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Access gaps for essential medicines remain stark in low- and middle-income countries. The Access to Medicine Foundation tracks pharmaceutical companies' efforts on healthcare equity to help address this. In their latest report, the Foundation reveals efforts of pharmaceutical companies to monitor the number of patients reached by products Knowing the number of patients reached across a range of health areas is essential so that efforts can be targeted to address access barriers. #AccessToMedicines | #HealthForAll https://lnkd.in/e8TxU4mV

More than 80% of the world's population live in low-and middle-income countries (LMICs), yet still cannot depend on stable access to lifesaving medications for treatable health conditions. Tracking the number of patients actually reached by pharma companies' healthcare products is essential for pinpointing and targeting access gaps. This first-of-its-kind report from ATMF looks at if and how pharmaceutical companies measure the "reach" of their products, giving an overview of the current industry status quo and illuminating areas of opportunity >> https://lnkd.in/eZSb2-EA #healthequity #patientreach #pharma

At The Evidence Base®, we're delighted to, once again, be media partner for the World Evidence, Pricing and Access Congress, Europe's largest gathering focused on #marketaccess, pricing, and evidence in healthcare (March 5–6, 2025, Amsterdam, The Netherlands). The event will bring together over 1500 attendees from the #pharmaceutical, #biotech, and #HTA sectors, featuring 300 speakers across 9 themed tracks. Read our 'Looking ahead' article here. #heor #healtheconomics #outcomesresearch #healthoutcomes #rwe #rwd #realworldevidence #realworlddata #marketaccess #pharma #biopharma #medicalaffairs #healthtechnologyassessment #healthdata #healthequity #healthpolicy #valueassessment

𝗣𝗿𝗶𝗼𝗿𝗶𝘁𝗶𝘇𝗶𝗻𝗴 𝗣𝗮𝘁𝗶𝗲𝗻𝘁 𝗜𝗻𝘃𝗼𝗹𝘃𝗲𝗺𝗲𝗻𝘁: 𝗧𝗵𝗲 𝗞𝗲𝘆 𝘁𝗼 𝗦𝘂𝗰𝗰𝗲𝘀𝘀𝗳𝘂𝗹 𝗗𝗿𝘂𝗴 𝗗𝗲𝘃𝗲𝗹𝗼𝗽𝗺𝗲𝗻𝘁 | 𝗟𝗲𝗶𝗱𝗲𝗻, 𝗡𝗲𝘁𝗵𝗲𝗿𝗹𝗮𝗻𝗱𝘀 | 𝟮𝟬 𝗝𝘂𝗻𝗲 𝟮𝟬𝟮𝟰 𝟭𝟲:𝟯𝟬 - 𝟭𝟴:𝟯𝟬 𝗖𝗘𝗧 A new Netherlands Discussion Group session by CASSS – Sharing Science Solutions is in just over 2 week on the 𝟮𝟬𝘁𝗵 𝗼𝗳 𝗝𝘂𝗻𝗲! 🎉 So, clear you schedules for the afternoon and join us at Leiden University Medical Center. We have an insightful and interesting meeting planned for you! Registration, attendance, and drinks and conversation afterwards are 𝗳𝗿𝗲𝗲 𝗼𝗳 𝗰𝗵𝗮𝗿𝗴𝗲. You only have to register (top right) via CASSS: https://lnkd.in/eJHK4pPC 𝗔𝗯𝘀𝘁𝗿𝗮𝗰𝘁 In our collective endeavor to develop innovative pharmaceuticals to fight diseases, we embark on a journey of discovery, innovation, and the meticulous generation of data. This data eventually converges into a comprehensive new drug application. However, beyond the stringent scientific evaluation and regulatory approval process lies the true litmus test: the patients' experience with the drug. It is essential to consider whether the drug effectively addresses patients' needs, if it alleviates their most burdensome symptoms, if it's tolerable, if adherence to the treatment is feasible, and if the dosing schedule is practical. These are pivotal questions we should ask ourselves throughout the drug development journey. Recognizing the invaluable insights patients provide, it is fundamental to involve them from the get-go. In our upcoming event, Sophie Van Tomme will shed light on global trends in patient engagement and provide concrete examples demonstrating the integration of patient perspectives into the drug development process. To complement van Tomme’s industry perspective, Louise Veltrop-Duits will share the Dutch regulator’s perspective, underscoring the significance of patient participation in clinical research.

