Access to new drugs for rare disorders in Canada

Rawson, Nigel S. B.; Adams, John S.

doi:10.1503/cmaj.69340

Cited by 2 publications

(2 citation statements)

References 5 publications

(5 reference statements)

Supporting

Mentioning

Contrasting

Order By: Relevance

“…Resource allocation based on an equitable, transparent process ensures procedural justice and is an important consideration in a publicly funded healthcare system such as in Canada 20. The caregiver experiences gleaned from our study highlight the need for effective and equitable delivery of orphan drugs to allow timely access to emerging, costly, life-sustaining therapies 21–23…”

Section: Discussionmentioning

confidence: 98%

Understanding caregiver experiences with disease-modifying therapies for spinal muscular atrophy: a qualitative study

et al. 2023

View full text Add to dashboard Cite

ObjectiveSpinal muscular atrophy (SMA) is a neuromuscular disorder that manifests with motor deterioration and respiratory complications. The paradigm of care is shifting as disease-modifying therapies including nusinersen, onasemnogene abeparvovec and risdiplam alter the disease trajectory of SMA. The objective of this study was to explore caregivers’ experiences with disease-modifying therapies for SMA.DesignQualitative study including semistructured interviews with caregivers of children with SMA who received disease-modifying therapies. Interviews were audio recorded, transcribed verbatim, coded and analysed using content analysis.SettingThe Hospital for Sick Children (Toronto, Canada).ResultsFifteen family caregivers of children with SMA type 1 (n=5), type 2 (n=5) and type 3 (n=5) participated. There were two emerging themes and several subthemes (in parentheses): (1) inequities in access to disease-modifying therapies (variable regulatory approvals, prohibitively expensive therapies and insufficient infrastructure) and (2) patient and family experience with disease-modifying therapies (decision making, hope, fear and uncertainty).ConclusionThe caregiver experience with SMA has been transformed by the advent of disease-modifying therapies. Consistent and predictable access to disease-modifying therapies is a major concern for caregivers of children with SMA but is influenced by regulatory approvals, funding and eligibility criteria that are heterogenous across jurisdictions. Many caregivers described going to great lengths to access therapies, highlighting issues related to justice, such as equity and access. This diverse population reflects contemporary patients and families with SMA; their broad experiences may inform the healthcare delivery of other emerging orphan drugs.

show abstract

Section: Discussionmentioning

confidence: 98%

Understanding caregiver experiences with disease-modifying therapies for spinal muscular atrophy: a qualitative study

et al. 2023

View full text Add to dashboard Cite

show abstract

“…12 Several obstacles to accessing new drugs exist in Canada. 1,[13][14][15][16] These include government-controlled health technology assessments (HTAs) and price negotiations, government drug plan gatekeepers taking much time to list drugs in their formularies, 17,18 and stringent criteria and out-of-pocket costs that patients must satisfy to gain coverage for medicines even after they are listed. 12 HTAs are performed by the Canadian Agency for Drugs and Technologies in Health (CADTH) for all of Canada, except the province of Quebec.…”

Section: Introductionmentioning

confidence: 99%

Timeliness of Health Technology Assessments and Price Negotiations for Oncology Drugs in Canada

Rawson,

Stewart

2024

CEOR

View full text Add to dashboard Cite

Purpose To evaluate whether time targets for Canadian Agency for Drugs and Technologies in Health (CADTH) reimbursement reviews and pan-Canadian Pharmaceutical Alliance (pCPA) price negotiations are being achieved for oncology drugs. Materials and Methods Recommendations, dates of submission and publication, and indications for oncology medicines issued between January 2014 and December 2023 were recorded from CADTH’s reimbursement reports webpage. The date any negotiation began and the date it was completed (successfully or not), or when a decision was made not to pursue negotiation was extracted from the pCPA’s webpage. The duration of each CADTH review and pCPA negotiation was calculated, together with time between CADTH’s recommendation and start of the pCPA negotiation or a decision not to negotiate. Percentages of reviews completed within CADTH’s target and of times taken by the pCPA to decide whether to negotiate and by its price negotiations completed within the relevant targets were calculated. Results CADTH achieved its 270-days target in 88.2% to 100% of reviews issued between 2015 and 2019 but only in 65.9% to 73.1% of reviews issued in the last three years of the decade. CADTH’s “typical timeline” of 180 days was achieved in under 40% of reviews issued in 2015 and not attained in any review in 2021, 2022 or 2023. The pCPA’s target of 60 days for deciding whether to negotiate was achieved for all recommendations issued in 2014 but dropped below 40% for the last seven years of the decade; its target of 130 days for negotiations was achieved for over 85% of the recommendations in 2014 but decreased to only 14.3% in 2016 and then gradually increased to 61.5% in 2023. Conclusion CADTH’s “typical timeline” and the pCPA’s targets were not met sufficiently to be meaningful. Their processes take too long for cancer drugs.

show abstract

Access to new drugs for rare disorders in Canada

Cited by 2 publications

References 5 publications

Understanding caregiver experiences with disease-modifying therapies for spinal muscular atrophy: a qualitative study

Understanding caregiver experiences with disease-modifying therapies for spinal muscular atrophy: a qualitative study

Timeliness of Health Technology Assessments and Price Negotiations for Oncology Drugs in Canada

Contact Info

Product

Resources

About