Thanks to advances in science, notably the completion of the Human Genome Project 20 years ago, we are able to treat diseases more effectively than ever before. Researchers have more sophisticated tools not only to identify new drug targets, but also to make drugs to hit those targets. A wide variety of therapeutic agents help make these drugs, including chemically synthesized small molecules; larger molecules made of amino acids like peptides, proteins, and antibodies; and nucleic acids like RNA or DNA. Choosing among these approaches requires looking at the target attributes. Is it inside the cell or outside? Is the target an enzyme, a gene, or a lipid particle? Do we need to block the target or stimulate it? Science is — and should be — the guide to determine the optimal approach for patients.
That’s why, to researchers like me, a provision in the recently enacted Inflation Reduction Act is puzzling. For no clear reason, it draws a distinction between large and small molecule medicines. As part of the IRA’s Medicare price control provisions, price negotiation for small molecule medicines is allowed nine years after Food and Drug Administration approval compared with 13 years for large molecule biologics. There is no scientific reason for this distinction, and it will have a real and detrimental impact on drug discovery and patient care. Nine years is not enough time to recoup the deep investments into small molecule R&D before government price controls take effect. As a result, companies will deprioritize small molecule programs, lowering the potential to create drugs using these technologies.
Congress should correct this imbalance by allowing negotiation after a full 13 years for both small molecule medicines and their large molecule counterparts. Without this parity, research in small molecule medicines will languish. And that will hurt patients with challenging diseases. Estimates from 2017 suggest that 85% of disease targets were “undruggable.” But, because of their size, precision small molecules can reach a number of these targets.
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