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CATALIS In Support of the Quebec Government's Policy on Rare Diseases  

On June 6, the Quebec government unveiled its Politique québécoise pour les maladies rares (Quebec policy on rare diseases – in French only). In Quebec, 700,000 people with rare diseases, half of whom are children, face significant challenges such as errors in diagnosing their disease and the absence of effective treatments. Through its new ministerial policy, Quebec is ahead of the other Canadian provinces by focusing on three areas of action to improve the quality of life of people with rare diseases: increasing awareness and training of health professionals; fair and easier access to a diagnosis, care, and services; and promoting research, innovation, and data collection.

It is within this context that CATALIS, funded by the Quebec Life Sciences Strategy, is proud to work with the Quebec government to fulfill its mission of supporting patients with rare diseases. Thanks to its FAST TRACK Evaluation Service and its Personalized Clinical Trial Support Service for patients and healthcare professionals, CATALIS is helping the government to meet the targets for accelerating the launch and facilitating the conduct of clinical research in Quebec, including finding treatments for rare diseases.


“It's thanks to the collaboration of our researchers that our life sciences sector is performing well and achieving scientific breakthroughs that are transforming the lives of the Quebec population. CATALIS is one of the organizations that have made this success possible by making clinical research in Quebec more effective.”

Pierre Fitzgibbon, Minister of the Economy, Innovation and Energy, Minister responsible for Regional Economic Development and Minister responsible for the Metropolis and the Montreal Region

3 New Studies on Rare Diseases by Novartis Authorized in Record Time Thanks to the FAST TRACK Evaluation Service 

It is within the framework of this assistance to the government that CATALIS is delighted to announce that 3 new studies on rare diseases by Novartis Canada have once again been authorized in record time via the FAST TRACK Evaluation Service, including a study on Zolgensma for the treatment of children aged 2 to 12 with spinal muscular atrophy.


I’m very impressed with Quebec’s determination to drive change and accelerate clinical research for those living with rare diseases. The Quebec policy on rare diseases reinforces the importance of speed and effectiveness of clinical trials to address the urgent need for patients to have access to potentially lifesaving treatments. Our collective efforts must strive to include access to these innovative treatments at time of Health Canada approval. I’m thrilled to continue working with CATALIS to focus our efforts on breaking barriers to advance research that can positively impact the lives of patients and their families.

Andrea Marazzi, Novartis Canada Country President

STRENGTH Study: Zolgensma for Children Aged 2 to 12 With Spinal Muscular Atrophy

In October 2021, Minister Dubé announced that Zolgensma, a drug produced by the Novartis pharmaceutical company, was added to the list of drugs offered in Quebec health facilities to treat children under the age of 2 with spinal muscular atrophy.

Spinal muscular atrophy is a rare genetic neuromuscular disease. It is the leading genetic cause of death in young children and is characterized by a progressive and permanent loss of the cells that control muscles. In the most severe cases, children experience increasing muscle weakness and paralysis, as well as difficulty swallowing and breathing.

As this drug is not currently available for children over 2 years of age, Novartis is using this new phase III study to explore the efficacy of gene therapy with a single dose of Zolgensma for children aged 2 to 12 years. This is an ultra-competitive study being conducted in 15 hospitals in 10 countries, with a recruitment target of only 28 pediatric patients worldwide.

Dr. Maryam Oskoui, this study’s head researcher, highlighted the important role CATALIS’ FAST TRACK Evaluation Service plays in positioning Quebec at the forefront of clinical research – an advantage that has allows the province’s littlest patients to participate in this new study.

The Research Institute of the McGill University Health Centre (RI-MUCH) authorized the STRENGTH study in 12 weeks, putting it in first place among activated sites worldwide. The study is now open for recruitment and should allow the enrolment of 3 to 4 Quebec patients.


“This study is important because it represents the hope for a less invasive and more effective treatment than the ones our young patients are currently receiving. It goes without saying that by speeding up approval times for clinical studies, Quebec is becoming very competitive on the international stage, which increases our chances of conducting innovative studies like the STRENGTH study.”

Dr. Maryam Oskoui, Scientist at the RI-MUHC

MASter-1 Study: Authorization in 8 Weeks to Explore a New Treatment for an Extremely Rare Disease

In addition to the STRENGTH study on Spinal Muscular Atrophy, CATALIS’ FAST TRACK Evaluation Service also served to allow Novartis’ MASter-1 study to be authorized by the CHU Sainte-Justine in 8 weeks; the hospital’s site was the first to be activated in Canada, out of a total of 17 sites in 10 countries. 

