Charles Wentworth’s Post

The FDA just announced a significant step forward in the fight against rare diseases with the launch of the Rare Disease Innovation Hub. With over 10,000 rare diseases affecting more than 30 million people in the U.S., around half of whom are children, the need for innovative treatments has never been more critical. Key goals of the hub include: •Serving as a central point for engagement with patient groups, scientific organizations, and industry stakeholders.  •Enhancing collaboration across FDA centers to tackle scientific, clinical, and policy challenges. •Advancing regulatory science through innovative approaches in trial design, biomarker development, and more. •This collaborative model will integrate efforts from existing FDA programs, such as the CDER Accelerating Rare disease Cures (ARC) program and the CBER Rare Disease Program, and will be supported by a newly created director of strategic coalitions, who will ensure community input shapes priorities effectively. A public meeting is planned for this fall to provide more information and gather stakeholder feedback. It is refreshing to see the rare disease patient community having such a prominent seat at the table, which will no doubt be a boon for the development of new therapeutic options. I’m optimistic about the positive impacts ahead. Read the full announcement: https://lnkd.in/dhjUtjuh #RareDisease #PatientEngagement #HealthcareInnovation

Today at the CHEST Annual Meeting, an oral poster presentation prepared by Dr. Christian Sandrock, University of California Davis Health, was presented by InflaRx titled “Vilobelimab in combination with tocilizumab may synergistically improve mortality in critically ill COVID-19 patients: A post-hoc analysis of the Phase III PANAMO study," which presents a subgroup analysis where tocilizumab was used as part of standard of care with vilobelimab. The post-hoc analysis presented shows a significant reduction in 28-day all-cause mortality in critically ill COVID-19 patients who were treated with both InflaRx’s C5a inhibitor vilobelimab and tocilizumab, an anti-IL-6 receptor antibody. The combination may have further potential to improve survival in these COVID-19 patients. In 2022, InflaRx originally reported topline results from the PANAMO Phase III global study, one of the largest 1:1 randomized, double-blind placebo-controlled trials in invasively mechanically ventilated (IMV) COVID-19 patients in intensive care units in adult critically ill COVID-19 patients. PANAMO included a total of 369 patients and was used to support the emergency use authorization (EUA) granted by the U.S. Food and Drug Administration (FDA) in April 2023 for GOHIBIC (vilobelimab) for the treatment of critically ill COVID-19 patients. Find out more about this post-hoc analysis here: https://lnkd.in/dUKkRPYg

Public Policy Projects' Rare Disease roundtable series has been a fascinating programme to be involved in. Through a series of high-level, but practically minded discussions with clinicians, researchers, advocates, and policymakers, expertly steered by Dr Shehla Mohammed and Alastair Kent OBE, we have covered a broad range of important topics in this series. In the latest roundtable, held yesterday, we discussed the need for a better structure to develop effective novel rare disease treatments. Highlights of this lively discussion included: 💡 The need to develop specialist UK centres for (ultra) rare conditions 💡 How we can create mechanisms to increase participant recruitment for rare disease clinical trials? 💡 The importance of educating patients and their families to what's involved for participation in clinical trials 💡 Reassuring patients who leave traditional clinical paths to take part in these clinical trials for novel treatments that there will be a 'clear route back' should they leave the trial 💡 Involving regulators more closely in the rare disease protocol design to explain the challenges of these trials and candidly discuss the potential limitations of the clinical data. 💡 The importance of the pharmaceutical and life sciences industry (of all sizes) to work collaboratively - particularly in the collaborative sharing of early stage (pre-commercial) data. For info, the final roundtable in this series will take place on 16th May. We are also running a rare disease Patient Advisory Group on 2nd May. If you are a rare disease patient, or represent a patient group or charity and would like to find out more information, please reach out to me on LinkedIn or email me at chris.rice@publicpolicyprojects.com.

It was great to attend this roundtable last week organised by Public Policy Projects, as part of a series we are sponsoring, discussing ways in which clinical trials for rare disease treatments in the UK could be improved. A particularly interesting discussion for me was around the high density of clinical trials and product development that we see within certain disease areas, often ultra-rare conditions. Is there more that industry and researchers can be doing to avoid duplication of research, through innovative trial design or collaborative evidence generation? #clinicaltrials #rarediseases #patientcentricity #patientengagement

Advancing Rare Disease Therapies Through an FDA Rare Disease Innovation Hub Public Meeting FDA’s Rare Disease Innovation Hub, in collaboration with the Reagan-Udall Foundation for the FDA, will host a public meeting on October 16, 2024. The meeting will bring together rare disease patient advocates, academic researchers, regulated industry, and other key stakeholders to discuss how the recently announced Rare Disease Innovation Hub can best engage with members of the rare disease community and prioritize its work. During this public meeting, FDA seeks to gather stakeholder input in the following areas: Cross-cutting rare disease-related, scientific, regulatory, or policy issues (those not related to a particular disease or condition) that should be prioritized for consideration by the Rare Disease Innovation Hub Rare disease-specific (but not application-specific) scientific, regulatory, or policy issues that should be prioritized for consideration by the Rare Disease Innovation Hub Rare disease-related activities or initiatives currently being undertaken independently by CDER or CBER that you believe would benefit from being undertaken by the Rare Disease Innovation Hub as a joint activity Approaches that the Rare Disease Innovation Hub should follow for engagement with patients and caregiver groups, industry organizations, and scientific/academic organizations #fda #raredisease #innovation #policyissues #regulatory https://lnkd.in/g5-QNpGU

