David Chung’s Post

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 

A great initiative to help rare diseases: The rare disease innovations HUB. The Hub will work across rare diseases but will especially focus on products intended for smaller populations or for diseases where the natural history is variable and not fully understood, as we recognize that development of therapies for these conditions can be particularly challenging. The Hub would have three primary functions: Serve as a single point of connection and engagement with the rare disease community, including patient and caregiver groups, trade organizations, and scientific/academic organizations. The Hub will help the larger rare disease community navigate important intersections across the FDA that affect patients with rare diseases, such as medical devices, including diagnostic tests, and combination products. Enhance intercenter collaboration to address common scientific, clinical and policy issues related to rare disease product development, including relevant cross-disciplinary approaches related to product review, and promote consistency across offices and Centers. Advance regulatory science with dedicated workstreams for consideration of novel endpoints, biomarker development and assays, innovative trial design, real world evidence, and statistical methods.

Let's talk about Rare Disease! The FDA has announced their plan to establish a Rare Disease Innovation Hub (the Hub). The Hub will work across rare diseases but will especially focus on products intended for smaller populations or for diseases where the natural history is variable and not fully understood, as we recognize that development of therapies for these conditions can be particularly challenging. The Hub would have three primary functions: -Serve as a single point of connection and engagement with the rare disease community, including patient and caregiver groups, trade organizations, and scientific/academic organizations, for matters that intersect CDER and CBER. The Hub will help the larger rare disease community navigate important intersections across the FDA that affect patients with rare diseases, such as medical devices, including diagnostic tests, and combination products.  -Enhance intercenter collaboration to address common scientific, clinical and policy issues related to rare disease product development, including relevant cross-disciplinary approaches related to product review, and promote consistency across offices and Centers.  -Advance regulatory science with dedicated workstreams for consideration of novel endpoints, biomarker development and assays, innovative trial design, real world evidence, and statistical methods. Read more here: https://lnkd.in/gwdypCir

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

🦓 Needle in the Haystack – How We Can Improve Patient Finding for Rare Disease 🦓 🔶 40 years after the US Orphan Drug Act, around 90% of rare diseases still lack treatment, and diagnosis is often slow and cumbersome. 🔶 To improve the process of drug development in rare diseases and bring new treatments to patients, patient-finding strategies are key. 🔶 While one might think that the unmet need of a rare disease automatically drives demand for a new experimental or approved treatment, the reality is often quite different. 🔶 In a recent article in RARE Revolution Magazine, The Healthonauts' Louise von Stechow explored strategies to improve rare disease patient finding: 🦓 Assemble the Puzzle Pieces: Improved disease understanding through comprehensive genetic characterization, clear and holistic symptom descriptions, and patient journey mapping. 🦓 Uncover Hidden Patterns: Innovative methods such as advanced genetic profiling and artificial intelligence to identify rare disease variants and phenotypic matches (for example, based on facial features). 🦓 Learn from the Experts: Integrating patient and caregiver experiences and patient advocacy into patient-finding strategies. 🧡 Read the full article here: https://lnkd.in/e2f9Gkud #raredisease #orphandrugs #drugdevelopment

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

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

https://lnkd.in/e9QBugY2 An interesting article about the challenges faced specifically by trials targeted at rare diseases. Rare diseases pose a number of specific challenges: - Recruiting sufficient numbers of trial participants - these diseases are rare, so the patient pools available are limited. - Rare diseases can often be severe. Patients can be reluctant to participate if there is a chance they will only receive a placebo. The article discusses use of what is termed Complex Innovative Designs. This is a general term, but such approaches enable the modification of the study design elements dynamically as the trial progresses. These adaptions are based on the emerging data as the trial progresses. The idea is to allow the answering of multiple questions about the drug in question, using fewer resources, and enabling the expansion of the trial pool over time as more patients participate. Of course, such approaches can be applicable in trials with larger patient pools. The benefits are just as applicable. Indeed we saw such techniques being used during rapid vaccine development during COVID-19. The key is to test the drug and specific dose just enough to determine the safety and effectiveness of the parameters, and then adjust. #clinicaltrials #clinicalresearch #clinicaltrialmanagement

🌍 The Rare Disease Treatment Market: Opportunities Amid Challenges! The rare disease treatment market is set to soar from USD 216.65 billion in 2024 to an impressive USD 380.62 billion by 2029, with a CAGR of 11.93%. 🚀 This growth is driven by increasing rare disease cases, innovative therapies, and government initiatives like the US FDA’s Accelerating Rare Disease Cures (ARC) Program. 🔬 Why is rare disease research so challenging?  With over 6,000 rare diseases affecting more than 300 million people globally and its crucial to recognise that, collectively these conditions are not rare. Research is often slowed by small patient populations, lack of data, and limited funding. Each rare disease presents unique complexities, making clinical trials harder to design, and drug development is often riskier and more expensive. At Biorizon Consulting, we have extensive experience overcoming these barriers. Our team offers deep expertise in preclinical trial design, strategic market analysis, and regulatory navigation, ensuring your research and development programs are designed to succeed in this challenging field. Whether it’s tailored strategies for rare diseases or helping you leverage the latest scientific advancements, we provide the support needed to turn these complexities into actionable insights. Let’s accelerate your rare disease research together—contact Biorizon Consulting  for expert guidance in navigating these hurdles!