Claude Waddington’s Post

New clinical research entity first to operate across both Australia and New Zealand - 13 site locations across Trans-Tasman for studies in new and emerging therapies Over the past 30 years, 18,000 clinical trials that have been registered in Australia, with the sector contributing $1.4 billion to Australia’s economy annually. Trials are a critical aspect of evidence-based medicine, and are essential for testing how new treatments, tests and vaccines will work. In New Zealand they contribute $146 million to the New Zealand economy. Australia and New Zealand have established themselves as ideal locations to host clinical trials due to their diverse participant populations, sophisticated healthcare and research environments as well as government backed support. Both countries have adopted the International Council for Harmonisation (ICH) Guideline for Good Clinical Practice (GCP) to ensure all trials collect high quality and credible data that is internationally recognised. To capitalise on this unique position and growth, a coordinated multi-site clinical research operation was missing. Now, three of the largest clinical trial organisations across Australia and New Zealand have united to form Momentum Clinical Research – an end-to-end clinical research network with sites that have been operating for over 20 years. Combined, they have 13 site locations across the Trans-Tasman and are set to open more locations to allow more studies to take place and enable everyday people to access new and emerging therapies. In February 2024, New Zealand’s renowned P3 Research Ltd joined forces with Australia’s AusTrials and Holdsworth House to form Momentum Clinical Research. The combined entity can now provide sponsors a single point of contact for their current 13 sites across the two countries, Momentum Clinical Research aims to provide quick responses to feasibility requests, faster study starts and accelerated recruitment – all leading to the delivery of high-quality results for Phase 1b-IV clinical trials. Momentum Clinical Research has become the only Trans-Tasman network of clinical trial sites. Australian Health Journal spoke with AusTrials Research Director, Dr Munro Neville, on the Australian clinical trial landscape more broadly and reasons for creating a Trans-Tasman clinical research organisation. Further growth in the industry is expected, with the Australian Government having pledged $750 million to increase clinical trial activity between 2022-2032. Similarly, the New Zealand Government has invested $98 million into health research funding from 2017-2027 to facilitate the conduct of clinical trials.

Exploring Clinical Trial Phases Let's explore the phases of clinical trials using a detective work scenario: 🕵️♂️ Phase I: Gathering Clues In Phase I, imagine you're a detective collecting clues at a crime scene. You carefully gather evidence, like fingerprints or footprints, to understand what happened and who might be responsible. Similarly, in Phase I clinical trials, researchers gather initial clues about a new treatment's safety, tolerability, pharmacokinetics (PK), and dosage by testing it on a small group of 20 - 80 healthy volunteers. This phase helps investigators determine if the treatment is safe enough to proceed to the next step. 🔍 Phase II: Building the Case Once you've gathered enough clues, it's time to build your case. In Phase II, you expand your investigation to include a larger group of suspects, gathering more evidence to support your theory. Similarly, researchers in Phase II clinical trials study the new treatment in a larger group of 100-300 patients to gather evidence about its effectiveness and side effects. This phase helps strengthen the case for the treatment's potential benefits and guides researchers toward the next phase. (Note: It's common for Phase II trials to reach a dead end due to various reasons, leading to a return to preclinical or Phase 0 research.) 🔎 Phase III: Solving the Mystery In Phase III, you're on the verge of solving the mystery. YESSS!!! You conduct a comprehensive investigation, gathering extensive evidence and interviewing witnesses to uncover the truth. Similarly, researchers in Phase III clinical trials conduct large-scale studies involving thousands of patients (1000-3000) to thoroughly investigate the new treatment's effectiveness against the standard of care. This phase aims to provide conclusive evidence to support the treatment's approval by regulatory authorities. 🔦 Phase IV: Monitoring the Outcome Even after solving the mystery, your work isn't over. In Phase IV, you continue to monitor the situation, ensuring that the culprit remains apprehended and the community remains safe. Similarly, in Phase IV clinical trials, researchers monitor the long-term effects of the treatment in real-world settings after it's been approved for use. This phase helps ensure the treatment's ongoing safety and effectiveness and provides valuable insights for healthcare providers and patients. It's also known as the post-marketing surveillance phase. By using this detective work analogy, the phases of clinical trials become easier to remember: Phase I: Gathering Clues / Safety, Tolerability, PK, dosage Phase II: Building the Case / Effectiveness and side effects Phase III: Solving the Mystery / Effectiveness against standard of care Phase IV: Monitoring the Outcome / Long-term side effects & Post-Marketing Surveillance #clinicaltrials #medicalresearch #healthcareinnovation #patientcare #science #clinicalresearch #healthcarediscovery #medicaladvancements #pharmaceuticals

