A range of factors — including small patient populations, complex manufacturing processes, and lack of specialized expertise — are positioned to both drive up costs and require new options for stakeholder engagement and risk sharing along the development pathway. New approaches in development are needed to support the next generation of novel drugs on the horizon.
Manufacturers, suppliers, and regulators have recognized that introducing new technologies also introduces a degree of unfamiliarity and uncertainty.
This article highlights the diverse interests and range of opinions about pricing innovative and often high-cost therapies, but also demonstrates that most players in the sector see an urgent need for new pricing models to accommodate cell and gene therapy products.
Gene and cell therapy research has progressed despite the considerable challenges that require new approaches to the standard drug-development process and timeline. Here are some alternate development and financing models to consider.
Testing for evidence of microbial contamination in bioprocessing has a long history — and can be expensive, slow, and burdensome. So suppliers, testing facilities, and regulators have been seeking improvements.
Part 2 of this two-part article reviews lessons from Spark Therapeutics’ pivotal program for Luxturna, a gene therapy approved for the treatment of patients with retinal dystrophy associated with confirmed biallelic mutation in the RPE65 gene, and summarize key considerations for the clinical development and commercialization of gene therapies.
With several first-in-class gene therapies now approved, distinct new challenges are now coming into focus. This article provides a high-level overview of the gene therapy pipeline for non-oncology rare diseases and discuss key issues impacting clinical development.
This article posits that the orphan market is actually a collection of seven distinct business model archetypes that are driven by key disease, patient, and market characteristics. By disaggregating and characterizing these business models and their key success factors/risks, readers may be able to focus their corporate strategies to optimize the value and impact of their orphan therapies.
Stem cell researchers can start taking advantage of the power of benchtop cell sorters and their ability to improve results.
Despite progress, the current patient response rates and side effects associated with immunotherapies have created a sense of urgency to more accurately identify which patients would most benefit from a particular treatment option.
As the gene and cell therapy sector develops and its products start to move from development to commercial manufacture — from smaller or academic establishments and development laboratories to larger facilities with greater capacity — the requirement for the technology transfer of these products will only increase.
Looking back at the hurricane season from the perspective of supply chain risk — and specifically risks that have impacted the life sciences supply chain — there are many lessons to be learned.
FY2017 saw another year of increase in the number of drug GMP warning letters issued by the FDA, though not as dramatic a difference as between FY2015 and FY2016. This article presents a detailed summary of those warning letters, as well as a comparison of trends since fiscal year 2013.
Kymriah and Yescarta have overcome the first hurdle to commercialization by receiving the FDA’s stamp of approval. Now, Gilead and Novartis must prove they can handle the supply chain complexities involved with manufacturing and distributing these personalized, time-sensitive therapies to wider patient populations.
In recent years, we’ve seen a rise in regulatory concern over adequately determining the risk factors that challenge aseptic and sterile products processing. There needs to be a renewed emphasis on assurance and controls, including leveraging the best available technologies and conducting thorough, science-based risk assessments of processes.
This article explores some of the policy changes happening now in the regulatory rare disease space as the FDA’s leadership team implements these changes at the agency.
The Orphan Drug Act of 1983 (ODA) has been wildly successful in its primary goal of spurring innovation that has led to lifesaving therapies. Unfortunately, the design of the ODA and several of the incentives the act provides have led to a host of unintended consequences.
Medical breakthroughs in the treatment of cancer have arrived at an accelerated pace. Seemingly each day, a new “next-generation” technology makes headline news, or a CEO touts a treatment as a game changer. While many of these will miss, others will succeed and undoubtedly make a difference in the lives of thousands of patients and their families.