New report shows high-impact R&D happens after cancer medicines are first approved

WASHINGTON, July 10, 2023 /PRNewswire/ -- A new report from the Pharmaceutical Research and Manufacturers of America (PhRMA) showcases nine critical oncology treatments by highlighting the vital role of post-approval research and development (R&D) in advancing cancer treatment options and delivering unprecedented progress in the fight against cancer over the past 10 years.

The report builds off previous research that shows for certain cancer medicines, most of the high-impact clinical research happens after a medicine is initially approved. More than 60% of oncology medicines approved a decade ago received additional indications, with most being received seven or more years after initial approval. That suggests policies that discourage post-approval R&D could severely limit progress and patient benefit.

The report describes five mechanisms through which the full clinical value of a cancer medicine may emerge following its initial approval from the U.S. Food and Drug Administration (FDA):

• Additional benefits within an approved indication: After initial approval, data generated over years of research may prove that the benefits of an oncology treatment in its FDA-approved indication are greater than anticipated at approval.
• Use in earlier treatment line or in earlier disease stage: Cancer medicines are often initially approved in patients with more advanced stages of disease and in those who have exhausted other treatment options. Additional research may reveal over time that the medicine is beneficial before patients try other treatments or in earlier disease stages, broadening the population of patients who may benefit.
• Use in additional types of cancer: Additional R&D, driven by insights into cancer biology, may demonstrate a medicine is beneficial in cancer types beyond those targeted in the initial approval.
• Use in combination with other agents: R&D done once a medicine is on the market may demonstrate a medicine is more effective when used in combination with another therapy than when used alone.
• Use as tumor-agnostic treatments based on biomarkers: After initial approval, additional studies may establish a cancer medicine is beneficial to patients with a specific biomarker?regardless of where the cancer originated in the body.

Unfortunately, as we've discussed before, the Inflation Reduction Act puts this progress in jeopardy by setting the price of medicines before many of these critical advancements can be fully realized. Specifically, the law disincentivizes the investment necessary to conduct post-approval R&D by shortening the timelines by which companies may viably explore this research.

In particular, the law discourages the development of small molecule medicines, which represent the majority of cancer medicines approved by the FDA. That is because the law establishes a "pill penalty" where those medicines ? that typically come in pill or tablet form ? are subject to shorter price setting timelines earlier than other medicines. Small molecule medicines are a critical component of the cancer treatment arsenal because they have the unique ability to target processes inside cells that allow tumors to grow and spread throughout the body.

To see why policymakers should support public policies that reward and incentivize innovation and advance treatment outcomes for cancer patients, read the report and see the nine case studies here.

SOURCE Pharmaceutical Research and Manufacturers of America (PhRMA)