A New Era For Patient Recruitment & Retention In Rare Disease? Pharma and biotech companies continue to prioritize diversifying their pipelines to address high and rare unmet needs, but rare diseases present challenges in R&D, especially patient recruitment and retention. This article looks at the challenges and promising changes to come... #raredisease #patientrecruitment

I’m delighted to hear that the FDA is set to launch a Rare Disease Innovation Hub which will help enhance collaboration and accelerate the development and approval of treatments for rare diseases. The Hub will bring together various FDA Centres to leverage existing programmes and create a unified strategy for better patient outcomes. It primarily aims to: 🔹 Connect with the rare disease community  🔹 Enhance intercentre collaboration  🔹 Advance regulatory science In a similar vein, LifeArc is also working to improve infrastructure and collaboration within the rare disease ecosystem through our Translational Centres for Rare Diseases. We are investing £40 million in these Centres, which were created to address fragmented expertise, dispersed patient populations, and limited funding, all of which stall rare disease research. Each Centre focuses on unmet needs in different fields including rare respiratory, kidney, and mitochondrial diseases, and a Centre that aims to boost the capacity and efficiency of rare disease trials across the UK. It’s great to see this same collaboration and infrastructure-building happening across the pond, as we all work to unlock new diagnostic tests, treatments, and cures. Read more: https://lnkd.in/eKag_6E9 

Yesterday marked a significant milestone in the fight against Alzheimer’s disease (#AD) as the European Medicines Agency (EMA) recommended Leqembi for treating AD patients. This recommendation comes after a re-examination, limiting the treatment to a specific patient population (those with one or no copies of ApoE4) following an initial rejection. For the first time, European #AD patients will have access to a disease-modifying therapy that holds the promise of slowing cognitive decline in individuals with mild cognitive impairment or mild dementia. This brings a moment of hope to millions affected by this devastating disease. This achievement is also a testament to the remarkable contributions of the European biotech industry. Leqembi was discovered by the Swedish biotech company BioArctic, led by the outstanding Gunilla Osswald and her dedicated team. Their courage, resilience, and determination to innovate in a field overshadowed by two decades of setbacks are truly inspiring. While this is a breakthrough, challenges remain. From the cost of treatment to the infrastructure required for regular MRI scans to monitor ARIA side effects, significant barriers must be addressed. Additionally, the clinical efficacy basis for approval—a 0.53-point improvement on the 18-point CDR-SB scale—raises questions about whether it meets the minimum clinically important difference (MCID) threshold that reflects patient satisfaction. Nevertheless, Leqembi’s success marks a turning point. The growing scientific knowledge base, fueled even by data from “failed” trials, creates momentum for further innovations. With this progress, there’s renewed optimism for deploying capital and expertise to develop next-generation therapies that can overcome the current limitations of #AD treatments. https://lnkd.in/ds3t6pGr

We're delighted to announce a first-of-its-kind partnership with Metrodora Institute to jumpstart the diagnosis and treatment options for a range of complex, chronic diseases including Long COVID, ME/CFS, ALS, Sjögren's syndrome, and many more: https://lnkd.in/ex7X9pjB Together we will develop and validate a new class of diagnostics that provide clinicians with an accurate view of a patient’s risk of disease and, using the molecular profile of underlying disease mechanisms, report back effective prescribing decisions and drug discovery opportunities to improve patient outcomes. The first products developed will be in Long COVID and ME/CFS building on our groundbreaking research into the underlying causal disease biology of these conditions affecting over 75 million patients globally, and that are without effective diagnosis or treatment options. Targeted precision medicine clinical trials leveraging our complementary diagnostic approach are expected to readout results as early as this year. Get the full story here: https://lnkd.in/ex7X9pjB #PrecisionMedicine #DrugDiscovery #DrugDevelopment #ClinicalTrials #Diagnostics #LongCovid #MECFS #Sjogrens #ALS #NeuroImmune #ChronicDisease Action for M.E. Solve ME/CFS Initiative ME/CFS Research Foundation Metrodora Foundation Long Covid Support Long Covid-19 Foundation World ME Alliance

It's an exciting day for everyone at PrecisionLife as we announce a new partnership with Metrodora Institute to make the promise of Precision Medicine clinically actionable in a number of neuro inflammatory chronic diseases. The partnership will accelerate the development of a new class of genotypic #diagnostic tests and clinically validate novel disease mechanisms via #precisionmedicine #drugrepurposing clinical studies. This major new clinical partnership brings focus and resource to a wide range of diseases affecting millions of patients with unmet medical needs including #longcovid #mecfs #als #endometriosis #sjogrens #asthma. Targeted precision medicine clinical trials leveraging our complementary diagnostic approach are expected to readout results as early as this year.