MASter-1 is a phase II study investigating a new treatment, MAS825, for an extremely rare infant autoimmune disease, NLRC4-GOF, which causes macrophage activation syndrome and necrotizing enterocolitis.

Currently, cortisone, which inhibits the development in children, is the only treatment used to reduce inflammation caused by the disease. The Novartis study explores the efficacy of a new drug that could be less harmful to pediatric patients with the disease and, therefore, offer them a better quality of life.

Although the clinical trial is currently scheduled to recruit 2 to 3 pediatric patient in Quebec, Dr. De Bruycker, the study’s lead researcher, thinks that this study’s potential medical breakthroughs could also have a beneficial impact on many other children with autoimmune diseases who are looking for better treatments.



“As a doctor, you often feel powerless in the face of the devastation caused by rare diseases in children. Access to a potential new treatment through this clinical trial is an unparalleled source of hope for CHU Sainte-Justine patients and their families and could save lives.”

Dr. Jean-Jacques De Bruycker, lead researcher at the CHU Sainte-Justine



“In many cases, there is no therapeutic solution for patients with rare diseases. With CATALIS’ support, CHU Sainte-Justine is pleased to facilitate access to innovative clinical trials for patients with these diseases and to help identify promising treatments.”

– Dr Jacques L. Michaud, Director, CHU Sainte-Justine Research Centre

NEPTUNUS-2 Study: Sjogren’s Syndrome Clinical Trial Approved in 8 Weeks via CATALIS’ FAST TRACK Evaluation Service

In NEPTUNUS-2, a phase III study, Novartis and Dr. Isabelle Fortin are testing the efficacy and safety of Ianalumab in patients with Sjögren Syndrome, a rare disease. This study, which is taking place in 195 sites in 30 countries and aims to recruit 489 patients across the globe, was authorized in Quebec in 8 weeks by the Centre intégré de santé et de services sociaux du Bas-Saint-Laurent (CISSS Bas-St-Laurent).


“I found the FAST TRACK Evaluation Service motivating and stimulating. It upended some old attitudes and habits for the good of patients’ access to treatment. Thank you to the CATALIS team for their great work!”

– Dr. Isabelle Fortin, rheumatologist, CISSS Bas-Saint-Laurent


A fourth Novartis study on symptomatic osteoarthritis of the knee was also authorized by the Centre intégré universitaire de santé et de services sociaux de l’Estrie – Centre hospitalier de l’Université de Sherbrooke (CIUSSS de l’Estrie – CHUS)  in 9 weeks via the CATALIS FAST TRACK Evaluation Service.

The Personalized Clinical Trial Support Service Assists Patients with Various Diseases, Including Rare Ones

CATALIS, under the banner Clinical Trials Quebec, is continuing the pilot implementation of its Personalized Clinical Trial Service. This province-wide, bilingual resource is free of charge; it is designed to help Quebec patients and their caregivers, regardless of their age or medical condition. This service provides patients with a user-friendly and personal support in their search for a clinical trial that meets their specific criteria while answering their questions about clinical research.

Based on the Politique québécoise pour les maladies rares (Quebec policy on rare diseases – in French only) introduced earlier this year by the Quebec government, the Personalized Clinical Trial Support Service works closely with the Regroupement québécois des maladies orphelines (RQMO, a provincial coalition of orphan diseases) and collaborates with seven other patient associations.


“Of the thousands of known rare diseases, only a few hundred have a specific treatment. The others are so-called orphan diseases. The Personalized Clinical Trial Support Service will allow people with orphan diseases and parents of children with orphan diseases to learn about and possibly participate in ongoing clinical trials or be aware of the therapeutic advances made for their disease.”

Gail Ouellette, president and scientific director of the Regroupement québécois des maladies orphelines (RQMO)

Since the launch of its pilot in July 2022, the Personalized Clinical Trial Support Service has been working with the following patient associations: the Regroupement québécois des maladies orphelines (RQMO), the Amyotrophic Lateral Sclerosis Society of Quebec (ALS-Quebec), Muscular Dystrophy Canada (MDC), the Multiple Sclerosis Society of Canada – Quebec Division, the Federation of Quebec Alzheimer Societies, Colorectal Cancer Canada, Myeloma Canada, and Arthritis Society Canada.