📑 come let's talk about clinical trials !!! Clinical Trial: A research study that investigates the safety and effectiveness of a medical intervention, such as a new drug, treatment, or medical device, in human participants. Informed Consent: The process by which participants in a clinical trial are provided with detailed information about the trial, including its purpose, risks, and benefits, and they voluntarily agree to participate. Placebo: A substance that has no therapeutic effect, often used in clinical trials to compare the effects of a new treatment to those of a placebo. Randomization: The process of randomly assigning participants in a clinical trial to different treatment groups (e.g., experimental and control) to reduce bias and ensure the validity of the results. Blind (Single-Blind, Double-Blind): Refers to whether participants, researchers, or both are aware of the treatment group assignments. In a double-blind trial, neither participants nor researchers know who is in which group. Control Group: The group of participants in a clinical trial that does not receive the experimental treatment. Their outcomes are compared to those of the treatment group to assess the treatment's effectiveness. Protocol: A detailed plan or set of rules that outlines the objectives, design, and conduct of a clinical trial, including criteria for participant eligibility and data collection procedures. Phase I, Phase II, Phase III, Phase IV Trials: Clinical trials are typically categorized into these phases. Phase I tests the safety of a new intervention, Phase II assesses its effectiveness, Phase III evaluates its effectiveness and monitors side effects in a larger group, and Phase IV monitors the intervention's long-term safety and effectiveness after it is on the market. Adverse Event (AE) or Adverse Drug Reaction (ADR): Any undesirable event or side effect that occurs in a participant during the course of a clinical trial, whether or not it is related to the investigational treatment. Principal Investigator (PI): The lead researcher responsible for conducting and overseeing a clinical trial. Inclusion and Exclusion Criteria: Criteria that define who is eligible (inclusion) and who is not eligible (exclusion) to participate in a clinical trial based on factors like age, gender, and medical history.

Dear Linkedin Family, Today I would like to share some knowledge about Clinical Trials. What are Clinical Trials? Clinical trials are research studies conducted to evaluate the safety and efficacy of medical treatments, interventions, or devices in human subjects. These trials are essential for advancing medical knowledge, developing new treatments, and improving patient care. Clinical trials can involve various types of interventions, including medications, vaccines, medical devices, surgical procedures, behavioral therapies, and preventive care strategies. Clinical trials typically follow a structured protocol, or plan, that outlines the objectives, methodology, participant eligibility criteria, and data analysis procedures. Phases of Clinical Trials – Clinical trials are conducted in several phases to evaluate the safety and effectiveness of a new treatment. Each phase serves a specific purpose and contributes to our understanding of the intervention under investigation. The main phases include: 🌟 Phase 1: Safety and Tolerability In Phase 1, researchers assess the safety and tolerability of a new drug or treatment in a small group of healthy volunteers. This phase focuses on determining the drug's pharmacokinetics, dosage range, and potential side effects. It lays the foundation for subsequent clinical trials by providing crucial safety data. 🔍 Phase 2: Efficacy and Dosing Phase 2 involves testing the efficacy of the drug in a larger group of patients with the target condition. Researchers aim to gather data on the drug's effectiveness, optimal dosage, and potential adverse effects. This phase helps refine the treatment's dosage regimen and identifies promising candidates for further study. 💡 Phase 3: Confirmation and Validation Phase 3 is the pivotal stage where researchers evaluate the drug's efficacy, safety, and overall benefit-risk profile in a large and diverse patient population. This phase often involves randomized controlled trials and aims to confirm the treatment's effectiveness compared to existing standards of care. The data collected in Phase 3 are crucial for regulatory approval and informing clinical practice. 🚀 Phase 4: Post-Marketing Surveillance Phase 4, also known as post-marketing surveillance, occurs after the drug has been approved and made available to the general population. Researchers monitor the drug's long-term safety profile, identify rare adverse effects, and assess its real-world effectiveness in a broader patient population. This phase provides valuable insights into the drug's safety and informs regulatory decisions and clinical guidelines.

