ZURZUVAE® (zuranolone) Receives Positive Opinion from CHMP for the Treatment of Women with Postpartum Depression
'This is an important milestone for maternal health in the European Union,' said Priya Singhal, M.D., M.P.H., Head of Development at Biogen. 'ZURZUVAE represents a novel therapeutic approach for the targeted treatment of postpartum depression, with data demonstrating potential for symptom improvement as early as day three of a 14-day course. If approved, this represents a significant opportunity to advance the treatment of a serious condition that remains both underdiagnosed and undertreated.'
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Vancouver Sun
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'Nobody knows how to tackle this': A new era in B.C. as emerging drugs cost millions of dollars per patient to treat rare diseases
The B.C. NDP government's handling of a costly treatment for a rare disease in a 10-year-old girl has put the spotlight on a new reality: drugs that cost millions of dollars per patient. The development of these drugs was encouraged when the U.S. government passed legislation in the mid-1980s that provided longer trademark exclusivity periods, tax incentives, regulatory support and smaller, more-targeted clinical trials. The European Union created a similar framework in the early 2000s. The pace of the approval of these drugs has increased over time. Start your day with a roundup of B.C.-focused news and opinion. By signing up you consent to receive the above newsletter from Postmedia Network Inc. A welcome email is on its way. If you don't see it, please check your junk folder. The next issue of Sunrise will soon be in your inbox. Please try again Interested in more newsletters? Browse here. While Canada doesn't have its own so-called orphan-drug legislation, as it is not a major developer of drugs, the federal government has approved the use of more than 100 of these drugs. The B.C. government has listed 35 of these expensive drugs that it will fund. The costs for these drugs are very high. At the low end, they include $119,000 a year for Zavesca, which is used to treat certain rare genetic disorders including Niemann-Pick type C disease that primarily attacks the nervous system. At the high end, they include Zolgensma, which is listed at a one-time cost of $2.9 million. It is a gene therapy used to treat spinal muscular atrophy. In B.C. spending on expensive drugs for rare diseases was $22 million eight years ago, according to provincial pharmaceutical spending reports. Last year, it hit just under $200 million. The annual increases jumped significantly starting in 2022, increasing by about $50 million a year. Medical observers and experts are concerned that if increases continue at that pace, spending on these expensive drugs could hurt the ability of health-care systems to treat larger populations. The just under $200 million B.C. spent on these drugs last year provided treatment to 600 people. That's about 10 per cent of the money the province spends on its pharmacare program, which provides drugs to more than one million people. These costs underscore the complex nature of decision making in this area, where governments must contend with factors such as how beneficial the drugs are, research standards, corporate profit, ethics, the interest or imperative to help patients, patient advocacy groups, emotion and politics. 'Nobody knows how to tackle this,' says Joel Lexchin, a professor emeritus at York University with an expertise in pharmaceutical policy. 'Canada is not unique in terms of figuring out do you pay for all of these drugs? Do you pay for some of them? Do you pay for none of them? Do you pay for everybody who has the disease? Do you just pay for those who have the worst form of the disease?' said Lexchin, author of the 2016 book ' Private profits versus public policy: the pharmaceutical industry and the Canadian state.' Lexchin, who was also an emergency physician, said he knows of no country that has a good policy to contend with the myriad issues. These include the high costs of the drugs, and patient groups and clinicians who want publicly funded treatment even as the effectiveness of rare disease drugs is sometimes unclear. These types of drugs have often not been studied for long periods and trials take place with fewer people than for drugs for common diseases. There's also a lack of understanding of why these drugs cost so much because the drug companies don't release that information, said Lexchin. And then there's what is called the 'rule of rescue,' which describes the strong human impulse to help identifiable individuals facing death or serious harm even when the costs of doing so are high, added Lexchin. 