Trends and Issues Shaping the Gene Therapy Industry
The gene therapy field is on the cusp of something special in realizing the transformative potential of gene therapies for patients and their families. After a year as president and chief operating officer of Orchard, and nearly 20 years of leading biotech companies through the often challenging but rewarding path to commercialization, I’ve spent some time reflecting on the unique trends and issues specific to bringing gene therapies from the academic to the commercial setting.
From my perspective, here are some of the key trends with the most formative potential impact on the industry, as well as insights on how Orchard is positioned for commercial execution and continued leadership in the development of hematopoietic stem cell (HSC) gene therapies for a range of severe inherited diseases.
Redesigning payment, reimbursement and market access structures
The advent of gene therapies has necessitated efforts to overcome payment and access barriers that exist due to the chronic care delivery model—which did not envision one-time, potentially curative therapeutic options. In the U.S., we have recently seen momentum on a federal and state level that may allow a gene therapy manufacturer to enter into certain types of value-based payment (VBP) arrangements while beginning to deal with best price reporting issues and other complexities that date back several decades. Ensuring that the statutes and regulations addressing VBP allow for enough flexibility to accommodate the nuances associated with each therapy, such as patient population size in therapies for ultra-rare conditions, will be an important consideration in the years ahead. Additionally, much work remains on the operational front with payers, particularly in the U.S., to address questions such as the portability of these payments when a patient changes insurer.
Lastly, the types of gene therapies we at Orchard are developing are administered in highly specialized, qualified treatment centers with disease and transplantation expertise. From a patient access perspective, this means close collaboration with advocates, treatment centers and payers to support patient travel, lodging and other logistical needs as well as reimbursement structures that allow treatment centers to care for patients across borders.
Driving efficiencies in manufacturing processes
I envision the emphasis on driving efficiencies in manufacturing processes, alternative conditioning regimens and screening technologies will accelerate as we seek to mature the sector. Today, many processes remain relatively cost-, time- and/or labor-intensive, limiting our ability to scale overall commercial infrastructures to support the treatment of conditions affecting larger patient populations. This is an area of intense focus for Orchard, especially as we begin to explore the use of our HSC gene therapy approach in larger indications. We must invest today in innovative manufacturing technologies, including novel transduction enhancers, stable producer cell lines and closed automated processing of drug products, to improve manufacturing efficiency over time.
Prioritizing diagnostic initiatives
We also need to support screening initiatives to identify newborns with rare genetic diseases who may benefit from approved treatments. The newborn screening systems in Europe and the U.S. have a variety of intricacies and time-intensive requirements for ensuring a condition is placed on a local screening panel. In addition to supporting pilots that provide critical data for newborn screening to be recommended at a national, regional or local level, Orchard is also exploring creative diagnostic programs and supporting critical education efforts to ensure children can be identified as early as possible.
Continuing business development transactions and partnerships
On the business development front, we are seeing more and different types of arrangements emerge between academia, small biotech and big pharma—from straightforward M&A activity to more creative licensing deals. There is also greater interest in collaboration and deal-making much earlier in the development lifecycle, with innovative structures in place to address the higher risk of investing in earlier stage assets. As we continue through 2021, I anticipate this trend will strengthen and grow as each big pharma company continues to evolve and augments its gene therapy strategy.
At Orchard, 2021 will be a year of continued execution and scientific progress, coming off the heels of our first EMA regulatory approval as a company. As we finalize European launch plans in the first part of this year, our focus is on building a strong commercial infrastructure that can serve us well today and as we grow in the future. This will include qualifying select treatment centers, engaging with payers and actively advocating for newborn screening modernization that could support early diagnosis. In addition to our continued commitment to supporting the metachromatic leukodystrophy (MLD) community around the world, we also remain committed to advancing our programs in Wiskott Aldrich syndrome, mucopolysaccharidosis type I (MPS-I) and mucopolysaccharidosis type IIIA (MPS-IIIA) with urgency for patients in need.
In addition, I am particularly excited for the company to continue exploring the application of HSC gene therapy in a growing range of larger indications—an important piece of Orchard’s evolution as a company. To date, we have treated more than 160 patients across seven diseases in clinical studies using the HSC gene therapy approach, and we possess more than a decade of follow-up data from the earliest treated of those patients. Building on this understanding and experience, we recently announced new research programs in less-rare neurodegenerative and immunological genetic diseases, including frontotemporal dementia with progranulin mutations, amyotrophic lateral sclerosis (ALS) and NOD2-Crohn’s disease, a genetic subset of the Crohn’s population. It’s exciting to be at this point in our journey, where we are ready to demonstrate the opportunity for this unique business model.
I believe this is just the beginning of the gene therapy revolution. At Orchard, we are committed to thinking through the trends, challenges and considerations we need to address now to build a foundation that will potentially transform the lives of more people suffering from certain severe, often-fatal inherited conditions in the future.
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