#USFDA #LDTs #MedicalLabs FDA wants to regulate thousands of lab tests that have long skirted oversight USFDA has laid out a proposal to begin regulating laboratory medical tests, a multibillion-dollar industry that the agency says poses a growing risk to patients because of potentially inaccurate results. The proposed rule would end decades of regulatory ambiguity and formally bring thousands of tests performed in large laboratories under FDA oversight. The change will help ensure tests used to diagnose cancer, heart disease and thousands of other conditions are safe, accurate and reliable. A growing number of clinical diagnostic tests are being offered as laboratory developed tests without assurance that they work. USFDA has long worried that many tests offered by laboratories are not as accurate or reliable as those that undergo FDA review. What are Laboratory-Developed Tests (LDTs) ? The tests targeted by the FDA's latest action are developed and used by high-tech laboratories, including those at academic medical centers and companies. They include tests for complex diseases like cancer, as well as simpler conditions like high cholesterol and sexually transmitted infections. In the 1970s and '80s, most lab-based tests were "lower risk, small volume" products used mostly for local patients, Over time, laboratory-developed tests have grown into a multibillion-dollar nationwide business, with labs processing thousands of blood, urine and other samples per week from hospitals and clinics. Others advertise directly to consumers - including some claiming to measure the risk of developing ailments like Alzheimer's and autism. Laboratory-developed tests have long skirted FDA oversight, though the agency has always maintained that it has the authority to step in. Debate over regulating the space stretches back to the 1990s, with several government advisory groups recommending greater FDA oversight. Why does the FDA want to regulate them now? FDA officials have long voiced concerns about the accuracy of some tests, pointing to patients who have received inaccurate results for heart disease, Lyme disease, cancer and other conditions. Inaccurate tests can lead to patients getting an incorrect diagnosis, skipping treatments or receiving unnecessary medication or surgery. More than a decade ago, the agency drafted tougher guidelines for the industry, but they were never finalized. What is the FDA proposing? FDA would gradually phase in tighter regulation of lab tests over five years. At the end of the process, most new tests would be subject to FDA standards and regulatory review. New regulations should reduce health care costs due to fewer "unsafe or ineffective tests, including tests promoted with false or misleading claims https://lnkd.in/gM6fiNdG

Rama Venugopal's insights into the USFDA's proposal to regulate laboratory medical tests are both timely and informative. With 30 years of experience in business consulting, management systems consulting, and strategic consulting, Rama highlights the significance of the FDA's move to address a growing risk in the medical industry. The FDA's intention to formally oversee thousands of lab tests that have operated without proper regulation for years is a commendable step towards ensuring patient safety. This initiative, aimed at making tests for various conditions, including cancer and heart disease, safe, accurate, and reliable, is of utmost importance. Rama's post also sheds light on the evolution of laboratory-developed tests (LDTs) from their origins as "lower risk, small volume" products to a multibillion-dollar industry. The lack of FDA oversight in this sector has raised concerns about the accuracy of test results, potentially affecting patient diagnoses and treatments. The proposed regulations, set to be phased in over five years, seek to address these concerns and enhance the quality of healthcare by reducing the prevalence of unreliable tests. This development underscores the importance of rigorous oversight and standards in the medical testing field. Thank you, Rama, for sharing this valuable information. It's a step in the right direction for patient safety and healthcare quality. #USFDA #LDTs #MedicalLabs

Cambridge-headquartered AstraZeneca has launched a global health-tech business, Evinova, to accelerate innovation across the life sciences sector. https://lnkd.in/e72S8iAk Evinova was launched today within AstraZeneca to bring to market digital health solutions that are science-based, evidence-led, and human experience-driven. AZ will combine its deep scientific experience with its digital and AI expertise to serve the wider healthcare community and create new standards for the sector. Drawing on AstraZeneca’s experience of developing novel therapeutics and with insights from thousands of patients and clinical researchers, Evinova will provide established technology solutions to pharma, biotech and clinical research organisations (CROs) to support clinical research globally. Digital transformation is happening at pace within the healthcare industry. AstraZeneca has made strategic investments and significant advances in this space in recent years. This includes developing innovative digital solutions to improve the patient experience in clinical trials and accelerate getting new medicines to patients. Evinova will prioritise bringing to market scaled digital technology solutions already being used by AstraZeneca in 40 countries globally to optimise clinical trial design and delivery. This will seek to reduce the time and cost of developing new medicines, bring care closer to home for patients and reduce the burden on health systems. Evinova will also pursue opportunities in digital remote patient monitoring and digital therapeutics with a pipeline of digital innovations in these areas. Globally-leading CROs Parexel and Fortrea have entered into agreements to offer Evinova digital health solutions to their wide customer base. To accelerate industry adoption and sustain and expand the global reach of its products, Evinova is collaborating with Accenture and Amazon Web Services. Pascal Soriot, Chief Executive Officer, AstraZeneca said: “The future of medicine development can be accelerated with digital solutions. We believe Evinova’s combination of scientific expertise and track record in developing AI-enabled digital technologies at scale, provides a real opportunity to fundamentally improve patient care, drive healthcare transformation and reduce carbon emissions.” Cristina Ortega Duran, President of Evinova, added: “We are excited to bring the portfolio of globally-scaled digital solutions developed to serve AstraZeneca’s drug development pipeline to the wider life sciences community. We believe this will help propel the sector forward in digital health as we know healthcare professionals and regulators need digital solutions that work across pharma companies and support patients broadly. “Coming from within the sector and with proven experience, Evinova is well placed to deliver evidence-led, science-based and human experience-driven solutions with the aim of improving patient experience and outcomes.”