'If they get publicity, you'll spend a lot of money.' In British Columbia, the Ministry of Health established a review process to approve expensive drugs for rare diseases in 2007. The Health Ministry makes the final decision using advice from a body that includes an overarching advisory committee under which there are a number of subcommittees in specialized areas. The subcommittees are made up of area experts, with the subcommittee chairs all sitting on the advisory committee. The advisory committee also includes health ethicists, health economists, representatives from hospital pharmacies that are often involved with the administration of these drugs, external physicians and representatives of the Ministry of Health and the Provincial Health Services Authority . There is an appeal process. Recently, a subcommittee and the wider advisory group recommended that treatment be halted for Charleigh Pollock, a 10-year-old Vancouver Island girl who suffers from a rare neurodegenerative condition called Batten disease for which there is no cure. Those who get the disease in childhood usually don't live to adulthood. The committee recommended treatment stop because discontinuation criteria was met, beyond which the benefits versus risks were not believed to be favourable. The province had said the treatment was not stopped because of the drug's cost. Pollock was being treated by a drug called Brineura that costs more than $800,000 a year. The B.C. government supported the decision to stop treatment last month, with Premier David Eby saying politicians shouldn't interfere with medical decisions. However, facing mounting public pressure, in a dramatic reversal the B.C. government said last week it will reinstate treatment. As a result, five members of the advisory committee have quit. Dr. Sandra Sirrs, the chair of the subcommittee that recommended the treatment stop, was among those who quit. Sirrs says the government's decision undermines and complicates the navigation and decision making in an already extremely complex area where B.C. had a good process. 'The hypocrisy of it is just astonishing to me. They say no one wants politicians making decisions about your health. Well, that's exactly what they did,' said Sirrs. Sirrs had been part of the review process for drugs for rare diseases since its inception. She has also quit as the medical lead for rare diseases at the Provincial Health Services Authority, which provides provincewide specialized health services. Sirrs says patients with rare diseases should not be discriminated against, but they also should not be given an unfair advantage. Decisions on a drug should not be made based on negative publicity generated by media attention, she said. It ultimately harms the ability to negotiate prices for drugs for rare diseases with pharmaceutical companies, ending up with prices being based on a government's willingness to pay, said Sirrs. Lexchin, the York University professor, says governments should be challenging pharmaceutical companies on drug costs, but that is something Canada can't do alone and would need help from the U.S. and the European Union. He said drug approval should also come with conditions, including agreement from companies that testing and studies will continue for drugs where there is no definite proof they are beneficial. Conditions can also be put on patients, where drugs are provided for set periods of time and where benefit has to be shown, said Lexchin. Sirrs added there also needs to be a mechanism to act on new evidence, including delisting drugs, something that is not in place in Canada. Pollock's parents, her physicians, and doctors researching and treating Batten disease in the U.S. disagreed with the B.C. advisory committee's decision. Public backlash ramped up after the 10-year-old's mother started a GoFundMe campaign and enough money was raised for another treatment. The drug treatments are given through an infusion into the fluid of the brain, a procedure that takes about four hours every other week. The girl is the only person in B.C. with the disease and the only person to have the treatment discontinued in Canada, according to the province. As with some other rare disease drugs, the ultimate benefit of Brineura is not clear, according to the Canada Drug Agency, which provides evidence-based advice on drugs to provinces. Brineura, produced by U.S.-company BioMarin Pharmaceuticals, was approved by Health Canada in 2018. A review of the most recent scientific evidence on Brineura, published last month by the Canada Drug Agency, noted that emerging evidence suggested a potential benefit on quality of life, seizure control and mortality outcomes, which were deemed important to those with lived experiences. But the evidence had limitations because of a lack of concurrent control groups, patients being aware of the treatment they received, and confounding factors such as use of seizure medications and other illnesses. The 50-page review said the findings limited the ability to make definitive conclusions on the relative benefit of the drug. In a written response to Postmedia's questions, the B.C. Ministry of Health said it is paying for the cost of all covered expensive drugs for rare diseases except for two that are being funded through the federal government's new national strategy for drugs for rare diseases. The province is receiving $194 million over three years. Brineura is not covered under the federal strategy. The B.C. Health