Healthcare
Professionals
We appreciate and thank all the healthcare providers of VHL patients who are essential components of the patient care journey. VHL research has come a long way and is continually being updated and making novel advancements. The patient receives the best care when their healthcare team has the resources necessary to ensure the best possible care for the patient.
We are dedicated to supporting healthcare providers in learning the most up-to-date VHL research and advancements in treatment options. There is hope for a brighter future for our community of warriors!
Funding Recommendations
Belzutifan gets positive CADTH Recommendation
On August 3rd, 2023 the Canadian Agency for Drugs and Technologies in Heath (CADTH) made a positive recommendation regarding the Merck VHL drug Belzutifan.
In part the recommendation was:
The CADTH pCODR Expert Review Committee (pERC) recommends that belzutifan be reimbursed for the treatment of adult patients with von Hippel-Lindau (VHL) disease who require therapy for associated non-metastatic renal cell carcinoma (RCC), central nervous system (CNS) hemangioblastomas, or non-metastatic pancreatic neuroendocrine tumors (pNET), not requiring immediate surgery only if the conditions listed in Table 1 are met.
The full recommendation can be found in the attached document:
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Dr. Maryam Soleimani, CVHLA Scientific and Medical Advisory Council Chair, BC Cancer said the following about the CADTH recommendation:
“Belzutifan represents a true paradigm shift in the management of von-Hippel Lindau (VHL) disease. With the recent positive recommendation from CADTH, the next step is ensuring equitable access across Canada for all patients with VHL disease. This is an exciting time in VHL care and I believe it is the beginning of many more advancements to come”
Big thank you’s go to Durhane Wong-Rieger, Bob Bick, Christine Collins and Gail Ouellette who were instrumental in the submissions.
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https://spharm-inc.com/the-drug-review-and-approval-process-in-canada-an-eguide/
The CADTH approval represents about half of the Regulatory Review process in the above diagram.
Here is a quick overview from the pan-Canadian Pharmaceutical Alliance (pCPA) website of the drug approval process in Canada, with links for more information.
The following pCPA diagram and information gives an overview of the drug review process in Canada
Health Canada
Health Canada reviews drugs for safety, efficacy and quality before authorizing them for sale in Canada.
CADTH & INESSS
In Canada there are two health technology assessment organizations which review the clinical and cost-effectiveness of a drug product; the Canadian Agency for Drugs and Technologies in Health (CADTH) and in Quebec, l’Institut national d’excellence en santé et en services sociaux (INESSS). CADTH and INESSS provide a recommendation to public drug plans on whether or not a drug should be reimbursed for public funding.
pCPA
The pCPA negotiation process begins for the majority of new drugs, once a recommendation is published by CADTH and/or INESSS. pCPA uses the recommendations from CADTH and INESSS and other factors to determine whether or not it will enter into a negotiation for a drug. Following a successful negotiation, pCPA will issue a letter of intent which sets the terms of the agreement between pCPA and the drug manufacturer.
Public Drug Plans
Public Drug Plans make a final decision to fund a drug once a negotiation has been successfully completed and enter into a product listing agreement with the drug manufacturer.
pCPA Office
The pCPA office is hosted and staffed through Ontario and works closely with the jurisdictions providing support in negotiations, administration, communications, standardization, analytics, process design, and policy related to brand and generic products.
CVHLA RESEARCH
$480,000
In 2018 the CVHLA formed a partnership with the Cancer Research Society (CRS) that has turned our $120,000 of research money into $480,000 of Canadian Research over the last 3 years.
$240,000
Your CVHLA donations of $60,000 were matched by the Cancer Research Society (CRS) to create a $120,000 research grant. A second grant of $120,000 was awarded to a second group by the CRS, totaling $240,000 in VHL research for 2020.
$240,000
Your CVHLA donations of $60,000 were matched by the CRS to create a $120,000 research grant that was awarded to Dr. Najafabadi McGill University.A second grant of $120,000 was awarded to a second group by the CRS, totaling $240,000 in VHL research for 2018.
Dr. Sandra Turcotte
2020 Canadian VHL Alliance $120,000 Grant with Partner
Cancer Research Society
Winner of the $120,000 Grant, in partnership with the CRS was Dr. Sandra Turcotte, University of Moncton.
Harnessing autophagy and lysosomes as a potential therapeutic approach in hereditary renal cell carcinoma with von Hippel-Lindau disease.
Summary: Von Hippel-Lindau (VHL) is an inherited neoplasia syndrome caused by mutations on the VHL gene and resulting in the development of tumours in different organs such as the eye, ear, adrenal gland, brain, pancreas and kidney. About 70% of VHL patients will develop Renal Cell Carcinoma (RCC) which occurs earlier in their lifetime than other people developing RCC. No curative treatment is available for ccRCC. This project is based on our previous work in kidney cancer and aims to exploit lysosomal vulnerabilities in VHL-mutated cells to develop novel therapeutic strategy for patients with ccRCC associated with VHL disease.
Dr. Hamed Najafabadi
2018 Canadian VHL Alliance $120,000 Grant with Partner
Cancer Research Society
In 2018 the winner of the $120,000 Grant, in partnership with the CRS was is Dr. Hamed Najafabadi from McGill University in Montreal.
Clear cell renal cell carcinoma (ccRCC) is the most common form of kidney cancer. In the majority of ccRCC tumours, the von Hippel Lindau (VHL) gene is inactivated, which leads to the constitutive activity of the hypoxia-inducible factors (HIFs) and downstream activation of hypoxia signaling genes. The activation of hypoxia signaling, in turn, triggers a cascade of molecular events that are integral to tumour maintenance and progression. This project aims to systematically identify the genes and pathways involved in the regulation of hypoxia signaling in ccRCC.
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