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BackgroundDrugs and high-risk medical devices are increasingly likely to receive Food and Drug Administration (FDA) approval through expedited pathways, which has implications for informed treatment consent (i.e., consent in clinical practice).ObjectiveTo obtain expert opinion about the clinical and ethical implications of the increasing availability of new drugs and devices approved through expedited development and regulatory review pathways.DesignQualitative study using individual semi-structured videoconference interviews.ParticipantsNational leaders in medicine, ethics, and law (n=12) with expertise in medical product regulation, payor policymaking, bioethics, physician practice, patient advocacy, public health expertise/advocacy, clinical trials, the pharmaceutical and device industry, institutional review board oversight, and real-world evidence.Main measuresPrincipal themes in 3 domains: expedited regulatory pathways, physician and patient understanding of and reliance on FDA approval, and informed treatment consent.Key resultsRespondents pointed out that more common use of expedited pathways translates to increased reliance on surrogate measures, some with uncertain clinical significance. While expedited development and review can have advantages, participants expressed worry that physicians were unaware when medical products were expedited and did not communicate about uncertainties in knowledge about new drug or device approvals effectively with patients. Many participants felt that informed treatment consent discussions about new drugs or devices should include some explanations of expedited pathways and use of surrogate measures.ConclusionsExperts identified advantages of expediting development and of FDA flexibility in applying its standards to new drugs and medical devices, but highlighted concerns that patients may not be adequately informed about the risks of shorter review times or about uncertainties in the evidence that result. There is a need to identify approaches to ensure effective clinical use of drugs and devices when approved through expedited pathways.

Background Insulin is the primary treatment for type 1 and some type 2 diabetes but remains costly in the United States, even though it was discovered more than a century ago. High prices can lead to nonadherence and are often sustained by patents and regulatory exclusivities that limit competition on brand-name products. We sought to examine how manufacturers have used patents and regulatory exclusivities on insulin products approved from 1986 to 2019 to extend periods of market exclusivity. Methods and findings We used the publicly available Food and Drug Administration (FDA) Approved Drug Products with Therapeutic Equivalence Evaluations (Orange Book) to identify all approved biosynthetic insulin products. Individual products approved under the same New Drug Application (NDA)-e.g., a vial and pen-were considered as separate products for the purposes of analysis. We recorded all patents and regulatory exclusivities listed in the Orange Book on each product and used Google Patents to extract the timing of patent application and whether patents were obtained on delivery devices or others aspects of the product. The primary outcome was the duration of expected protection, which was determined by subtracting the FDA approval date for each product from its last-to-expire patent or regulatory exclusivity (whichever occurred later). We performed a secondary analysis that considered overall protection on insulin lines-defined as groups of products approved under the same NDA with the same active ingredients manufactured by the same company. We also examined competition from follow-on insulin products-defined as products approved with the same active ingredients as originators but manufactured by different companies (approved via a specific drug approval pathway under section 505(b)(2) of the Food, Drug, and Cosmetic Act). During the study period, the FDA approved 56 individual products across 25 different insulin lines and 5 follow-ons across 3 different insulin lines. Thirty-three (59%) of the 56 products were drug-device combinations. Manufacturers of 9 products approved during the study period obtained patents filed after FDA approval that extended their duration of expected protection (by a median of 6 years). Approximately 63% of all patents on drug-device combinations approved during the study period were related to delivery devices. The median duration of expected protection on insulin products was 16.0 years, and the median protection on insulin lines was 17.6 years. An important limitation of our analysis is that manufacturers may continue to add patents on existing insulin products while competitors may challenge patents; therefore, periods of protection may change over time. Conclusions Among several strategies that insulin manufacturers have employed to extend periods of market exclusivity on brand-name insulin products are filing patents after FDA approval and obtaining a large number of patents on delivery devices. Policy reforms are needed to promote timely competition in the pharmaceutical market and ensure that patients have access to low-cost drugs., Author(s): Anders Olsen 1,2, Reed F. Beall 1,3, Ryan P. Knox 1,4, Sean S. Tu 5, Aaron S. Kesselheim 1, William B. Feldman 1,6,* Introduction Insulin remains a central treatment [...]

