Coralie Mansukoski

Here’s an interesting publication detailing how trial efficiency can reshape early-stage development in #biotech... In Phase I studies, every decision affects the development pathway, making trial design and timing critical. By introducing flexibility into these early phases, clinical teams can meet complex study demands without compromising on speed or quality—a balance essential in today’s fast-paced therapeutic field. With streamlined processes, from recruitment through data collection, biotech R&D professionals can better allocate resources, achieve reliable results, and move confidently toward later stages of development. #BiotechR&D #BioTech #BioPharm #EarlyStageDevelopment #ClinicalTrials #TrialEfficiency #DrugDevelopment

3

Emma McGrath attended the short course Technology Assessment for Rare Diseases on Sunday as part of #ISPOREurope 2024. She captures her learnings below. Speakers: Hwee-Lin Wee -Lin Wee, PhD joshua byrnes , PhD Diana Beatriz Bayani, MSc, PhD Session 1: Overview of rare diseases Looking at the case study of Spinal Muscular Atrophy (SMA) Limited research and development to receiving treatments for rare diseases such as regulatory barriers, recruitment and unpredictability. The European Organisation for Rare Diseases (EURORDIS) and the Orphan Drug regulation initiatives to encourage R&D for rare diseases. Singapore’s approach to finance rare diseases addressing the need for an appropriate financing framework, that is both sustainable and will prevent a schism between those who can afford precision medicine and those who cannot. Session 2: Comparing the Experiences of England and Singapore. Key features of rare disease policies, HTA pathways and funding Case Studies: Comparing the experiences of England and Singapore Implications for future policy and practice Session 3: Economic Evaluation of rare diseases Types of economic evaluation Cost-effectiveness planes and quantifying the uncertainty Spotlight on extrapolations and their uncertainty over time Session 4: Multicriteria Analysis (MCDA) requires the aggregation of criteria into a single expression of value. MCDA has been used to organise data and questions relevant to the development of clinical practice guidelines (CPG) for a rare genetic condition, Prader-Willi syndrome (PWS) Pros of MCDA are how it Improves transparency, promotes consistency, Does not replace judgement Cons of MCDA: Requires careful design considerations and is time consuming. #shortcourse #ISPOR #learning #raredisease #economicevaluation #multicriteriaanalysis

13

Quicker mutation identification can significantly impact treatment paths and outcomes for lung cancer patients. Our new members’ survey shows 40% of our community had to wait more than 4 weeks for their EGFR+ mutation identification, with over 10% waiting more than 10 weeks. This highlights the critical need for better testing practices to ensure timely treatment for lung cancer patients. Patients need faster, more reliable testing to improve overall survival.

5

Want to improve your understanding of the key barriers facing #AdvancedTherapies and how we are working to overcome these? ➡️ https://buff.ly/44lGFOu Our experience and knowledge of #cell and #gene therapies has enabled us to identify these barriers which we call “Challenge Themes”, where we focus our innovation activities, and utilise our unique, collaborative approaches to identify and demonstrate effective solutions. Discover the Challenge Themes ☝️ #CellTherapy #GeneTherapy #ATMPs

2

⏳ Last chance to submit an abstract for our upcoming meeting on the use of irreversible modulation in drug discovery: https://lnkd.in/eW4dwG6B Don't miss this chance to share your research at AstraZeneca's new R&D hub The Discovery Centre in Cambridge! 🔬💊💉 #DrugDiscovery #ScientificConference #CallForAbstracts #Pharmacology #Pharmaceuticals #Pharma #ResearchAndDevelopment #IrreversibleModulation

3

🚀 Reflecting on an Inspiring Keynote by Michael Lewis, Professor of Life Science Innovation, University of Birmingham Scientific Director Innovation at #OCT2024 in London 🚀 We had the pleasure of hearing Michael Lewis present his latest insights into the UK clinical trial landscape. In his thought-provoking keynote, "Are We Achieving Ambition?", Michael explored the strides made by the NIHR in advancing clinical research in the UK. Michael highlighted the NIHR’s prioritisation of maternal health research, ensuring this critical area receives the attention it deserves. He also emphasised the significant funding provided to address health inequalities, a crucial step in creating a more equitable healthcare system. Notably, Michael shed light on the NIHR's focus on early diagnosis of dementia and research into motor neurone disease (MND), showcasing their commitment to tackling these challenging conditions. With an impressive £1.3 billion invested in research, the NIHR has significantly bolstered the UK's capabilities. Michael also discussed the strategic focus on learning from COVID-19 and its recovery programmes, building capacity and capability, improving outcomes for long-term illnesses, and strengthening careers in research delivery. There is still more to do to in terms of streamlining research, particularly around approval to site opening and enabling Digital Tools to track, oversee and find patients. He concluded with grading the NIHR as a B- “making progress but much more to achieve”💡 So we are certainly looking forward to raising the grades across the U.K. research landscape !! #ClinicalTrials #UKHealthcare #Innovation #Research #NIHR #MentalHealth #HealthEquity #Dementia #MND #COVID19 #MichaelLewis #OCT2024!