Ministry would not provide information on the number of people funded under each of the rare disease drugs on its list of 35, citing privacy concerns because so few people were receiving each treatment. The province would also not say when each of the drugs was added to the list but noted approval started in the early 2000s. The ministry said when it makes decisions on funding rare disease drugs it generally only considers those with 'positive recommendations' from the Canada Drug Agency and those for which a price has been negotiated by the pan-Canadian Pharmaceutical Alliance. The alliance combines the buying power of the provinces and territories to lower drug prices. The ministry said there are drugs for another 12 rare diseases under review by Canada and seven under negotiation at the pharmaceutical alliance. ' If a drug is covered, conditions/criteria for coverage are based on the (Canada Drug Agency's) expert recommendations,' the ministry said in an email sent by public affairs officer Amanda Lewis. B.C. Health Minister Josie Osborne was not made available for an interview. The Health Ministry also did not respond to questions on whether it is concerned it has undermined its own system with the recent decision to resume treatments for the 10-year-old girl or they have concerns expensive drugs for rare diseases will leave less money for others areas of health-care spending and what can be done about that. In the past few weeks, one of the strongest opposition voices against the decision to halt the drug treatment for the 10-year-old girl came from the Canadian Organization for Rare Disorders. After Osborne said no stone was left unturned in assessing the drug, the patient advocacy group's CEO, Durhane Wong-Rieger, said the B.C. health minister did not know what she was talking about. But some bioethics observers have raised concerns about corporate donations that these advocacy groups get from pharmaceutical companies. The Canadian Organization for Rare Disorders lists 20 corporate partners on its website , almost all of them in the pharmaceutical industry. Those included some of the biggest names in the industry such as U.S.-based Pfizer and Amgen, and Danish company Novo Nordisk. The total annual revenues of these companies is nearly $600 billion. In some years, pharmaceutical company representatives have sat on the patient advocacy group's board. 'Drug companies have their own agenda,' noted Sharon Batt, an adjunct professor in bioethics at Dalhousie University and author of the 2019 book 'Health Advocacy Inc.' Batt said partnering with and taking money from the drug industry became the norm in the 1990s after government funding to patient advocacy groups was cut. She says she'd like Canada to adopt some kind of transparency rules where anyone can check a website that discloses funding from the drug industry for all patient groups, similar to one in the U.S. for doctors. Batt said she would also like the federal government to reinstate the policy of providing funding to non-profits that work in the public interest so they are free of corporate sponsorship. She said the evidence that pharmaceutical companies use patient advocacy groups to influence policy is now overwhelming, not just in Canada but internationally. The Canadian Organization for Rare Disorders did not respond to a Postmedia question on how much it receives from pharmaceutical companies. In a written statement, the organization said it receives funding from a variety of sources. ' CORD develops policy positions and advocates independently of its funders and always in the interest of Canadians impacted by rare diseases,' said the group. ghoekstra@ For more health news and content around diseases, conditions, wellness, healthy living, drugs, treatments and more, head to – a member of the Postmedia Network.
Toronto Star
3 days ago
- Toronto Star
ZURZUVAE® (zuranolone) Receives Positive Opinion from CHMP for the Treatment of Women with Postpartum Depression
CAMBRIDGE, Mass., July 25, 2025 (GLOBE NEWSWIRE) — Biogen Inc. (Nasdaq: BIIB) announced the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) adopted a positive opinion recommending marketing authorization for ZURZUVAE® (zuranolone) for the treatment of postpartum depression (PPD) in adults following childbirth. If approved by the European Commission (EC), ZURZUVAE will be the first treatment authorized within the European Union specifically indicated to treat depressive symptoms for women with PPD. ZURZUVAE is a once-daily, oral, 14-day treatment which was approved by the U.S. Food and Drug Administration (FDA) in 2023. 'This is an important milestone for maternal health in the European Union,' said Priya Singhal, M.D., M.P.H., Head of Development at Biogen. 'ZURZUVAE represents a novel therapeutic approach for the targeted treatment of postpartum depression, with data demonstrating potential for symptom improvement as early as day three of a 14-day course. If approved, this represents a significant opportunity to advance the treatment of a serious condition that remains both underdiagnosed and undertreated.'