So, phages need to get to patients. How does that happen? Traditionally, this was by staged progression through a tightly defined series of clinical trials, from “first in man” safety trials in healthy volunteers (Phase 1) through to multiple large scale trials in patients with the target condition in order to support authorization for widespread use (Phase 3). But that costs a lot of money. A phase 3 trial can involve thousands of patients with typical costs of more than $40,000 per patient. Often cited figures are $4M for phase 1, and $20M for phase 3 – though it can be much higher. A 2021 report by the Congressional Budget Office cites averages of  $28M for phase one and $282M for phase 3. Unsurprisingly, researchers look for alternative routes to get their much-needed therapies to patients. One approach is “compassionate use” (in the US. “expanded access”), which can allow the use of an unauthorised medicine that is still  in development where there is no satisfactory authorised therapy. The extraction of useful data from such studies is heavily outweighed by the need to treat the patient, and in some cases even recording such data can be unacceptable. A more recent development is localised formulation of patient-specific therapeutics, the so called “magistral” approach now used for phage therpeutics in certain European countries. Of course, phage therapy has a long history – more than a hundred years. That led some groups to hope for a much shortened approvals process. But the problem there is that the historical data is often of very limited value when seeking approval for use in a modern setting. As has been noted, vaccines have been around even longer, and they still need to go through the full regulatory process. More on these different approaches later. See: https://lnkd.in/eaSFkmX2. #bacteriophage #phage #clinical trials #compassionate_use #magistral #phagetherapy #thephageadviser drh@thephageadviser.com

"Identity Diagnosis", a Campaign by Edelman Spain to define Rare Disease Patients beyond their Illnesses.   Rare Disease Patients experience a continuous Journey that begins with the complex Search for a Diagnosis, through constant Tests and medical Consultations. During this Period, there is a great emotional Shock, Uncertainty and Difficulties associated with the Lack of Information that cause People with Rare Diseases to often feel excluded and defined by their Pathology, rather than by their Personality.   In this Context, Edelman Spain has collaborated with Alexion, AstraZeneca Rare Disease, and has also had the Support of the Spanish Federation of Rare Diseases (FEDER), to launch the 'Identity Diagnosis' Campaign within the Framework of World Disease Day. An Initiative that advocates the Importance of listening to Patients and involving them in Decision-making about their Illness, in order to more effectively address their Unmet Needs.   Edson Paixão Marcia Moscatelli Rogério Acquaroli Edson Arakaki Antoine Daher Eliana Tameirão Raphael Fernando Boiati Silvio Ferrari Vanessa Tubel Gabriela Romariz Gil Gurfinkel Rosangela Wolff Moro Simone Warmbrand Tcherniakovsky Roberto Giugliani Hélio Osmo   #planejamentoestrategico #strategicplanning #businessplanning #focus #reinviting #businessreinvention #healthtech #innovation #health #healthcare #pharmaceutical #biotechnology #biotech #desenvolvimento #businesspriorities #lifesciences #marketaccess #lifesciences #pharma #pharmaceuticals #heathtechs #governance #businessadvisor #healthbusinesscouncil #healthbusinessadvisor #healthcarebusinessadvisor #disruptivegovernance #raredisease #edelmanspain #edelman #identitydiagnosis #diagnosticodeidentidad  

In a recent article by Cytel, the indispensable role of key opinion leaders in the realm of rare disease clinical trials is illuminated. Rare diseases present unique challenges in drug development, from limited patient populations to complex regulatory pathways. Here are some key takeaways: 1. Expert guidance: KOLs, with their deep expertise and insights, play a pivotal role in guiding clinical trial design and execution in the context of rare diseases. Their involvement ensures that trials are tailored to address the specific needs and challenges of these conditions. 2. Patient advocacy: Beyond their scientific acumen, KOLs often serve as advocates for patients with rare diseases. Their advocacy can influence trial protocols, ensure patient-centric approaches, and ultimately improve the chances of success in clinical development. 3. Navigating regulatory hurdles: Regulatory bodies have unique considerations for rare disease therapies. KOLs, well-versed in both scientific advancements and regulatory landscapes, can help navigate these complexities, accelerating the path to regulatory approval. 4. Collaborative networks: Establishing collaborative networks involving KOLs, researchers, industry stakeholders, and patient communities fosters a synergistic approach to tackling rare diseases. Such partnerships facilitate knowledge exchange, resource sharing, and innovative solutions. The collaboration between pharmaceutical companies and Key Opinion Leaders is paramount in advancing rare disease therapies. Their expertise, advocacy, and collaborative efforts are instrumental in overcoming the challenges inherent in rare disease clinical trials. Read the full insightful article here: https://bit.ly/44Y1jWi #RareDisease #ClinicalTrials #KeyOpinionLeaders #PatientAdvocacy

