This is a challenge for every modality, of course, but it’s especially critical for gene therapy, where we have to make decisions early on about whether to include patients with genetic variants in clinical trials — and understand what that might mean for the biomarkers we use to measure efficacy. Current Challenges. First, we talk through the ideal, mediocre, and restrictive labels for our investigational therapy. Additionally, gene therapy is applicable far beyond just rare diseases, and gene therapies are being developed for indications affecting larger numbers of patients, including cancer, ophthalmological conditions, CNS disorders including Alzheimer’s and Parkinson’s, and many more. These questions are important to think through early, as the answers can help shape trial design. Sadly, the treatment Jesse was given led to his death, shaking up the world of gene therapy. We therefore want to put our collective brain trust to work early to think through innovative models for reimbursement. Get the latest articles from Cell & Gene delivered to your inbox. Opportunities and Challenges in Cell and Gene Therapy Development*. These conversations involve post-approval hypotheticals, but they do not involve promoting investigational products. © 2020 Syneos Health. After all, even after we as an industry nail the science, we will face substantial clinical, regulatory, and logistical challenges in bringing these therapies to market. “We were helicopter parents, ready to plot our babies’ lives through college and beyond. As it stands for many genetically modified cell therapies, a patient’s genetic sample is sent off to one of a very small number of central manufacturing plants around the world to be edited. Our Company, including a Contract Research Organization (CRO) and Contract Commercial Organization (CCO), is purpose-built to accelerate customer performance to address modern market realities. Will patients ultimately want to have choices at certain stages, such as the flexibility to choose from among several conditioning regimens? The short duration of meaningful revenue These clinical trials demonstrate that the recent attention being paid to gene and cell therapy is not just hype. Sometimes targeted cells stop the new genes from entering. In truth, though, this is just the beginning. Confronting the Challenges of Reimbursement. And the time to start thinking about the label is years earlier, during the design of clinical trials. Beyond eligibility, considering the final patient experience is crucial in protocol design. Gene therapy as a field has greatly advanced since Jesse received his treatment, with seven approved gene therapies available in the U.S. or EU market as of July 2020 and hundreds more in development (Figure 1). For many of the 400 million people worldwide affected by one of more than 7,000 types of rare diseases, gene therapy is the onlyoption that provides a glimmer of hope for diseases that are currently poorly treated. Optimising manufacturing is still a big challenge for gene therapy. For Jesse, the novel therapy showed promise of a chance at a new life—one free from ornithine transcarbamylasedeficiency (OTCD), a genetic disease that had plagued him his entire life. Promise of personalized medicine. Do It Yourself: Make a detailed note on the harmful effects of gene therapy. And we need to understand who among them would be ideal candidates for the therapy if approved, and how to address the many questions that are bound to arise. Terms of Service apply. The changing industry landscape. Here are the challenges I’m thinking about now: We all want approved therapies to be available to every patient who can benefit. Or, do we expect that every patient will follow precisely the same protocol? To get people thinking about these issues, I like to run a thought exercise with each program team. given the lack of a stable base from which to build. Other gene therapy pioneers have tested pay-for-performance and value-based models; these may well be successful, but it’s still too early to declare them the definitive answer for all gene and cell therapies, especially as the range of approved indications and technologies broadens. But we know that expanding the supply chain to a global, commercial scale — even in rare diseases with relatively small patient populations — is very different. We need to start having discussions now with commercial and government payers and with the external voices who help shape public policy on pricing. We need to learn more — from them, not from textbooks — about the strengths, limitations, and burdens of the standard of care. This is a foundational element for all our work. This presentation will highlight some of the existing analytical challenges and provide guidance on development of an appropriate strategy to help overcome these. As I look back at these recommendations, I must admit, they seem staggering. At AVROBIO, we’ve sought to build strong supply chains for clinical trials, and we’ve sought to give patients more flexibility in scheduling with innovations such as improved cryopreservation to extend drug product shelf life. Every step along the way needs to be carefully planned. We need to understand what might motivate them to take a leap of faith and enroll in a clinical trial for an investigational gene therapy. Privacy Policy and It’s never too early to start thinking about the end goal. Although the first gene therapies have been approved and offer significant clinical benefit, they have run into challenges that require rethinking the drug development and delivery system across key stakeholders. You can withdraw your consent at any time. Yet that approach could lead to an overly restrictive label and crimp access. The “bolus-like” revenue curve associated with a first gene therapy presents a challenge to achieving sustainable growth . The goal is to ensure we are all aware of the future ramifications of every decision we make now. A correct diagnosis takes an average of five years, with a 40 percent error rate.

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