- Ontario Public Drug Program got best price for Kuvan, but gave worst funding criteria -
TORONTO, May 27, 2015 /CNW/ - Members of the Ontario PKU community gather at the Ontario Legislature today to urge the government to revise the extremely restrictive access criteria for Kuvan (sapropterin dihydrochloride), the first and only pharmaceutical treatment for the rare, brain-threatening disease, PKU (phenylketonuria). More than two years after the Ontario Public Drug Program listed Kuvan at the best price in the world, the medication remains out of reach for the estimated 150 patients who can benefit from it, and who rely on public funding – an unacceptable situation for Ontario PKU families.
"It is clear the system is broken. More than two years after Kuvan was listed for reimbursement in Ontario, not a single PKU patient has been able to access this treatment through public funding, leaving them vulnerable to brain damage," says John Adams, President and CEO of Canadian PKU and Allied Disorders Inc. (CanPKU), and father to a 28-year-old son with PKU. "Despite holding numerous meetings with the Ontario Public Drug Program to raise our concerns, and Ontario PKU physicians asking for revisions to the criteria, no changes have been made. We are here at Queen's Park to urge Premier Wynne and Health Minister Hoskins to cut the red tape and expand the criteria for Kuvan."
A brain-threatening disease without access to proper treatment
In PKU, the body is unable to process an essential amino acid found in dietary protein, called phenylalanine (or "Phe"), and the resulting accumulation of Phe in the blood is toxic to the brain. If left untreated, symptoms can range from mild cognitive impairment to severe intellectual disability, along with other neurological problems. To stave off the negative health consequences of Phe, patients must adhere every day of their lives to a complicated and highly-restrictive, very low-protein diet, including special medical foods and synthetic formulas. Everything a PKU person eats and drinks could result in brain damage.
Despite patients' best efforts in dietary planning, adherence to the low-Phe diet and frequent blood monitoring, many are still unable to adequately control their blood Phe levels, leaving their brains at risk for neurocognitive impairment with psychosocial impacts.i Though medical foods can help patients adhere to the Phe-restricted diet, Kuvan – approved by Health Canada in April 2010 – is proven to reduce blood Phe levels when taken in conjunction with diet.ii
"Just four months after my daughter started treatment with Kuvan, her teacher was amazed by the improvements to her concentration, focus and ability to work more independently," says Cristian Baigorria, father to 14-year-old Cande, who has PKU and has received Kuvan on a compassionate basis from the manufacturer since 2010. Cande would not qualify for public funding under the current criteria. "If one day Cande is no longer able to access Kuvan because the government won't agree to fund it, it will be a huge challenge for us and may negatively impact her brain development and her future. We are afraid to even think about it."
Government not following clinical guidelines, expert advice
June 1, 2015 will mark the 50th anniversary of the introduction of a government program, now called Newborn Screening Ontario, which tests every child born in Ontario for PKU. But, the government's job is far from over once patients have been diagnosed. According to the World Health Organization, if a jurisdiction screens for early identification of PKU to reduce suboptimal outcomes, it also has the responsibility to uphold an important criteria of a screening test – to treat the condition adequately on an ongoing basis.iii
Ontario provides relatively comprehensive funding for the low-Phe synthetic formulas and medical foods,iv which have formed the basis of PKU treatment since the 1960s. However, prompted by increasing evidence of suboptimal outcomes in PKU patients managed only with a restrictive diet, the world's leading PKU specialists recommend the use of Kuvan to improve patient outcomes.v In 2014, clinical guidelines published for PKU, co-authored by two Canadian experts, included a comprehensive plan for early intervention and lifelong treatment composed of a combination of customized therapies, including Kuvan.vi
To harm or protect?
"Today, this government has an important decision to make. Will it continue to harm the brains of Ontarians who have the genetic misfortune of being born with this metabolic disorder by restricting access to Kuvan, or will it heed the guidance of medical experts and protect PKU patients' brains from toxic levels of dietary protein and expand the reimbursement criteria?" says Mr. Adams. "The government has the responsibility to do everything reasonable to protect the brains of Ontarians living with PKU, and the longer the criteria revisions take, the longer the brains of PKU patients – children, teens and adults – remain needlessly at risk. This is a case of medical malpractice by the Ontario Public Drug Program."
About Canadian PKU and Allied Disorders Inc.
Canadian PKU and Allied Disorders Inc. is a nonprofit association of volunteers, dedicated to providing accurate news, information and support to families and professionals dealing with PKU and similar, rare, inherited metabolic disorders. Our mission is to improve the lives of people with PKU and allied disorders and the lives of their families. By allied disorders we mean other rare, inherited metabolic disorders also detected by newborn screening. For more information, visit www.canpku.org or www.CanPKUAction.org.
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References
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i Brumm VL, Bilder D, Waisbren SE. Psychiatric symptoms and disorders in phenylketonuria. Mol Genet Metab. 2010; 99:S59-S63.
ii Kuvan [product monograph]. Toronto, ON: BioMarin Pharmaceutical (Canada) Inc.; 2010.
iii Wilson JMG, Jungner G. Principles and Practice of Screening for Disease. Geneva, Switzerland: World Health Organization; 1968:27. Accessed on May 25, 2015. Available at whqlibdoc.who.int/php/WHO_PHP_34.pdf.
iv Canadian PKU and Allied Disorders Inc. Coverage for PKU Diet Formulas & Medical Foods - May 2014. Accessed on May 25, 2015. Available at: http://www.canpku.org/images/pdf/2014canada_pku_coverage_report.pdf.
v Cunningham A. et al. Recommendations for the use of sapropterin in phenylketonuria. Mol. Genet Metab. 2012; 106: 269-276.
vi Vockley, Andersson, Antshel et al. Phenylalanine hydroxylase deficiency: diagnosis and management guideline, Genetics in Medicine. 2014. doi:10-1038/gim.2013.57. Accessed on May 25, 2015. Available at: http://www.nature.com/gim/journal/vaop/ncurrent/full/gim2013157a.html.
SOURCE Canadian PKU and Allied Disorders Inc.
PDF available at: http://stream1.newswire.ca/media/2015/05/27/20150527_C8242_PDF_EN_17046.pdf
John Adams, CanPKU President & CEO, 214-766-7012 mobile, [email protected]; Stephanie Ronson, Cohn & Wolfe, 647-259-3278, [email protected]
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