"𝗧𝗮𝗹𝗸 𝘁𝗼 𝗽𝗮𝘁𝗶𝗲𝗻𝘁𝘀 𝗮𝘁 𝘁𝗵𝗲 𝗱𝗿𝘂𝗴 𝗱𝗲𝘀𝗶𝗴𝗻 𝗽𝗵𝗮𝘀𝗲. 𝗧𝗵𝗲 𝘀𝗼𝗼𝗻𝗲𝗿 𝘆𝗼𝘂 𝘁𝗮𝗹𝗸 𝘁𝗼 𝗽𝗮𝘁𝗶𝗲𝗻𝘁 𝗼𝗿𝗴𝗮𝗻𝗶𝘀𝗮𝘁𝗶𝗼𝗻𝘀, 𝗿𝗲𝗽𝗿𝗲𝘀𝗲𝗻𝘁𝗮𝘁𝗶𝘃𝗲𝘀, 𝗮𝗻𝗱 𝗲𝘅𝗽𝗲𝗿𝘁𝘀, 𝘁𝗵𝗲 𝗯𝗲𝘁𝘁𝗲𝗿 𝘁𝗵𝗲 𝗲𝘃𝗶𝗱𝗲𝗻𝗰𝗲 𝗮𝗻𝗱 𝘁𝗵𝗲 𝗻𝗮𝘁𝘂𝗿𝗲 𝗼𝗳 𝘁𝗵𝗲 𝗱𝗮𝘁𝗮 𝗰𝗼𝗹𝗹𝗲𝗰𝘁𝗲𝗱 𝘄𝗶𝗹𝗹 𝗯𝗲." Stefan Joris, Board Chairman of RaDiOrg – RARE DISEASES ORGANISATION BELGIUM, shared this impactful statement during the RWE4Decisions Annual Symposium, emphasising the importance of integrating patient perspectives early in the drug design phase to enhance the quality and relevance of real-world evidence (RWE). Collaboration, transparency, early involvement of stakeholders – these were central themes discussed in the panel dedicated to launching the new Stakeholder Actions to Generate Better RWE, in which Stefan took part. Find below his favorite Actions ⤵️ and, if you’re curious to find out more about their practical implementation, watch the Symposium recording here: https://lnkd.in/dYW_zCWX 5.1 Ensure that opportunities and resources to develop patient expertise in the field of RWD are clearly communicated to the patient community to develop skills that support multi-stakeholder and patient-centred generation of real-world evidence (RWE) to inform health technology assessment (HTA) and to engage in policy and system developments relating to use of health data. 5.4 Support development of a process for iterative multi-stakeholder dialogues throughout the lifecycle of a medicine to encourage alignment of views on identification, collection, analysis and evaluation of RWD for decision-making. 5.6 Disseminate clear, unbiased, patient-relevant information about RWD and RWE to patient communities, including the value of secondary use of data and information to support Post-Launch Evidence Generation. 📥 Learn more about these actions and explore how you can advocate for patient-centered RWE approaches in your community: https://lnkd.in/dHk93NaG #RWE4Decisions #HTA #RWE

"Identity Diagnosis", a Campaign by Edelman Spain to define Rare Disease Patients beyond their Illnesses.   Rare Disease Patients experience a continuous Journey that begins with the complex Search for a Diagnosis, through constant Tests and medical Consultations. During this Period, there is a great emotional Shock, Uncertainty and Difficulties associated with the Lack of Information that cause People with Rare Diseases to often feel excluded and defined by their Pathology, rather than by their Personality.   In this Context, Edelman Spain has collaborated with Alexion, AstraZeneca Rare Disease, and has also had the Support of the Spanish Federation of Rare Diseases (FEDER), to launch the 'Identity Diagnosis' Campaign within the Framework of World Disease Day. An Initiative that advocates the Importance of listening to Patients and involving them in Decision-making about their Illness, in order to more effectively address their Unmet Needs.   Edson Paixão Marcia Moscatelli Rogério Acquaroli Edson Arakaki Antoine Daher Eliana Tameirão Raphael Fernando Boiati Silvio Ferrari Vanessa Tubel Gabriela Romariz Gil Gurfinkel Rosangela Wolff Moro Simone Warmbrand Tcherniakovsky Roberto Giugliani Hélio Osmo   #planejamentoestrategico #strategicplanning #businessplanning #focus #reinviting #businessreinvention #healthtech #innovation #health #healthcare #pharmaceutical #biotechnology #biotech #desenvolvimento #businesspriorities #lifesciences #marketaccess #lifesciences #pharma #pharmaceuticals #heathtechs #governance #businessadvisor #healthbusinesscouncil #healthbusinessadvisor #healthcarebusinessadvisor #disruptivegovernance #raredisease #edelmanspain #edelman #identitydiagnosis #diagnosticodeidentidad