The implementation of CATALIS' Personalized Clinical Trial Support Service has been made possible thanks to the Quebec government and will be offered free of charge to all Quebecers and health professionals in early 2023.

Astrazeneca and Vertex Join the Catalis Network

CATALIS Quebec is very proud to announce that pharmaceutical companies AstraZeneca and Vertex are joining the CATALIS Network. As new members, they join Abbvie, Bayer, Boehriger Ingelheim, GSK, Merck, Novartis, Pfizer, Roche, Sanofi and Takeda. Together, the partners support CATALIS in its mission to optimize the clinical research environment to accelerate the development of innovative patient care and maximize private investment in the province.

We’re excited to be partnering with CATALIS and believe their FAST TRACK Evaluation Service is a significant step in helping to optimize and accelerate the launch timeline of clinical trials in Quebec – strengthening the province’s position and reputation as a world-class hub in medical research. By simplifying the regulatory environment and accelerating clinical launch timelines we’re able to better attract new clinical studies to Quebec in such areas as breast and lung cancer, severe asthma, cardiovascular diseases and more – helping Canadian patients to gain early access to and potentially benefit from new medical innovations.

– Kiersten Combs – President, AstraZeneca Canada



“Quebec’s leadership in clinical research and in rare diseases makes it a unique place in Canada. As Vertex continues to advance its clinical and research programs, we are confident that our partnership with CATALIS will help to provide to Quebec patients a quick access to potentially innovative treatments.”

Michael Siauw, General Manager, Vertex Pharmaceuticals (Canada) Incorporated

Catalis' Management Is Being Supported by Two New Advisory Committees

Committed to having a comprehensive understanding of Quebec’s clinical research environment and listening to its Network of partners, CATALIS Quebec’s executives recently created two new advisory committees to oversee the strategic alignment of its operations.

In keeping with the government’s desire to modernize access to data, CATALIS recently created a new advisory committee on data, artificial intelligence, and real-world evidence. This new committee’s mandate is to identify the main issues and prioritize the needs of non-academic researchers for accessing and using clinical-administrative data from health network institutions for clinical research purposes. As such, nearly twenty provincial and international experts recently joined CATALIS’ brainstorming and have submitted a brief to support the Quebec government’s towards Bill 3.

In addition to the operations, scientific, innovation, and patient advisory committees, CATALIS recently created a new advisory committee specific to research pharmacies in Quebec’s health network institutions. The purpose of this committee is to identify pharmacies’ operational issues within the clinical research context to develop innovative provincial solutions to facilitate and accelerate administrative processes related to the evaluation and conduct of clinical trials, including better adaptation to decentralized clinical trials.

CATALIS takes this opportunity to acknowledge the work and commitment of the CATALIS Network’s partners, which include more than 120 specialized public-private experts who contribute to the success of CATALIS' government mandate.

2 New Employees Join the CATALIS Team


Xavier Bonhomme - Quality System and Training Specialist 


Xavier has over 34 years of professional experience in the pharmaceutical industry, in hospital settings and in academic research. He has 12 years of experience in hospital pharmacy manufacturing control in Europe. In Quebec, Xavier was introduced to clinical research at a major Montreal Clinical Research Organization (CRO) and spent 17 years working in quality assurance. In 2018, Xavier joined the MUHC Research Institute, where he developed a deeper understanding of the diversity of structures and methods that prevent the research community from achieving the success it deserves. Xavier wants to concretize his ideal of transposing best practices to the world of research, while privileging simplicity to the service of human potential. He is convinced that sharing ideas, tools and knowledge will help to promote the many strengths of Quebec’s clinical research.


Line Darbelli - Accounting and Administration Technician


Line has more than 10 years of experience in general accounting. She worked for the Adonis Group Inc. in accounting and was promoted to Accounts Payable Supervisor in 2020. In this role, she demonstrated her management and administrative skills through by supervising a team of 30 accounting clerks. In addition, Line was involved in one of the largest finance projects at Adonis ⁄ Metro: the integration of a new SAP accounting system. As an Accounting and Administration Technician, Line will put her skills to work for the CATALIS mission.

The Catalis Quebec Team Wishes You a Very Happy Holiday Season!

CATALIS Quebec is a non-profit partnership dedicated to the advancement and operational excellence of clinical research in Quebec.

Since 2017, CATALIS Quebec has been carrying out a large-scale government mandate to optimize Quebec’s clinical research environment. The CATALIS network's efforts are focused on providing more Quebec patients with access to tomorrow’s therapies.

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