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ORKAMBI™ Update: Letter from Vertex Pharmaceuticals

We have an  ORKAMBI™ update; please read this letter Cystic Fibrosis Canada received from Vertex regarding the CADTH reconsideration.

Cystic Fibrosis Canada is committed to working with all of the stakeholders to ensure ORKAMBI is made available to people with CF who have the potential to benefit. We are well placed to provide patient perspective, as well as independent, evidence-based analysis. It is our strong feeling that the expertise of people living with CF and CF physicians must be considered throughout the very complex, and often opaque drug approval process in Canada.

Vertex Pharmaceuticals Incorporated 50 Northern Avenue
Boston, MA
Tel: 617-341-6100

July 22, 2016

Norma Beauchamp
President and CEO
Cystic Fibrosis Canada
2323 Yonge Street, Suite 800
Toronto, Ontario M4P 2C9

Dear Norma:

PrORKAMBITM Common Drug Review (CDR) Submission

I am writing on behalf of Vertex to respond to Cystic Fibrosis Canada’s statement dated July 19, 2016 regarding the CDR submission for ORKAMBI (­access-to-orkambi%E2%84%A2-is-delayed?p). In your statement, you expressed disappointment that people with cystic fibrosis (CF) in Canada “are going to have to wait even longer to gain access to ORKAMBI”. I want to reassure you that Vertex believes all indicated patients with CF in Canada should have timely access to ORKAMBI. Therefore, we are committed to working with the Canadian Agency for Drugs and Technologies in Health (CADTH), the pan-Canadian Pharmaceutical Alliance (pCPA), and the public drugs plans towards this goal.

As noted on the ORKAMBI CDR Submission Status Report published on CADTH’s website, Vertex requested reconsideration of the Embargoed Canadian Drug Expert Committee (CDEC) Recommendation. I can confirm that we requested reconsideration on the grounds that the Embargoed CDEC Recommendation is not supported by the evidence that has been submitted or the evidence identified in the CDR review report. We hope CDEC will consider our request at its next meeting scheduled to be held on September 21, 2016.

The clinical benefits of ORKAMBI have been studied, well documented and recognized around the world, as part of the largest clinical trial program ever conducted in CF with over 1,100 patients enrolled. These trials included participants at CF clinics in British Columbia, Alberta, Ontario, Quebec and Nova Scotia. ORKAMBI is the only treatment approved by Health Canada that addresses the underlying protein defect in CF for patients age 12 years and older who are homozygous for the F508del mutation in the CFTR gene. This medicine has the potential to slow disease progression and provide a range of benefits across multiple measures in CF. In fact, in its Summary Basis of Decision, Health Canada stated that ORKAMBI “demonstrated a significant increase in effectiveness with an improved benefit/risk profile compared to existing therapies for CF in patients age 12 years and older who are homozygous for the F508del mutation in the CFTR gene, a condition that is not adequately managed by a drug marketed in Canada and as such, Priority Review status was granted”.’

In your statement, Adult CF Advisory Committee Chair Megan Parker said it “is imperative that the various bodies involved in the process, including Vertex, be driven by the evidence and patient need, to ensure access to ORKAMBI in a manner that serves the needs of patients.” We believe that our original CDR Submission included evidence that demonstrates the clinical benefits of ORKAMBI in treating the underlying protein defect in CF for indicated patients, and also shows how this treatment improves and maintains lung function, improves nutritional status and reduces pulmonary exacerbations that require hospitalization. Prior to the CDEC meeting, Vertex will work with CADTH to clarify the issues raised in the request for reconsideration. This process involves a teleconference between Vertex and CADTH.

We agree with your position that “the advice and opinions of the CF experts who lead Canada’s highly specialized CF clinics need to be carefully considered by the CDEC”. We are of the view that the recommendations for the clinical use of ORKAMBI developed by Cystic Fibrosis Canada’s CF Physician Panel on lumacaftor/ivacaftor – a document endorsed by 26 CF medical experts from across Canada ( – articulates the clinical benefits of ORKAMBI in treating indicated patients. Furthermore, we believe that the patient group input submitted by Cystic Fibrosis Canada highlights the current unmet needs of people with CF age 12 years and older who are homozygous for the F508del mutation in the CFTR gene. As CADTH permits the inclusion of external participants at this teleconference, we would like to invite your Chief Scientific Officer, Dr. John Wallenburg, to participate on the call with CADTH. We will also be extending an invitation to a member of your CF Physician Panel on lumacaftor/ivacaftor to participate as well. We believe this adds to the transparency of the process and ensures that the independent voices of patients and CF experts are represented.

We are pleased that some private insurers have approved coverage for patients who have been prescribed ORKAMBI. We will continue to work with the remaining private insurers to ensure that all patients with appropriate private insurance can have access to ORKAMBI as soon as possible.

Nearly two decades ago, a small group of Vertex scientists set out to discover and develop the first medicines to treat the underlying protein defect in CF. Together with the CF community we have made tremendous progress. Vertex is a company that is “All in for CF”; at Vertex our CF research program is united around a singular passion: to bring new medicines to all people living with this rare, life-shortening disease. In closing, I would like to reiterate our commitment to working with CADTH, the pCPA and public drug plans towards timely access to ORKAMBI for indicated patients.

Sincerely yours,

Andrew Partridge

Senior Vice President and Head of North America Vertex Pharmaceuticals Incorporated

CC: Dr. Brian O’Rourke, President and Chief Executive Officer Federal/Provincial Public Drug Plans