Ivonne Mitar

At Sandoz, we believe that driving biosimilar adoption can support more equitable access to high-quality healthcare and improve outcomes for patients across the globe. We’re excited about the opportunity to foster Day 1 access to generics and biosimilars in Europe, improve supply chain resilience and simplify the regulatory framework with the ongoing EU Pharma Review 🌍 This week, Rebecca Guntern, President Region Europe and Country President Switzerland, and Peter Stenico, Global Head of Biosimilars and Country President Austria, spoke at Reuters Events Pharma where they shared their insights on the importance of revising the EU pharmaceutical legislation, regulatory streamlining, and the adoption of smarter procurement processes to increase access to generics and biosimilars in Europe. See below for some key takeaways from Rebecca and Peter 👇 #Biosimilars #Healthcare #REPharma2024 http://bit.ly/43Qc64c

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▶ #Imlygic was the first #ATMP, which reimbursement price was set by the arbitration board after HTA. Imlygic (active substance: talimogene laherparepvec) was approved as a gene therapy for treatment of adults with unresectable melanoma in December 2015. Imlygic is available in Germany. 🏆German HTA rating:  The randomized, pivotal study OPTiM was submitted for #HTA, which compared Imlygic to GM-CSF. However, GM-CSF did not match the defined appropriate comparator by the German HTA body. Depending on prior treatments and BRAF V600-status the appropriate comparators were vemurafenib, vemurafenib in combination with cobimetinib, dabrafenib in combination with trametinib, pembrolizumab, nivolumab or therapy according to physician’s choice. Consequently, the additional benefit of Imlygic was not proven, due to the lack of HTA relevant evidence. 💵 Launch price: € 2,398.50 per vial. Continuous treatment with up to 4 vials in a 14-day cycle. Reimbursement price: €1,220.52 per vial. Rebate: 52 % 📘 Special features of reimbursement: Imlygic is reimbursed. The reimbursement price was set by the arbitration board. The reimbursement price contract contains a clause, which stipulates that if the median treatment duration of Imlygic is longer than 4.1 months, the reimbursement price would increase to € 1,526.33. However, an increase was never listed. 💡 Although an #RCT for the ATMP Imlygic was submitted for HTA, no additional benefit could be achieved due to the wrong comparator in the pivotal trial. The arbitration board designed the reimbursement price contract as a kind of “pay for performance” contract.

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Preferential pricing in Germany for drugs that had significant development investment in the country—that is the government’s plan. The change is expected to be included in the forthcoming Medizinforschungsgesetz (Medical Research Act), according to Thomas Müller, the Head of the Pharmaceuticals Department at the Ministry of Health. The objective is to boost clinical trial activity in Germany.   The proposed changes will relate to the rebate guardrails that were introduced with the Statutory Health Insurance Finance Stabilisation Act (SHIFSA) in 2022 and allow price premiums only for drugs with the highest levels of additional benefit (major or considerable). According to Müller, “in the future, we will link this rule with the manufacturers’ commitment to clinical research” in Germany. Companies will be exempt from the rule if they can prove that an as-yet-undisclosed percentage of patients in pivotal studies were treated in Germany.   The government also intends to make new model contractual clauses specified by the Ministry of Health at least partly compulsory, rather than merely advisory, as previously proposed. This move will be in imitation of the Spanish model of centralisation and standardisation that has been instrumental in massively boosting Spain’s share of global clinical trial activity.   Andreas Gerber, the Managing Director of Johnson & Johnson in Germany, welcomed this move. He noted that German centres recruit patients faster than their counterparts in Spain or France, and binding model contract clauses would streamline the setting up of trials even further. He urged the government to overhaul the AMNOG system—particularly for orphan drugs. In his view, greater flexibility on endpoints would be especially beneficial because endpoints such as overall survival are difficult, if not impossible, to represent in clinical trials for rare disease therapies.   Müller responded that the Ministry of Health is already considering such changes. A conference in November will bring together key stakeholders to discuss potential AMNOG reforms (tinyurl.com/5fztm6eh). He promised there will be reforms, “but it won’t be easy because there are many players with very different interests.” #Germany #drugpricing #rebates #guardrails #AMNOG