BACKGROUND AND OBJECTIVES: Adult cancer drugs have historically been exempted from pediatric testing requirements. In 2017, Congress passed the Research to Accelerate Cures and Equity (RACE) for Children Act to expand mandatory pediatric testing to cancer drugs; the law took effect in 2020. With this study, we sought to evaluate how the pediatric testing of molecularly targeted adult cancer drugs changed after the RACE Act. METHODS: In this retrospective cohort study, we used publicly available Food and Drug Administration data to compare pediatric post-approval requirements, trials, and trial characteristics, including timing, in adult cancer drugs before and after the RACE Act. RESULTS: Between 2017 and 2024, the Food and Drug Administration approved 61 adult cancer drugs with molecular targets relevant to pediatric cancer; 40 were submitted before 2020, and 21 were submitted after 2020. The 40 pre-RACE Act drugs were associated with no pediatric post-approval requirements, whereas the 21 post-RACE Act drugs were associated with 15 pediatric post-approval testing requirements. Approximately two-thirds (26/40, 65%) of pre-RACE Act drugs and 57% (12/21) of post-RACE Act drugs were evaluated in pediatric trials. Among pre-RACE Act cancer drugs, pediatric trials were initiated a median of 0.04 years after approval (interquartile range: -3.3 to 1.9 years), whereas post-RACE Act trials were initiated a median of 2.8 years before approval (interquartile range: -4.3 to 0.3 years). CONCLUSIONS: The RACE Act has been associated with greater numbers of pediatric postapproval testing requirements and the earlier initiation of pediatric trials, although early pediatric trial rates appear unchanged. Formalizing pediatric testing requirements may lead to the timely completion of pediatric studies to the benefit of pediatric patients with cancer. [ABSTRACT FROM AUTHOR]

Purpose: Research and regulatory approval for pediatric uses of prescription drugs often lag years after adult approvals, during which time substantial off‐label use of medications in children can occur. We evaluated whether US Food and Drug Administration (FDA) regulatory actions affected the pediatric use of omalizumab, a biologic drug used to treat asthma. Methods: In this serial cross‐sectional study, we identified quarterly cohorts of children (0–18 years) with moderate‐to‐severe asthma within two large national claims databases of those with commercial insurance and Medicaid from 2003 to 2019. Using an interrupted time‐series analysis, we fit segmented linear regression models to identify changes in the incidence of omalizumab use in 6–11‐year‐old children compared with 12–18‐year‐olds after two time points: (1) 2009Q3 when an FDA advisory committee voted against use for 6–11‐year‐old children and (2) 2016Q2 when FDA expanded omalizumab's labeling to include 6–11‐year‐old children. Results: We identified 9298 new pediatric omalizumab users (84% Medicaid). Among 6–11‐year‐old children, the incidence of omalizumab use did not change following the FDA's initial review of evidence in 2009 and increased after 2016 Q2 FDA approval for this age group in both Medicaid (58 per 100 000 children with asthma, 95% confidence interval [CI] 27–89, p < 0.001) and commercial insurance (57 per 100 000, 95% CI 21–94, p = 0.003) compared with 12–18‐year‐old children. Conclusions: The use of omalizumab among asthmatic children aged 6–11 years remained steady after FDA advisory committee concerns in 2009 and increased after FDA expanded the indication to include this population in 2016. Additional market incentives may help to ensure the timely generation of evidence and regulatory approval of medications for children. [ABSTRACT FROM AUTHOR]

Policy PointsWith increasing public attention to cases of inaccurate and misleading laboratory‐developed tests, there have been calls for regulatory reform.To protect patients from faulty laboratory tests, we need a framework that balances comprehensive test review with laboratory flexibility.The Verifying Accurate Leading‐edge IVCT [In Vitro Clinical Test] Development (VALID) Act would have helped ensure laboratory test safety and validity through a much‐needed expansion of Food and Drug Administration (FDA) oversight. However, Congress did not pass the VALID Act in 2022, forcing the FDA to start the regulatory reform process on its own. [ABSTRACT FROM AUTHOR]

The article discusses a 2024 study on the attitudes of patients 65 years and older on the use of routine clinical data in making treatment decisions. Results show participants' interest in such data and in how drugs work for them, and their concerns on confounders like populations' similarity of conditions with them, and on common minor side effects. Also noted are participants' common view that their physicians determine the evidence's applicability to them, and recommend a medication.