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Regulatory Focus – Study: Breakthrough designated drugs led to better clinical outcomes for patients with NSCLC

Regulatory Focus – Study: Breakthrough designated drugs led to better clinical outcomes for patients with NSCLC

Drugs granted a breakthrough therapy designation (BTD) by the US Food and Drug Administration (FDA) were more likely to yield better outcomes for patients with non-small cell lung cancer (NSCLC) compared with drugs that didn’t carry the designation, according to a recent analysis published in Clinical Cancer Research.

“Our findings show that the criteria for a breakthrough therapy designation are identifying products that provide maximum benefits for patients,” Jeff Allen, PhD, president and CEO of Friends of Cancer Research, stated in a press release. “Efforts to expedite development and review of breakthroughs are working to help address high unmet needs, and this data suggests that additional efforts to ensure timely access to these products are warranted.”

Allen and colleagues compared clinical outcomes and clinical guidelines in the National Comprehensive Cancer Network (NCCN) Drugs and Biologics Compendium for NSCLC drugs between January 2013 and October 2021. They identified 52 drugs approved during that time that met the criteria, which included 41 drug applications where breakthrough therapy designation was granted and 11 drugs without breakthrough therapy designation.

Overall, 16 (31%) approved drugs had a primary endpoint or a co-primary endpoint of overall survival; of these, 14 drugs had breakthrough therapy designation and 2 drugs did not have breakthrough therapy designation. The analysis showed drugs with breakthrough therapy designation had a 31% reduction in risk of death compared with a 15% for drugs that did not have breakthrough therapy designation, and breakthrough therapy designation drugs also reduced risk of progression by median 48% compared with median 41.9% for drugs without the designation.

Among drugs with breakthrough therapy designation, there were more category 1 evidence recommendations by the NCCN, indicating a high quality of data and consensus, compared with drugs without breakthrough therapy designation (59% vs. 50%). More breakthrough therapy designation drugs were also considered a preferred intervention by NCCN than drugs without breakthrough therapy designation, the authors said (59% vs. 10%).

Expedited coverage at CMS for breakthrough therapy drugs?

Allen and colleagues argued the findings of the analysis show there should be a process to expedite coverage with the Centers for Medicare & Medicaid Services (CMS) for cancer drugs granted breakthrough therapy designation. While an expedited approval process exists under FDA’s breakthrough therapy designation, they argued, the process is not expedited on the CMS side.

“An expedited program at CMS would not include automatic coverage, but rather enable processes to ensure BTD therapies and novel technologies sufficiently meet approval and reimbursement requirements more quickly, and as appropriate, have processes in place for ensuring a product is covered at the time of approval to not delay patient access,” they wrote.

One method to allow for expedited coverage of drugs granted breakthrough therapy designation would be to follow the processes outlined in the Parallel Review pilot program that helped FDA and CMS coordinate review and coverage of medical devices. This process would involve FDA alerting CMS when a new product has received a breakthrough therapy designation and the ability for product sponsors to participate in an Expedited Coverage pilot that would determine whether the product provides a benefit for Medicare beneficiaries, the authors explained.

“Expediting development is a resource intensive process for both the FDA and sponsors, and more drugs are approved using BTD and/or other expedited pathways each year. For the processes proposed above to be successful, CMS will need additional resources to support their involvement,” Allen and colleagues said. “A coordinated, well-supported, and timely process for determining coverage of BTD products is necessary to ensure the value brought by BTD facilitates earlier patient access to effective treatments.”

Clin Cancer Res Collins et al.