Beatriz Perales Zamorano

Ascentage Pharma announced that updated results from three studies of olverembatinib, have been released in posters at the 2024 European Hematology Association Hybrid Congress, taking place in Madrid, Spain. Olverembatinib (HQP1351) is an oral, third-generation BCR-ABL1 tyrosine kinase inhibitor (TKI) developed by Ascentage Pharma for the treatment of chronic myeloid leukaemia (CML), acute myeloid leukaemia, acute lymphoblastic leukaemia (ALL) and solid tumours, including gastrointestinal stromal tumours (GIST). Olverembatinib is an ATP binding-site inhibitor of wild type BCR-ABL1 kinase and a broad spectrum of BCR-ABL1 mutants, including mutant T315I, which confers resistance against all first- and second-generation TKIs.  Chemenu has been working to develop more compounds for drug discovery. Here comes the building blocks we can provide: https://lnkd.in/gaVnBaRP #Olverembatinib #HQP1351 #GZD824 #TKI #CML #buildingblocks

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Is CAR-T-cell therapy sustainable for Spain’s National Health System (SNS)? What can be done to enhance outcomes with these treatments? And what inequalities in access are emerging across the country? These questions have recently been addressed by specialists in the field (tinyurl.com/35packda).   Adrián Alegre, Director of the CAR-T programme at La Princesa University Hospital in Madrid, believes these treatments are sustainable: “First of all, they are administered only once, so they are finite therapies, in contrast to other expensive anti-tumour combination options that are maintained until relapse. Centralisation by an expert committee in the Ministry guarantees the indication for patients who meet the criteria. Moreover, the payment agreement is risk sharing, that is, payment for clinical results.” In his view, CAR-T-cell therapy has “cost-benefit advantages, although it would be useful to carry out pharmacoeconomic studies comparing it with prolonged therapies with new strategies, such as bispecific antibodies.”   Adrián Mosquera, a haematologist at the Santiago Health Research Institute (IDIS), sees a need to invest in research to distinguish patients who will respond to CAR-T-cell therapy from those who will require combination therapy to achieve complete remission: “There are patients who relapse, even with an initial response. If we can understand or anticipate this lack of optimal response, we can explore other strategies, such as the intensity with which conditioning is performed, other bridging therapy strategies or consider alternative maintenance, to increase the effectiveness of the CAR-T cells and improve response.”   Growing regional inequalities in access to CAR-T-cell therapies are a cause for concern. Manuel Muñoz, head of Haematology at the Regional University Hospital of Málaga, says: “Depending on the criteria of one regional sub-committee or another, a patient may or may not have access to CAR-T therapy. In some cases, approval varies depending on the community where you live, and that is becoming worrying. It is a situation that did not occur at the start of CAR-T and that we are now seeing and need to address.”   Variations can relate to bridging therapy: some autonomous communities allow the use of polatuzumab, whereas others restrict access. Muñoz says: “The Ministry should determine where these deviations are, to correct them. For a start, the guidelines could be updated. CAR-T therapy began in a particularly limited way with ministerial commissions, regional sub-committees, pharmaco-clinical guidelines. However, scientific evidence and experience move faster than regulations, so depending on how strict the interpretation is, there is a risk of deviating from scientific evidence and depriving a patient of therapy.” #Spain #marketaccess #cartcelltherapy

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When patient drop-out continues to be a challenge in today's clinical trials, obtaining a 95% patient retention rate is such a huge success! We want to celebrate it by sharing with you how Almirall obtained 95% patient retention in the AKtive study, a Phase IV, three-year study in which patients were required to report outcomes periodically and avoid sunlight or UV light exposure. Read the case study by clicking here: https://lnkd.in/edUvVrFJ