38

2 Comments

Harveer Dev and Simon Pacey with industry partners AstraZeneca are working with patients on #ChangingTheStoryOfCancer. Read how one patient is involved in a trial which could transform #prostatecancer outcomes in future. 👇 https://lnkd.in/ecmR2YFu

17

2 Comments

The next chapter in RNA-based medicine What are RNA-therapeutics, and how will they revolutionise medicine? Julie Anderson answers these questions and tells us about CPI’s role in the industry. Read the blog from CPI here > https://bit.ly/4dFcjLo

1

We are proud to introduce Dr Demi Wiskerke, PhD, Associate Consultant at Scendea and the author of our latest whitepaper, "Regulatory Pathways for Advanced Therapies: Meetings and Designations Overview". - The whitepaper explores how early regulatory interactions and key designations, such as Platform Technology and AMT, can accelerate the development of advanced therapies. Read and download the full whitepaper here: https://lnkd.in/gcy7JvCN - #whitepaper #regulatoryaffairs #drugdevelopment #advancedtherapies

41

2 Comments

Editor Rebekah Jordan spoke to Kevin Kreutter, Ph.D., senior vice president, Drug Discovery, Empress Therapeutics about how the company is using AI to analyse genetic data, to accelerate and refine small molecule drug discovery. "Empress adds a new, orthogonal source of data from the trillions of microbial cells that reside within and are an evolutionarily conserved part of the human body. To give you a sense of scale, our metagenome, comprising the combined genomes of all microorganisms found inside and on the human body, encodes over 170 million proteins." Read more >> https://lnkd.in/erzdU-5B #AI #GeneticEngineering #DrugDevelopment #SmallMolecule

3

Our new episode it out discussing a very important issue - information clinical trials participants of their genetic testing results. Many companies opt-out and decide not to do it but is that always the best decision? You can learn more in the latest episode. #clinicaltrials #alzheimer

2

In a recent podcast, Allucent experts Sugato De, MS (VP, Regulatory Strategy, Head of MedTech) and Alex MacDonald, PhD (VP, Model Informed Drug Development) explored the exciting world of antibody drug conjugates (ADCs) and radionuclide drug conjugates (RDCs), discussing the tremendous growth potential of these targeted oncology therapies, the challenges faced by small and mid-sized biotech companies, and strategies for navigating the complex regulatory landscape. This podcast was hosted by BioPharma Dive and can be accessed here: https://lnkd.in/g8uSZp7X.   #Allucent #BringNewTherapiesToLife #DrugDiscovery #AntibodyDrugConjugates #Radionuclides #Oncology

34

Large-scale cancer screening trials are costly and time-consuming, hindering the progress of new screening tests. A new paper authored by Larry Kessler, Deputy Chair of the MCED Consortium, and members of Cancer Research UK (CRUK), explores the use of surrogate endpoints in cancer screening trials to overcome some of these challenges. The paper examines the current landscape, draws upon lessons learned from therapeutic trials, proposes criteria for the use of surrogate endpoints, and suggests potential candidates. Read the paper to learn more: https://lnkd.in/gZ-N4Z_M

18

Looking to the future at what lies ahead in #cancerresearch, our CSO, Stefano Gullà spoke with Megan Thomas at Drug Discovery World (DDW) to discuss how Artificial Intelligence🖥will play an increasing role in both research and clinical development of next generation cancer treatments! Stefano commented 'With ongoing advances in technology, the landscape is poised for continued innovation and the development of transformative therapies that will improve patient outcomes and ultimately lead to better cancer prevention, diagnosis, and treatment strategies.' 📰 Find the Global Cancer Research Guide on page 40 in #DDW’s Summer issue 2024 here: https://bit.ly/3XGUYgl

20

Cracking the EMA Code: Your Roadmap to European Rare Disease Drug Approval Expanding your rare disease therapy into Europe? Professor Trevor Jones, Partner Rare's Principal Advisor and industry veteran, kicks off our 7-part series "Cracking the EMA Code" with an insightful introduction to navigating the European market. Discover: 💚 Why Europe is a crucial market for rare disease therapies 💚 Key regulatory and market access considerations 💚 Strategies for successful European expansion 💛 Follow our 7-part series throughout the month of June and gain the knowledge you need to bring your life-saving treatment to patients in need. Download the first article now on our website: https://lnkd.in/daDvB-Jb #raredisease #orphandiseases #genetherapy #pharma #biotech #marketaccess #EU #europe #drugdevelopment #patientcentricity

4