Latest news with #Zolgensma
Vancouver Sun
4 days ago
- Health
- Vancouver Sun
'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.
Gulf Today
18-07-2025
- Health
- Gulf Today
Al Jalila Children's Hospital crosses 100 treatments for Spinal Muscular Atrophy
Al Jalila Children's Hospital, part of Dubai Health, and the only children's hospital in the UAE, has completed over 100 treatments for patients with Spinal Muscular Atrophy (SMA). Dr Mohamed Al Awadhi, Executive Director of the Dubai Health Women and Children's Campus, said, "Reaching more than 100 treated cases of SMA reflects Dubai Health's commitment to making world-class care accessible to all children. At Al Jalila Children's Hospital, we follow an integrated, patient-centred approach that supports every phase of the treatment journey. Every step is managed with precision and compassion to put the patient first. Yaqeen's case highlights the strength of our multidisciplinary model and our commitment to ensuring that children with complex conditions like SMA receive the highest standard of care under one roof.' Dr Haitham Elbashir, Consultant Pediatric Neurorehabilitation at Al Jalila Children's Hospital, said, "Zolgensma is a transformative therapy that offers children with SMA the chance to regain strength and function early in life.' The completion of over 100 SMA treatments at Al Jalila Children's reflects the strength of Dubai Health's specialised pediatric services and its commitment to delivering high-quality care for children with complex medical needs. WAM
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First Post
11-07-2025
- Health
- First Post
Major cancer drugs could become cheaper as India considers import duty waiver
The documents show that blockbuster cancer drugs like pembrolizumab (brand Keytruda), osimertinib (brand Tagrisso), and trastuzumab deruxtecan (brand Enhertu), used to treat lung and breast cancer, will be fully exempted from customs duty read more India will likely slash the prices of medicines used to treat critical conditions like HIV Aids and cancer as a government panel has recommended waiving off some customs duties on high-impact drugs. News18 has accessed a document of the proposed cuts in prices that are expected to provide relief to patients with cancer and other chronic diseases. The central government has constituted an interdepartmental committee that has suggested customs duty exemptions and concessions for high-impact medical imports. STORY CONTINUES BELOW THIS AD Established in August 2024 by the Drug Controller General of India (DCGI), the panel is headed by Joint Drug Controller R. Chandrashekar and includes representatives from the Indian Council of Medical Research (ICMR), the Department of Pharmaceuticals, and the Directorate General of Health Services (DGHS). Its goal is to make life-saving treatments, such as those for cancer, rare diseases, organ transplants, and advanced diagnostics, much more affordable for Indian patients. More from Health US faces worst measles outbreak in decades amid falling vaccination rates under Trump Which drugs will get cheaper? The documents show that blockbuster cancer drugs like pembrolizumab (brand Keytruda), osimertinib (brand Tagrisso), and trastuzumab deruxtecan (brand Enhertu), used to treat lung and breast cancer, will be fully exempted from customs duty. These medicines often cost up to lakhs per dose and have remained inaccessible to patients due to the high import burden. Apart from cancer drugs, the committee has also proposed making several other medicines cheaper, including transplant drugs, critical care medicines, and advanced diagnostic kits that are slapped with heavy import duties and have no equivalent in the Indian market. A second category of essential but widely available drugs has been proposed for a reduced import duty of 5 per cent. This list includes hydroxyurea, used to treat cancer and sickle cell anaemia, and low molecular weight heparin, marketed as Enoxaparin, which is commonly used to prevent and treat blood clots and deep vein thrombosis. Therapies also included Along with life-saving drugs, the document also mentions price cuts for medical therapies to treat rare diseases. Customs duty exemptions might be applied on a group of therapies used to treat conditions such as spinal muscular atrophy, cystic fibrosis, Gaucher disease, Fabry disease, lysosomal storage disorders, and hereditary enzyme deficiencies. Many of these therapies, such as gene-based and enzyme replacement treatments, rank among the most expensive drugs globally, with single courses costing several crores. Notable rare disease drugs on the list include Zolgensma, Spinraza, Evrysdi, Cerezyme, and Takhzyro. These medications are extremely costly and largely beyond the reach of most Indian patients. STORY CONTINUES BELOW THIS AD
Business Standard
25-06-2025
- Health
- Business Standard