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Rare diseases do not mean ‘rare’ symptoms to manage. For people living with generalized pustular psoriasis (#GPP) and their loved ones, the burden of the disease on their lives is tremendous, spreading across their entire lifetime. The CHMP positive opinion is another milestone for people living with GPP, offering continuous treatment to potentially help them prevent, as well as resolve debilitating flares.   GPP is a chronic and heterogenous disease, often leading to isolation and anxiety about future flares. The possibility of managing the condition, including stopping flares before they happen and reducing painful skin symptoms, means people living with the disease will have predictability and control in their lives.   I’d like to congratulate everyone involved – the Boehringer Ingelheim teams, scientists, healthcare professionals, and of course the GPP community, who inspire us every day in our pursuit of a flare-free future. This reiterates our commitment to address high unmet needs in rare diseases across therapy areas. #Dermatology https://lnkd.in/dfs5_HKf

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HCP perspectives: Access challenges in treating diabetes and obesity. Highlights of a candid discussion between specialists about the issues HCPs face in the diagnosis and treatment of diabetes and obesity. GoodRx https://brnw.ch/21wKcXy #partnercontent #HCPs #diabetes #obesity

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𝐑𝐨𝐜𝐡𝐞'𝐬 𝐂𝐓-𝟑𝟖𝟖 𝐀𝐜𝐡𝐢𝐞𝐯𝐞𝐬 𝐁𝐫𝐞𝐚𝐤𝐭𝐡𝐫𝐨𝐮𝐠𝐡 𝐢𝐧 𝐎𝐛𝐞𝐬𝐢𝐭𝐲 𝐓𝐫𝐞𝐚𝐭𝐦𝐞𝐧𝐭 Roche has announced promising results from a Phase Ib trial of its dual GLP-1/GIP receptor agonist, CT-388, designed for treating obesity and type 2 diabetes. Over 24 weeks, participants receiving weekly CT-388 injections achieved a significant placebo-adjusted weight loss of 18.8%. Additionally, 100% of participants experienced over 5% weight loss, with 70% losing over 15% and 45% over 20%. The treatment also normalized glycemia in all pre-diabetic participants. CT-388 showed a favorable safety profile, with side effects consistent with its drug class. These findings highlight its potential as a leading therapy in obesity and glucose control. Further trials, including one focusing on obese patients with type 2 diabetes, are ongoing. Dr. Levi Garraway, Roche’s Chief Medical Officer, emphasized the significant and meaningful weight loss achieved, underlining CT-388's potential as a best-in-class therapy for obesity and diabetes. Given the global obesity crisis, affecting over four billion people and burdening healthcare systems, CT-388 could be a crucial development in addressing this urgent health challenge. CT-388 targets the GLP-1 and GIP receptors, which help regulate blood sugar and appetite. Its unique dual-targeting approach minimizes receptor desensitization, potentially leading to prolonged effectiveness. Roche plans to release further data from ongoing trials in the second half of 2024. With the obesity market projected to grow to $100 billion by 2030, CT-388 positions Roche as a strong competitor against Novo Nordisk and Eli Lilly. Further trials are ongoing, with additional data expected later this year. Source: Roche, Financial Times 16.5.24 #biopharma #insights #glp1 Roche's CT-388 Achieves Breakthrough in Obesity Treatment

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Pfizer announced that the European Commission (EC) has granted conditional marketing authorization for DURVEQTIX® (fidanacogene elaparvovec), a gene therapy for the treatment of severe and moderately severe hemophilia B (congenital factor IX deficiency) in adult patients without a history of factor IX inhibitors and without detectable antibodies to variant AAV serotype Rh74. DURVEQTIX is designed to enable people living with hemophilia B to produce factor IX (FIX) themselves via a one-time dose, rather than multiple intravenous FIX infusions weekly or biweekly with the current standard of care. “There is a substantial medical and treatment burden for people with hemophilia B that receive standard of care today, with frequent infusions and many remaining at risk of breakthrough bleeds that can lead to pain and restricted mobility,” said Alexandre de Germay, Chief International Commercial Officer and Executive Vice President, Pfizer. “DURVEQTIX has shown the potential to offer long-term bleed protection in a one-time dose, reducing or eliminating bleeds for the appropriate patients with hemophilia B. These outcomes and their impact could become potentially transformative for hemophilia B care in the European Union.” Hemophilia B is a rare genetic bleeding disorder that prevents normal blood clotting because of a deficiency in FIX that causes those with the disease to bleed more frequently and longer than others. The standard of care for hemophilia B treatment is prophylactic infusions of FIX replacement therapy that temporarily replace or supplement low levels of blood-clotting factor. Despite prophylaxis and regular intravenous infusions, many people living with moderate to severe hemophilia B are at risk of spontaneous bleeding episodes. The current standard of care also places strain on healthcare systems’ budgets and resource utilization. According to the World Federation of Hemophilia / Fédération mondiale de l'hémophilie, more than 42,000 people worldwide are living with hemophilia B. This conditional marketing authorization is valid in all 27 European Union (EU) member states, as well as in Iceland, Liechtenstein, and Norway. The EC approval follows recent regulatory approvals by the U.S. Food and Drug Administration (FDA) and Health Canada, where it is marketed as BEQVEZTM. #rarediseases #hematology #hemophilia #hemophiliaB #genetherapy #BEQVEZTM #DURVEQTIX #Pfizer #sciencewillwin