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Spain’s long-awaited draft Royal Decree on HTA has finally been published. It sets out the broad framework for the future of HTA in the country, rather than providing detailed methodological directions.   STRUCTURE A Governance Council of 21 members, including six representatives of the autonomous communities, will oversee the HTA system.   Two Offices for the Evaluation of the Efficiency of Health Technologies—one for medicines, the other for other technologies—will conduct evaluations.   The Health Technologies Positioning Group will carry out a final assessment based on the evaluation reports prepared by the Offices as well as other relevant information. Where the Positioning Group cannot reach a consensus, decisions will be made by simple majority.   SCOPE The HTA process will be aligned with EUnetHTA’s nine domains and split into clinical and non-clinical parts.   Clinical evaluation reports will contain conclusions regarding overall added clinical value—using the methodology proposed by the Office and approved by the Governance Council—but will not make recommendations for subsequent decision making.   Economic evaluations will include an assessment of cost-effectiveness and budget impact. Value assessment will take account of relative effectiveness, societal value and impact on HRQoL.   Evaluations of clinical and non-clinical aspects will not contain judgements regarding the positioning within the healthcare system, pricing, reimbursement or coverage by the common portfolio of services.   If an evaluation identifies evidence gaps, measures may be proposed to solve them; these should be pragmatic, proportionate and viable.   TIMELINES Ninety calendar days will be allowed for completion of clinical evaluation reports. If an EU joint clinical assessment report is available, this timeline will be reduced to 20 calendar days. Similarly, 90 calendar days will be allowed for the non-clinical evaluation. These deadlines will be extended by 30 calendar days if it is necessary to collect additional information: manufacturers will have 10 calendar days to provide such information.   OTHER NOTABLE DETAILS Manufactures will have opportunities for early dialogue with the Office for the Evaluation of the Efficiency of Medicines.   Health technology developers will be required to provide reliable data on the costs of R&D and production, as well as the sources (public or private) of funding for these activities.   Companies will receive draft evaluation reports and have opportunity to correct technical/factual inaccuracies and indicate content they consider to be confidential but will not be permitted to make observations on the results of evaluations.   The use of real-world data will be encouraged, and the Ministry of Health will maintain information systems that allow the evaluation and monitoring of results.   A public consultation on the draft Royal Decree runs until 20th September 2024 (tinyurl.com/mrp33xa9).   #Spain #HTA #reform #royaldecree

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So excited to be part of this project... 😃 AEFI - Asociación Española de Farmacéuticos de la Industria has become a member of the #CriticalMedicinesAlliance of the #EuropeanCommission. Today, we have attended the #inaugural meeting, where the objectives of the Alliance, the challenges and the paths forward have been discussed. The Critical Medicines Alliance (CMA), set up in January 2024, is a consultative mechanism bringing together relevant stakeholders from #EU Member States, key industries, the civil society, and the scientific community. The Alliance aims to identify key areas and priorities for action, proposing solutions to strengthen the #supply of critical medicines in the EU, ultimately enhancing efforts to prevent and address #shortages effectively. From AEFI we are so proud to be part of this Alliance, and will work hard to contribute and to add value, on the interest of our members and of the whole healthcare industry in Spain. #biopharma #compliance #regulation #quality #supplychain #patients #future

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Artificial intelligence (AI) will play a growing role in pharmaceutical P&R in Spain in the coming years. Analítica Farmacia (AF; Pharmacy Analytics), an AI-based tool to generate predictive models to support P&R procedures and supply management, has been in development by the Ministry of Health (MoH) for the last two years.   According to César Hernández, Director General of the Common Portfolio of SNS and Pharmacy Services (DGCCSF), the government wants to move towards a healthcare model based more on demand than supply, and AF “can be an enormously valuable resource to help manage this move to a strategic planning environment.” He believes AF could streamline the pharmaceutical P&R process in three ways: (1) an earlier start; (2) a shorter duration because of greater predictability; (3) managing decisions as a continuum rather than an “all-or-nothing” phenomenon.   Carlos Martín Saborido, an advisor to the DGCCSF, believes a key objective of AF is to facilitate “precision reimbursement” for the growing number of precision medicines. At a recent conference, he explained that AF uses information from a wide range of sources, including horizon scanning, medical literature databases, clinical trials, grey literature, conference presentations, press releases and media reports. The aim is to “combine clinical evidence and process it with economic information to obtain economic impact scenarios,” according to Martín Saborido. Information can be filtered and presented in lists with a traffic light rating of the quality of evidence, risk of bias and level of uncertainty. ChatGPT is then used to produce a draft summary.   Martín Saborido notes that the pharmaceutical industry has extensive experience in the use of long-term scenario models, so the healthcare system needs to “copy this use of predictive analytics for its decision-making, as this is what has allowed the industry to make strategic decisions.” Consumption data from Seguimed, expenditure data obtained by internal calculation or from the Ministry of Finance, and epidemiological data feed into budget impact scenarios and analyses by indication, disease area or therapeutic group. Where short-term analyses are required, data may be obtained from DARWIN EU or the European Health Data Space, in addition to consumption, expenditure and epidemiological data. Spain’s regional healthcare systems can thus be better prepared for the medium- and long-term costs of new medicines.   #Spain could learn from other countries' use of #AI to manage dedicated funds for new drugs. Martín Saborido cited the example of the Innovative Medicines Fund in England. AI can make it much easier to move drugs into and out of managed entry agreements. The practical challenge is the need for patient-level data, which Spain hopes to have in place “in the near future.”   Martín Saborido notes a need for metadata. Dashboards must be developed to provide “a granular level of data digitisation” for decision making. #drugpricing