Baby Aasmika Undergoes Lifesaving Zolgensma Gene Therapy After Raising INR 8.41 Cr on Impact Guru with Support from 5.29 Lakh Donors
NewsVoir Kolkata (West Bengal) [India], June 25: In a heartening milestone for pediatric care and collective compassion, baby Aasmika Das from West Bengal has successfully undergone Zolgensma gene therapy at a leading hospital in Kolkata. This medical breakthrough was made possible after her parents raised INR 8.41 crore via the medical crowdfunding platform Impact Guru, with the generous support of 5.29 lakh donors from across India and the world. Aasmika's fundraiser also recorded the highest number of donors ever on a single campaign on Impact Guru. Aasmika was diagnosed with Spinal Muscular Atrophy (SMA) Type 1, a rare and life-threatening genetic disorder that weakens muscles and impairs vital functions such as breathing, swallowing, and movement. Her father, Suvongkar Das, expressed heartfelt gratitude, "We will forever be grateful to each of the 5.29 lakh donors who gave our daughter a second chance at life. It's because of your kindness that Aasmika has a fighting chance to grow up, laugh, play, and live the life every child deserves. You've given us the greatest gift a parent could ask for." SMA Type 1 is caused by a mutation in the SMN1 gene, leading to the deterioration of motor neurons. Left untreated, it is often fatal within the first two years of life. Zolgensma, one of the world's most expensive drugs, offers a one-time gene therapy that replaces the faulty gene with a functional copy, offering the promise of long-term recovery and normalcy. Piyush Jain, Co-Founder and CEO of Impact Guru, commented on the successful outcome, "Aasmika's story is a testament to the power of collective goodwill. At Impact Guru, we're proud to enable a platform where millions can unite to save a single life. The success of this campaign reflects not just the strength of our technology but also the generosity of the human spirit. We remain committed to making advanced healthcare accessible for all." Impact Guru's crowdfunding platform played a pivotal role in mobilizing donations through micro-contributions, social sharing, and unified calls for help. The campaign received overwhelming public support and went viral, reaching donors from every corner of the country.
The Wire
25-06-2025
- Health
- The Wire
Baby Aasmika Undergoes Lifesaving Zolgensma Gene Therapy After Raising INR 8.41 Cr on Impact Guru with Support from 5.29 Lakh Donors
Kolkata, West Bengal, India (NewsVoir) In a heartening milestone for pediatric care and collective compassion, baby Aasmika Das from West Bengal has successfully undergone Zolgensma gene therapy at a leading hospital in Kolkata. This medical breakthrough was made possible after her parents raised INR 8.41 crore via the medical crowdfunding platform Impact Guru, with the generous support of 5.29 lakh donors from across India and the world. Aasmika's fundraiser also recorded the highest number of donors ever on a single campaign on Impact Guru. Aasmika was diagnosed with Spinal Muscular Atrophy (SMA) Type 1, a rare and life-threatening genetic disorder that weakens muscles and impairs vital functions such as breathing, swallowing, and movement. Her father, Suvongkar Das, expressed heartfelt gratitude, 'We will forever be grateful to each of the 5.29 lakh donors who gave our daughter a second chance at life. It's because of your kindness that Aasmika has a fighting chance to grow up, laugh, play, and live the life every child deserves. You've given us the greatest gift a parent could ask for.' SMA Type 1 is caused by a mutation in the SMN1 gene, leading to the deterioration of motor neurons. Left untreated, it is often fatal within the first two years of life. Zolgensma, one of the world's most expensive drugs, offers a one-time gene therapy that replaces the faulty gene with a functional copy, offering the promise of long-term recovery and normalcy. Piyush Jain, Co-Founder and CEO of Impact Guru, commented on the successful outcome, 'Aasmika's story is a testament to the power of collective goodwill. At Impact Guru, we're proud to enable a platform where millions can unite to save a single life. The success of this campaign reflects not just the strength of our technology but also the generosity of the human spirit. We remain committed to making advanced healthcare accessible for all.' Impact Guru's crowdfunding platform played a pivotal role in mobilizing donations through micro-contributions, social sharing, and unified calls for help. The campaign received overwhelming public support and went viral, reaching donors from every corner of the country. Aasmika's successful administration of Zolgensma is not just a medical milestone - it is a testament to what India can achieve when empathy meets innovation. (Disclaimer: The above press release comes to you under an arrangement with Newsvoir and PTI takes no editorial responsibility for the same.).