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[EMA Refuses Marketing Authorization for Leqembi] On the first day of AAIC 2024, we received a very disappointing news: the European Medicines Agency (EMA) has refused to grant marketing authorization for Leqembi in Europe. The EMA's reasons for refusal were as follows: 1) The main study involved 1,795 people with early Alzheimer's disease, comparing Leqembi to a placebo over 18 months using the CDR-SB score. Leqembi showed improvement in CDR-SB scores compared to placebo (1.21 vs. 1.66 increase). 2) However, the EMA's human medicines committee (CHMP) considered the most important safety concern with Leqembi is the frequent occurrence of amyloid-related imaging abnormalities (ARIA), which can sometimes cause serious events requiring hospitalization. 3) The CHMP concluded that the benefits of Leqembi treatment are not large enough to outweigh the associated risks, leading to the recommendation to refuse marketing authorization in the EU.

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The pay gap: Best paid in pharma still not anywhere close to other industries And 2023 stock returns are all over the place: from -40% to more than 60%. Incidentally, there was no clear connection between pharma CEO salaries and stock performance in 2023. In fact, the correlation coefficient between CEO compensation and stock returns was negative (-0.1345) in a dataset of 14 prominent pharmaceutical CEOs. Selected six CEO outliers in the data at Moderna, Amgen, Johnson & Johnson, Vertex Pharmaceuticals, Eli Lilly and Company and Novo Nordisk for an interactive visual in this article: https://lnkd.in/ga_XVYaM

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Realized you've left it late to create your SDTM Define.xml or unsure how to start? No worries! At Cliniva, we have the expertise, the know-how, and the tools to create it for you. 🌟 📧 Contact us at info@cliniva.ie #CDISC #SDTM #Define #Standards #Programming

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Have confidential rebates for pharmaceuticals in Germany just been killed off? The Bundesrat (Federal Council) has rejected the government’s proposal (tinyurl.com/a48x7run) in the first reading of the Medical Research Act.   The Bundesrat followed the recommendations of its Health Committee. An explanatory statement noted: “The introduction of a secrecy option for the negotiated reimbursement amount leads to a great lack of transparency for the health economic considerations that are necessary today. At the same time, the high bureaucratic and financial cost of equalising the difference is offset by only questionable benefits.”   The procedures for equalising the difference between the reimbursement amount and the actual dispensing price were criticised as unclear. Surcharges paid for the production of drugs in pharmacies or repackaging medicines would also need to be corrected. The overpayment of VAT would also present problems.   The Federal Council remarked that “there is no stipulation that there would be an obligation to provide information on confidential reimbursement amounts to the state monitoring authorities, if required as part of monitoring activities.”   With regard to the growing problem of parallel exports from Germany to other EU Member States, the Bundesrat sees a need for a new solution: “Orientation towards prices in other European countries is still desirable, especially to reduce shortages in individual countries that arise owing to cost-related cross-border flows of goods.”   The health insurance funds were quick to welcome the Bundesrat’s position on #confidential #rebates: Stefanie Stoff-Ahnis, Chair of the GKV-Spitzenverband (National Association of Statutory Health Insurance Funds), said: “Secret prices undermine the principle of economic efficiency. If doctors are deprived of price transparency, they will no longer be able to prescribe economically. If secret prices prevail, they will significantly increase the already rising spending on medicines by many billions of euros. Secret prices mean considerable additional costs for people who pay insurance premiums without improving care. The pharmaceutical industry must not be given this gift to increase their profits.”   Jens Martin Hoyer, Deputy Chairman of the AOK Federal Association, which represents the country’s largest group of health insurance funds, suggested that “hardly anyone seems to think secret prices are a good idea: neither the medical profession, nor private health insurers—and even parts of the pharmaceutical industry—are explicitly against it. This begs the question: who will actually benefit from this in the end?”   Johannes Bauernfeind, Chairman of AOK Baden-Württemberg, remarked: “I consider the drug manufacturers' promise to grant higher rebates to the SHI system to be naïve.”   The pharmaceutical industry has yet to comment on the Bundesrat’s rejection of confidential rebates. It remains to be seen how the government will respond. #Germany