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Cardiovascular problems affect individuals of all genders, and it’s crucial to understand the risk factors involved. Smoking, high cholesterol, hypertension, and being overweight significantly increase the likelihood of developing heart disease.​ ​ At ESTEVE, prevention is key, especially in alignment with our purpose of improving people’s lives. That’s why we encourage you to trust healthcare professionals and steer clear of common myths.​ ​  👉 In a recent video, Dr. Enrique Santas, a cardiology specialist at the Hospital Clínico Universitario de Valencia, shares verified insights on this critical topic.​ Watch the video to learn more and empower yourself with knowledge for better heart health!​ ❤️ https://lnkd.in/eaQ36fFG

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A new early access system, longer periods for instalment payments for gene therapies and greater use of centralised procurement—these are some of the ambitions of the Spanish government, according to César Hernández, the Director General of the Common Portfolio of SNS and Pharmacy Services.   Speaking at the Farmaforum 24, he disclosed that his primary objective is to “rebalance the pharmaceutical system” in three respects: improving access to innovations so that they make investment in drug development profitable, promoting the launch of generics and biosimilars after patent expiry and ensuring that medicines that have lost profitability but still have significant value remain available.   The Ministry of Health wants to adopt measures for early access. Spain currently has “access to medicines in special situations,” a system that covers four situations—compassionate use, drugs authorised elsewhere but not in Spain, off-label prescribing and the use of medicines from marketing authorisation to reimbursement—and benefits around 40,000 patients per year. The Ministry reportedly wants to create “an environment for certain products, an access route, when the negotiation with the pharmaceutical company and the decision of the General Directorate of Pharmacy are pending.”   Hernández also sees a need to alter the current regulatory framework for instalment payments—particularly to enable outcomes-based agreements for single-administration gene therapies to be extended. He said: “If we want to pay for a gene therapy at a high price, but it involves achieving certain outcomes or replaces alternatives at the same price that we already spend money on, the payments should be spread over more than 5 years, which is what is currently allowed by law.”   Greater use of centralised procurement—at both national and EU level—is of interest. However, an obstacle in Spain is that 98% of the country’s healthcare budget is currently spent by the autonomous communities.   Changes to the reimbursement system and the internal reference pricing system (RPS) will be included in the forthcoming update to the Guarantees Law. According to Hernández, “we have already raised prices for medications that were in the RPS, so the way is open; the problem is that this dynamic can be generated only when a reference price order (RPO) has already been published.”   Hernández indicated that he is “not saying that health spending or pharmaceutical spending has to fall, but it must be appropriate; there is a need for screenings that allow early diagnosis to avoid suffering for patients, but also unnecessary health costs.”   The government wants to ensure that Spain is a major centre of drug development and production in Europe: “If many countries are competing with us or want to have policies that promote it, it is because it is an important pillar of the health, well-being, productivity and quality of life of our citizens.” #Spain #earlyaccess #drugpricing

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Today, the European Medicines Agency recommended not granting a marketing authorisation for Leqembi (lecanemab). The main reason for that decision was, according to the EMA statement, that "the observed effect of Leqembi on delaying cognitive decline does not counterbalance the risk of serious side events associated with the medicine, in particular the frequent occurrence of amyloid-related imaging abnormalities (ARIA), involving swelling and potential bleedings in the brain of patients who received Leqembi." You can see more details about the EMA statement at their website. At Ace Alzheimer Center Barcelona we believe this decision entails major concerns for the clinical, medical, and care community as well as for the field of research. First, the access of European patients to the same options and opportunities as the citizens from other countries. Secondly, the effect it can have in terms of research and investment in research. See more of our position in the open letter we sent to the EMA representatives: https://lnkd.in/d46T7fMt #Lecanemab #EMA #Leqembi #Alzheimers