Excited about the Texas Health Catalyst Program of the Dell Medical School at the University of Texas at Austin launching its new Call for Proposals: https://lnkd.in/gTRBPCFM. The IC2 Institute at The University of Texas at Austin studied the impact of the Catalyst and sister Pain-Storming programs. The impact was impressive: increasing innovation outcomes by 3.5X, providing almost $1M in seed funds for new products and companies, linking innovators to mentors throughout Central Texas, and seeing $66 in follow-on funding for every $1 Catalyst provided in seed funds. That is an attractive ROI. The emphasis this year, again led by Ruben Rathnasingham (must mention Nishi Viswanathan, MD, MBA, and Jay Brown for their leadership in the past), is improving the lives and health of those with rare diseases - and with UT's Year in AI initiative https://lnkd.in/gWwXKsxf - encouraging integration of advanced computing into these offerings. As someone with a rare disease, I see the value to encourage innovators and researchers to think outside the blockbuster market mindset.   Indeed, if I put on my old FDA hat and experience in drug development - this focus makes a lot of sense. The Orphan Drug Act of 1983 created a new “long game” strategy for drug development starting with Genentech’s HGH. Using Orphan indications as an initial indication offers a developer can explore many benefits, such as:  - competitive grant dollars for clinical development,  - market exclusivity that often is better than a patent,  - established manufacturing experience and data package, - relatively small clinical trial sizes and budgets, - established human safety and PK/PD data package, - more focused and responsive regulatory review,  - pricing advantages, and  - cost of goods-to-market scale up time for strategic development.    Industry has seen the drug development strategy pivot with an Orphan Drug approval. Often, the drug is tested for efficacy with a common disease. This give companies opportunity to participate in two market opportunities with the benefit of a safety data set, common manufacturing capabilities, and a non-first in human clinical pathway.    As noted/quoted from a recent publication (https://lnkd.in/gYghKzpb),  491 novel orphan drugs were approved between 1990 and 2022. 65% have been approved for a single rare disease, 15% have been approved for multiple rare diseases, and 20% have been approved for both rare and common diseases. Revenue estimates from 2021 show that one-third of the drugs approved for both rare and common indications and 6 percent of rare-only drugs were among the 200 top-selling drugs worldwide.    This is a strategy for drug development is a proven pathway for success. I hope many researchers at The University of Texas at Austin take advantage of this focus and opportunity. #raredisease #orphandrug #research #dellmed #startups #innovation

Summarizing 20 years of experience in Clinical Trials I want this time to touch the topic of surveys. I was always a huge fan of different questionnaires and surveys. Being on different positions in Clinical Trials management I often used wide spectrum of questionnaires and surveys to ask our patients about their status in clinical trials, collect their feedback in the end of their path in a particular trial, or find out how they feel the study is ongoing. Checking tons of surveys during my career of CRA and CLM I became the really strong believer, that most of patients are not answering on required surveys just to reply, they really reflect in that surveys own status, opinion and well being. That is 200% true especially in such complicated and sensitive therapeutic areas like Oncology and Hematology. Analyze of such surveys always gives a lot of data and really matters! But there is also another important group of people, professionals, Study sites' members, whose opinion is crucial to collect as well! We ask them in general not so often, but their point of view on this or that clinical trial is important too! That really helps to improve the way we do the trial, how we design that, how we modernize future trials to make them more effective and less burden for study site teams. We ask them and they are happy to reply. I like that and I see how it works. Our study site staff (our means sites with whome we are working in the trials), as I see, are not so much ready to give their feedback during F2F Investigators Meetings, or similar events, not often they share by their own initiative the situation with CLMs/CRAs who came on visit to sites. But when there is such kind of instrument like survey, especially anonimous one - that may really help them not to be shy to share their own thoughts and even offers, and reply on questions brought in that surveys! Don't hold yourself back in asking centers for their opinion on the study, it will really help both you and the clinics now and later. This is the win-win story, I believe in that. And for sure - do not forget to ask you patients - what do they think about the clinical trials! Good luck!

Myeloma Patients Europe Debuts the Myeloma and AL Amyloidosis European Clinical Trial Navigator Myeloma Patients Europe (MPE) has created an online tool called the Myeloma and AL Amyloidosis European Clinical Trial Navigator to help myeloma, MGUS, SMM, and AL amyloidosis patients access clinical trials across Europe. The Navigator simplifies the search for clinical trials, allowing patients, caregivers, and advocates to find and understand relevant studies. The Navigator has features such as filters, a mapping tool, and plans to expand to multiple languages. The tool is user-friendly and aims to increase access and connectivity for clinical trials in Europe. For more details please click the link! https://lnkd.in/ddgE8FDr #marketaccess #reimbursement #pricing #hta #heor #healtheconomics #medicaldevices #pharmaceutical