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ASCO 2024 is almost here with abstract titles out few days ago. Interesting updates will be coming across numerous cancers in many presentations. Within Plenary Sessions, chosen for discussion are LAURA and ADRIATIC trials in NSCLC and SCLC, respectively. Also, highly anticipated NADINA trial that Nature Medicine’s annual feature named as one of trials that will shape medicine in 2024. The NADINA clinical trial is the first one to compare neoadjuvant vs adjuvant immunotherapy treatment in macroscopic stage III melanoma preventing recurrence. Results can change need for current adjuvant standard with monotherapy. Also, possibly open deeper discussion beyond melanoma on neoadjuvant vs adjuvant contribution to efficacy in other tumor types. After full text publication goes live on 23rd of May we'll know much more. Link to ASCO program: https://lnkd.in/e_HQDYUu #ASCO2024 #Clinical Oncology #NSCLC #SCLC #melanoma #immunotherapy #targeted therapy #neoadjuvant #adjuvant Link to Nature Medicine publication: https://lnkd.in/exhTwBJ3

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In #ClinicalTrials investigating Antibody Drug Conjugates (#ADC\), worldwide regulators are asking sponsors to monitor for ILD toxicity. Understand the implications and how to meet #regulatory expectations in this quick read by Calyx's Rohit Sood MD., PhD., CFP. https://lnkd.in/e2-8WYC5 #MedicalImaging #Oncology #ChooseCalyx

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Well, well, maybe reading the full paper might have provided Nikolai Petrovsky the full perspective of all the flaws of the study and his conclusion… What was not surprising is the bias seen here in his analysis. Now let’s look at the results, the real ones and draw the real conclusions. With only 461 children included in the primary analysis, only 25 of whom were diagnosed with COVID, this was a tiny observational study clearly lacking statistical power to draw the conclusions Nikolai Petrovsky would want you to believe. As a result of this extremely small sample, with a 95% confidence interval for estimated vaccine efficacy (VE) ranging from -133.7% to 61.8% (wow), it is impossible to draw any conclusion at all on the protection, or lack of for the Pfizer mRNA. Concluding that the vaccine "provided no protection" based on a such a severely underpowered study is simply not justified. Read the actual study, here attached is the link to it. This way you will see how much spin ( and wrong one ) Nikolai Petrovsky is pushing here…LMIC countries should be able to generate scientifically and statistically sound and significant studies. And individuals like Nikolai Petrovsky should be able to distinguish between studies not up to par. Or can he ??? https://lnkd.in/e3a52Qtu

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Report 2 Three reports published by the European Federation of Pharmaceutical Industries and Associations show that disparities in the time it takes for European patients to access new medicines persist. Across the region, the average time to patient access is 531 days. Ranging from 126 days in Germany to 804 days in neighbouring Poland. The data shines a light on the delays and barriers to access that European patients face. Ensuring that medicines reach citizens regardless of where they live in Europe, is not only a shared goal but a shared responsibility. The current situation, where patients in one European country can wait more than 6 times as long as patients in a neighbouring country for the same medicines must be addressed. In the second report, The European Access Hurdles Portal generates even more detailed insights into the reasons behind the delays to patients getting access to medicines. Data in the portal report covers 66 medicines - that received a centrally authorised marketing authorisation between January 2021 and June 2023. The Portal also highlights issues in all the different categories of root causes: health system infrastructure, economic viability, P&R process and value assessment. However, there are differences between regions of Europe: delays in filing in Western Europe were largely due to the value assessment process and evidence requirements, while delays in Eastern and Southern Europe were mostly due to health system constraints and the corresponding impact this has on the economic viability for manufacturers to launch innovative medicines. When looking at why companies have not filed for pricing and reimbursement in the five countries with the lowest filing rate (Greece, Latvia, Lithuania, Cyprus, and Malta), specific reasons stand out. For example, in Greece, ‘Country filing requirements’ were found to be a major contributor to the low rate of filed products; this is likely due to the requirement in Greece that products only be considered for HTA if they are reimbursed in at least five of a list of 11 Western European countries. The granularity of the information demonstrates the value of the Portal in increasing the transparency of root causes of delays so, together we can move to pragmatic solutions that work for patients and help address Europe’s health inequalities.

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