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The possibility of a three-phase access process in Spain has been outlined in Diariofarma, based on recent conversations with national experts (tinyurl.com/5evtndta). The government is due to publish new royal decrees on HTA and #pricing and #reimbursement in the coming months.   ASSESSMENT The conjectured first phase would be assessment. For drugs subject to EU joint HTA from January 2025 (i.e., cancer therapies and ATMPs), this first phase would likely begin with analysis of the EU joint clinical assessment (JCA) report and determination of the drug’s positioning relative to therapeutic alternatives already available in the National Health System (SNS). For drugs not yet subject to EU joint HTA, the first phase could begin with an assessment similar to that currently done for therapeutic positioning reports in #Spain.   Many new drugs will likely have to undergo health economic evaluation. The Ministry of Health recently published a new “Guide to the Economic Evaluation of Medicines” (tinyurl.com/ytw3tfkc).   The Spanish Medicines Agency (AEMPS) would likely be responsible for the clinical assessment work, and could also handle the health economic evaluation, though it might delegate that activity to the Directorate General for the Common Portfolio of Services of the National Health System and Pharmacy or a network of external experts.   POSITIONING Based on the clinical and economic assessment reports, the second phase would likely be to determine the use of a new medicine in the SNS in relation to social needs, ethical considerations, unmet medical needs, organisation of the healthcare system, etc.   A wide range of stakeholders—potentially including autonomous communities, healthcare professionals, patients (or citizens) and the industry—could play a role in determining a drug’s positioning. It remains to be seen if a new technical commission will be established.   DECISION MAKING The last phase would be to reach a decision regarding public reimbursement of a medicine and determination of its price relative to the positioning decided in previous phases and taking account of information supplied, such as the criteria in Article 92 of the Law of Guarantees (disease severity, needs of specific groups, therapeutic value and cost-effectiveness, budget impact, degree of innovation).   The Interministerial Commission for Drug Pricing (CIPM) would likely play the leading role in this phase, though the Ministry of Health—and possibly other ministries (e.g., Industry, Economy, Finance)—might also be involved. Diariofarma suggests it would not be appropriate for the CIPM to propose changes to recommendations already made regarding positioning.   The CIPM is expected to consider factors such as disease severity, needs of specific groups, therapeutic value and cost-effectiveness, budget impact and degree of innovation in making a decision. The exact criteria will likely be defined in a forthcoming revision of the Law of Guarantees. #marketaccess #HTA

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Aurelio Arias from IQVIA sheds light on the critical role of collaboration and buyer pressure in tackling indirect emissions within the #pharmaindustry. In 2024, commitments will be set, and #WHO’s Operational Framework for Sustainable Healthcare Systems aims to guide and support climate-related initiatives. Since this report was published, even more countries have committed to low-carbon healthcare. Learn more https://bit.ly/3vYRCd9 #Sustainability #Healthcare #ClimateAction

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"Navigating Market Access in MENA: A Guide for Pharmaceutical Companies" Unlock the potential of the MENA region with this comprehensive guide for pharmaceutical companies. Learn effective strategies for navigating market access, understanding regulatory environments, and leveraging local partnerships. Discover how to conduct thorough market research and adapt your products and services to meet the specific needs of MENA consumers. Understand the importance of compliance with local regulations and how to engage with regulatory authorities. Explore best practices for building a robust distribution network and optimizing logistics. Equip your company with the insights and strategies needed to successfully enter and thrive in the dynamic MENA pharmaceutical market. #Regulatory #Intellectual_property #Market_entry_barriers #Market_research #Authorities #Supply_chain_management #Spain #KPIs

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Happy to learn about this #AI powered solution called "Analítica Farmacia" to facilitate #PricingAndReinbursement #decisionmaking specially in #precisionmedicine, curious to learn more about it's implementation and rollout from the National DGCCSF in #Spain across our complex #Healthcaresystem structure!

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Yesterday, we concluded the 2nd Congress on Rare Diseases for Latin America and the Caribbean. The congress emphasized strategies to strengthen rare disease policy development and implementation, improve access to diagnostics and treatments, and fostering innovation. Experts discussed the importance of integrating rare diseases into national health agendas through stronger regional collaboration, political will, and patient advocacy. The event also highlighted the need for comprehensive policies, equitable access to care, and the role of reference centers in improving care. Let us continue to promote collaboration across sectors, countries, and regions to ensure better outcomes for people living with rare diseases, their families, and caregivers. Watch a brief recap of Day 2 here: https://lnkd.in/g_quAvaH #AHF #RareDiseases #AHFCongress #ERCAL

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Key discussions emerged from the Veeva R&D Summit at the Marriott Madrid Auditorium, where Novo Nordisk's senior vice president, Thomas Senderovitz, emphasized the critical need for pharmaceutical companies to be involved in shaping AI regulations. Alongside James Weatherall from AstraZeneca, Senderovitz highlighted the necessity of harmonizing global AI regulations to facilitate uniform drug trials across different regions. Key Points: 🔹 Advocacy for increased pharma involvement in AI regulation 🔹 Challenges posed by disparate global regulatory systems 🔹 Anticipation of the FDA’s forthcoming AI guidance and the need for updates in Europe 🔹 Novo Nordisk’s advancements in AI, including the development of the AI super-computer Gefion Read more on this drive to establish unified global standards, ensuring more efficient and effective AI-driven research and innovation within the healthcare sector. https://lnkd.in/dffUNFcY #VeevaSummit #PharmaAI #NovoNordisk #AIRegulation #HealthcareInnovation #GlobalStandards

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Are coming to ICPP2024 in Pamplona, Spain? Join us at our Symposium: "The roles of heme & diseases related heme deficiency"

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