Eli Lilly and Company’s Post

Patients with life-threatening conditions need access to investigational medicines outside of clinical trials. Expanding Early Access Programs can help. Patrick Larcier, PharmD, MBA, Senior Director of Strategic Product Development Solutions at PharmaLex discusses the value of these programs in a guest piece for pharmaphorum. Learn about the impacts of the real-word data they give us here: https://ow.ly/si4550QtHre

The Niemann-Pick type C (#NPC) community has faced significant challenges over the years relating to treatment access. I've spoken with parents whose children cannot access medication. Children whose medication was stripped from them. Children whose lifespans are limited. Zevra Therapeutics is working to advance research and treatment in NPC. The company is currently seeking approval of arimoclomol. While many families frequently laud the impact of arimoclomol on their child’s development, the FDA has not yet seen the same benefits. In 2021, the FDA denied the drug’s approval, citing the need for additional qualitative and quantitative data. Gathering additional data and robust clinical evidence can be challenging, though, considering the small patient population. But, good news: the FDA has accepted Zevra’s New Drug Application (NDA) resubmission for arimoclomol. The Prescription Drug User Fee Act (PDUFA) date is set for September 21, 2024. I recently spoke with Daniel Gallo, PhD about the hurdles encountered during the NDA submission process, the trial data that supported the NDA resubmission, and why it is imperative to include the voice of the patient and caregiver in everything the company do 🔗 https://lnkd.in/eryRJHku #NiemannPickDisease #Arimoclomol #ZevraTherapeutics #InterviewSeries #RareDisease #MedicalWriting

The Praxis trial is a great example of a fully-decentralized trial, and these unicorns can be found when we look "beyond pharma" at how academic researchers are designing and executing studies today. But no trial with an investigational medicine or device is "siteless" -- to comply with GCP and existing regulations there is a "site", it's just a question if the participant is obligated to physically appear there for visits. https://lnkd.in/dyKjMd8Y

In her new Forefront article, Janelle Sabo from Eli Lilly and Company argues that, to ensure that clinical trial innovation continues, regulators, industry, and other stakeholders must continue to embrace a shared vision for modernizing the innovation ecosystem in which clinical research is governed and conducted. "As a result, today, we have safe and effective COVID-19 vaccines and a host of new therapies, all products of the innovations—from mobile recruiting efforts to telemedicine visits to direct shipping of investigational products—that allowed clinical trials to continue. Importantly, the process also demonstrated that well-controlled trials can incorporate these innovative approaches while meeting Food and Drug Administration (FDA) standards for data integrity, minimization of research bias, and patient protection." Read the full article here: https://bit.ly/3xkDhby

At the Duke-Margolis Institute for Health Policy conference, the Director of CDER at FDA discussed the future of clinical trials, showcasing significant strides towards regulatory modernization and efficiency. The FDA's dedication to integrating innovative methodologies in drug evaluation and research was emphasized, with key takeaways including the integration of adaptive trial designs and digital health technologies to streamline drug approval processes. The establishment of a translational medicine team within the Office of New Drugs highlights the FDA's focus on accelerating therapy approvals, especially for rare diseases. Additionally, the FDA's modernization of inspection protocols and enhancement of cross-sector collaboration are among its strategies to align with the dynamic landscape of clinical trials. The groundbreaking approval for a drug targeting Friedreich's ataxia underlines the FDA's embrace of real-world evidence and adaptive regulatory approaches. Furthermore, future FDA strategies include enhancing collaborations with NIH and CMS to leverage real-world data for better drug monitoring post-approval. For professionals and stakeholders in the clinical trials and regulatory sectors, these developments signify a pivotal era of innovation and collaboration. Stay up-to-date with the latest in #ClinicalTrials, #FDA, #RegulatoryInnovation, #DrugApproval, #RealWorldEvidence, and #TranslationalMedicine by visiting the full article here.

A 2023 Tufts Center for the Study of Drug Development (Tufts CSDD) survey among several hundred sponsor companies found that only 27% are routinely soliciting patient input into protocol design decisions. While stakeholders throughout the #clinicalresearch industry are well-intentioned in wanting to support #patientengagement, many factors are contributing to a high level of reluctance. Find out what these factors are: https://bit.ly/3Q4LEy2 #clinicaltrials

The failure of Relyvrio in its phase 3 clinical trial is unquestionably a terrible blow to ALS patients and their community. The data from the phase 2 trial engendered hope for improved quality of life and longer time with loved ones, and for that hope to prove unfounded is deeply painful. The clinical trial participants, site clinicians and administrators, drug sponsor Amylyx, all of its employees and its scientific advisors: they all invested of themselves and their resources together to develop and test this therapeutic and they are all to be commended. This is not the first drug to get this far down the path and then fail, and it won’t be the last, but it is heartbreaking each and every time.   It’s hard to find a silver lining in something so deeply disappointing. But it’s important to appreciate that our drug evaluation system did work in this instance. Relyvrio was approved on the basis of early but meaningful clinical trial evidence that it could help people facing a fatal diagnosis without a cure and with only a very short window for intervention. The risk/reward calculus for this kind of disease demands that we offer patients options earlier on the path to fully validated proof of benefit than would be justified for other diseases and conditions, and the FDA studied Relyvrio’s phase 2 results to ensure they met the appropriate hurdle. Amylyx has not tried to spin the negative phase 3 results. The FDA has the authority to force withdrawal of drugs that fail trials like this one, but in this instance it won’t have to: to the credit of the company and its leadership, Amylyx is voluntarily taking the drug from the market yet will provide it at no cost to patients already taking it who want to continue.    There are no bad actors here, but yet a tragedy. We trust Amylyx to release all of the clinical trial data that the field can learn from as it pushes ahead, and we look forward to the day when we remember this as just one chapter in the story that ends with the end of ALS.

While there certainly are good aspects of the #EUCTR, including a streamlined and centralized regulatory submission process, many clinical trial sponsors are facing challenges. Read Elizabeth E. Bodi, RQAP-GCP’s take on the panel, “Real Life Experience: the Good, the Bad, and the Ugly One Year into the EU Clinical Trial Regulation,” led by Mamta Puri-Lechner and joined by Dana Niedzielska, Carrie Melvin, Samantha Z., and Danielle Pelletier during Halloran’s CORE East conference. If you can relate to these stories, you are not alone. https://hubs.la/Q02gZFP90 Contact Halloran and share your EU submission challenges. We’re here to partner with you on your Investigational New Drug (#IND) application and regulatory strategy. #ClinicalTrials #RegulatorySubmissions

📰 #MTCMemberNews 📰 🤝 U.S. FDA has granted #MTCMember Theradaptive approval for phase I/II clinical trials! ✅ Check out the article to learn more - https://lnkd.in/eXVDxDbk

Explore the latest advancements in #RWD, #RWE and #HTA as featured on The Evidence Base® during September 2024. This month, featuring news from Proscia, Truveta, HealthVerity, Center for Innovation & Value Research, Panalgo, COTA, FDA, European Medicines Agency, Flatiron Health and the NHS. #heor #healtheconomics #outcomesresearch #realworldevidence #realworlddata #marketaccess #pharma #biopharma #medicalaffairs #regulation #regulatoryaffairs #healthtechnologyassessment #healthdata #healthequity #healthpolicy