Immune responses toward therapeutic proteins may cause significant adverse effects, such as loss of efficacy, hypersensitivity, and autoimmunity. For that reason, it is imperative to assess the potential of therapeutic proteins along and beyond the preclinical and clinical development phases. In consequence, an increasing body of evidence is created along the product's life cycle, encompassing multiple and diverse aspects of its immunogenicity potential.
The challenges to the clinical development and commercialization of orphan drugs are many. Although these challenges may be similar to nonorphan drug development and marketing, the solutions are often different in the context of a rare disease with which there is often little medical experience. In this article, six experts working in patient recruitment, clinical development, drug safety, product launch, and commercialization review unique hurdles faced in the rare disease market.
Manufacturers of biopharmaceutical products and medical devices understand that the regulatory approval of their product will bring postmarketing challenges, requiring solutions for continued surveillance of product safety and for demonstrating value and effectiveness based on real-world evidence. Here are several reasons to begin planning premarketing and to conduct postmarketing research with the aim of demonstrating product value.
Gaining access to specialty medications requires navigating multiple complex pathways, including determining insurance coverage options, co-pay amounts, the patient’s total cost of therapy, prior authorizations, and appeals. Obstacles along each path can lead to increased turnaround time and drive script abandonment. Technology brings advancements for removing these barriers, but buyer beware.
Open Enrollment is quickly approaching! With that, nearly every American will have an opportunity to select their insurance options for 2020, which means that your patients may face new hurdles to remain on therapy at the start of the new year. UBC Pathways can help your patients stay on therapy…even when UBC is not your patient support provider.