pdf
CADTH common drug review. Patient input for erenumab. https://www.cadth.ca/sites/default/files/cdr/relatedinfo/SR0578-Aimovig-patient-input.pdf
Québec veut attirer les pharmaceutiques avec les données de la RAMQ. Le Devoir, 21 août 2020. https://www.ledevoir.com/politique/quebec/584542/quebec-veut-attirer-les-pharmaceutiques-avec-les-donnees-de-la-ramq
Alliance des patients pour la santé Facebook page. See August 30, 2020 post. https://www.facebook.com/alliancepatients
Andrews JA et al. Real-world evidence of riluzole effectiveness in treating amyotrophic lateral sclerosis. Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration 2020, e-pub ahead-of-print. https://doi.org/10.1080/21678421.2020.1771734
Canadian patient support program data capabilities survey. RWE & OBA Working Group, 2020. https://www.20sense.ca/psp-data-survey-july-2020
L’IA pour améliorer le diagnostic du cancer de la prostate. CS Science. 24 août 2020. https://www.cscience.ca/2020/08/24/lia-pour-ameliorer-le-diagnostic-du-cancer-de-la-prostate/