Comparative Effectiveness and Safety of Biosimilar and Legacy Drugs
试验速览
- 阶段
- 不适用
- 发起方
- 入组人数
- 800
- 试验地点
- 1
- 主要终点
- Persistence on the initial treatment, measured as time in months to drug discontinuation.
研究概览
简要总结
In Canada and worldwide there is a need for updated independent real-world comparative effectiveness and safety data related to biologic drugs including biosimilar drugs. Biosimilar drugs hold potential to improve access to needed therapies at reduced cost enabling savings to be reallocated to other needs. However updated real-world evidence on comparative effectiveness and safety of biosimilar drugs is lacking. Investigators aim to demonstrate feasibility of creating network of clinical cohorts and other resources to provide real-world information on use of biosimilar drugs in Canada.
The core revolves around clinical datasets but investigators will complement with other data sources. Investigators will review data from National Prescription Drug Utilization Information System database that contains prescription claims-level data collected from publicly financed drug benefit programs in different provinces to conduct an environmental scan of the use of biosimilars and respective legacy drugs and other anti-Tumor Necrosis Factor agents covered by provincial drug plans from 2014-2017. Initial analysis will help to confirm that use of biosimilars is lower than corresponding legacy drugs.
Biologic drugs are relatively new and expensive drugs; biosimilar medicines are similar to original biologic drugs but cost less. If patients receive biosimilar drugs rather than originator biologics healthcare systems may be able to save money. Those savings can be used for other health care needs to benefit more Canadians. However investigators do not have detailed information on safety and effectiveness of these biosimilar drugs. The aim of study is to compare safety and effectiveness of biosimilar drugs to originator biologic drugs. Investigators will study patients with inflammatory rheumatic diseases (RA and AS) and Inflammatory Bowel Disease (CD and UC) and across Canada on these drugs. Primary focus is on patients without history of biologic drug use but investigators will also study patients switching to biosimilar drug from an originator biologic drug. Investigators will measure how long patients stay on treatment, if patients require new treatment, if the patients' disease control improves and occurrence of side effects such as infection that could be related to these drugs.
详细描述
Research question: What is the comparative effectiveness and safety of biosimilar drugs versus the equivalent legacy drugs?
Primary aims:
To compare, in biologic-naive patients, new users of biosimilar drugs versus new users of the equivalent legacy drugs:
- Frequency of discontinuation of the initial therapy
- Persistence on the initial therapy (time until drug discontinuation)
- Frequency of patients starting or increasing prednisone or other immunosuppressive drugs
- Frequency of and time to discontinuation of treatment due to ineffectiveness
- Frequency of and time to clinical remission/induction of response
- Frequency of and time to serious adverse events
Secondary aims
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •The study will include cohort members from both sexes, 18 years and older, with a clinical diagnosis of inflammatory rheumatic disease (either RA or AS), or IBD (CD or UC) who have given their informed consent. There are no disease activity criteria for entry. At the time of enrolment, patients in each disease group will be classified into the following treatment subgroups:
- •Biologic-naïve, starting any biosimilar or the equivalent legacy drug;
- •Patients switching to biosimilar or the equivalent legacy drug from an alternative biologic therapy;
- •Patients switching to a biosimilar (or starting a new cycle with the equivalent legacy drug), successfully completed and exited a previous course of therapy with the equivalent legacy drug.
排除标准
- •Under 18 years of age
- •No clinical diagnosis of inflammatory rheumatic disease (either RA or AS), or IBD (CD or UC)
- •Refused to participate or sign informed consent
研究组 & 干预措施
Originator (legacy) drug
Reference group
干预措施: Originator (legacy) drug (Drug)
Biosimilar
Exposed group
干预措施: Biosimilar (Drug)
结局指标
主要结局
Persistence on the initial treatment, measured as time in months to drug discontinuation.
时间窗: From cohort entry until the date of discontinuation of the initial therapy or date of death from any cause, whichever came first, assessed up to 54 months.
In each of the four conditions (RA, AS, CD, UC), the primary outcome will be persistence on treatment, measured as time from the cohort entry, either the date of first biologic/biosimilar prescription (for biologic-naïve patients) or the date of switching to a biologic/biosimilar, until the date of discontinuation of the initial therapy or date of death from any cause, whichever came first.
次要结局
- Time in months from cohort entry to treatment discontinuation/switching due to treatment failure or adverse events.(From cohort entry until the date of discontinuation/switching of treatment due to treatment failure or adverse events or date of death from any cause, whichever came first, assessed up to 54 months.)
- Proportion of participants discontinuing/switching the initial treatment due to treatment failure or adverse events.(Months 3, 6, 12, 18, 24, 36, 48, and 54)
- Proportion of RA participants achieving sustained disease remission assessed using the DAS28.(Months 12, 18, 24, 36, 48, and 54)
- Proportion of participants who modified therapy during follow-up.(Months 3, 6, 12, 18, 24, 36, 48, and 54)
- In RA participants, change from baseline in the Health Assessment Questionnaire-Disability Index (HAQ-DI) score at months 12, 24, and 48.(Months 12, 24, and 48)
- Change from baseline in the European Quality of Life-5 Dimensions (EQ-5D) score at months 12 and 24.(Months 12, 24, and 48)
- Proportion of RA participants achieving disease remission assessed using the Disease Activity Score 28 (DAS28).(Months 3, 6, 12, 18, 24, 36, 48, and 54)
- Proportion of RA participants achieving low disease activity (LDA) assessed using DAS28.(Months 3, 6, 12, 18, 24, 36, 48, and 54)
- Proportion of AS participants achieving an ASAS20 response(Months 3, 6, 12, 18, 24, 36, 48, and 54)
- Proportion of AS participants achieving sustained remission using the Ankylosing Spondylitis Disease Activity Score (ASDAS)(Months 12, 18, 24, 36, 48, and 54)
- Proportion of UC participants with induction of response within 3 months of initiation of treatment using the partial Mayo Score (PMS)(Month 3)
- Proportion of UC participants with sustained response after initiation of treatment using the PMS.(Months 6, 12, 18, 24, 36, 48, and 54)
- Proportion of UC participants achieving clinical remission using the PMS.(Months 3, 6, 12, 18, 24, 36, 48, and 54)
- Proportion of UC participants with sustained clinical remission using the PMS.(Months 6, 12, 18, 24, 36, 48, and 54)
- Proportion of UC participants with of loss of clinical response among responders using the PMS.(Months 6, 12, 18, 24, 36, 48, and 54)
- Proportion of CD participants with induction of response within 3 months of initiation of treatment using the Harvey-Bradshaw Index (HBI)(Month 3)
- Proportion of CD participants with sustained response after initiation of treatment using the HBI.(Months 6, 12, 18, 24, 36, 48, and 54)
- Proportion of CD participants achieving clinical remission using the HBI.(Months 3, 6, 12, 18, 24, 36, 48, and 54)
- Proportion of CD participants with sustained clinical remission using the HBI.(Months 6, 12, 18, 24, 36, 48, and 54)
- Proportion of CD participants with of loss of clinical response among responders using the HBI.(Months 6, 12, 18, 24, 36, 48, and 54)
- Change from baseline in the Short Inflammatory Bowel Disease Questionnaire (SIBDQ) score at months 12, 24, and 48 in UC and CD participants.(Months 12, 24, and 48.)
研究者
Dr. Sasha Bernatsky
Principal Investigator
McGill University Health Centre/Research Institute of the McGill University Health Centre