Ribonucleic acid (RNA)-based therapies are an emerging area of therapeutic development that offer the potential to produce novel treatments for a range of conditions, including several rare diseases. These products, which leverage genetically targeted technology, encompass any product comprised of non-replicating nucleic acids used to modify disease pathways. RNA-based therapies include products that leverage RNA interference (RNAi) and many antisense oligonucleotide (ASO) therapies—which reduce the production of unwanted proteins—as well as messenger RNA (mRNA) therapies, which introduce novel therapeutic proteins.

In a new white paper, Avalere shares findings from a pipeline assessment of RNA-based therapies to characterize the growing RNA-based therapy landscape. In the United States, there are at least 21 Food and Drug Administration (FDA) approved, on-market, RNA-based therapies, as well as a strong pipeline of products in clinical development. More specifically, at least 131 RNA-based therapies are currently in clinical trials, with many more in pre-clinical development.

Avalere explored the ways in which regulatory classifications for RNA-based therapies can influence product development, commercialization, and patient access. Some RNA-based therapies, such as products using RNAi, are regulated as small molecule drugs under a New Drug Application (NDA), while others, such as gene therapies leveraging viral vector delivery systems and mRNA vaccines, are regulated as biological drugs under a Biologics License Application (BLA). While product development and resource investment may often be comparable among these types of therapies, differences in FDA regulatory classifications can create important distinctions during review for approval and during time on market due to exclusivities and pathways for competition.

Small molecule drug and biological drug products are affected differently by policies such as the Inflation Reduction Act’s (IRA’s) Medicare Drug Price Negotiation program. Under the IRA, biological drugs are eligible to be selected for negotiation after 11 years on the market, compared to seven years on the market for small molecule drugs. The differential treatment of RNA-based therapies that are reviewed under NDAs compared to certain gene therapy or mRNA products reviewed under BLAs may introduce new incentives to the market, which over time could influence investment decisions and pipeline strategies.

To learn more, download the white paper.

Funding for this research was provided by Eli Lilly and Company. Avalere retained full editorial control.