Our focus on women’s health research means we’re sensitive to the concerns and needs of the female volunteers contemplating clinical trial participation. In order to effectively enroll women, it’s important to acknowledge these volunteers and consider their perspectives in every step of the clinical research process. Regularly engaging women, their physicians, and health advocates helps…
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Cell & Gene Therapy
PDUFA VII: Implications for Developers of Cell and Gene Therapies
One of the biopharmaceutical industry’s most closely watched forthcoming regulatory developments is the FDA’s imminent reauthorization of the Prescription Drug User Fee Act (PDUFA). PDUFA VII, as the reauthorization is commonly known, will cover the fiscal years 2023-2027. Many of the advancements included in PDUFA VII are driven in large part by an increase in…
Clinical Development
Study Start-Up: Why You Really Need to Get This Right
You get one chance to initiate a clinical trial. Botch the start-up and you’ll expend great effort correcting course and playing catch-up. The waste of money and time — commodities that are chronically scarce among the biotech and specialty pharma companies that comprise most of our customer base — can be devastating. That’s why we…
In recent years, the number of approvals for combination products and 505(b)(2) applications has been rising. Combination products consist of at least two constituent parts, each subject to its own statutory and regulatory requirements. Unlike fixed-dose combination therapies, which consist of drug-drug combinations, combination products can include any of the following: The U.S. Food and…
Immunotherapy has led to substantial advances in cancer therapy in recent years. Still, unpredictable response rates and immune-related adverse events have hampered the widespread use of immune checkpoint therapy to treat cancers. To tackle these challenges, sponsors are increasingly looking to combination therapies as a strategy for improving response and overcoming resistance. In our previous…
Cell & Gene Therapy
A Primer on Cancer Immunotherapy Part 1: Goals & Major Approaches
In recent years, immunotherapy has led to substantial advances in cancer therapy. In particular, the immune checkpoint inhibitors — PD-1/PD-L1 and CTLA-4 inhibitors — have revolutionized treatment for certain hematologic malignancies and solid tumors. The U.S. Food and Drug Administration (FDA) has approved six immunotherapies across 19 cancer types and two tissue-agnostic conditions. But widespread…
Beginning in 2017 with the approvals of tisagenlecleucel (Kymriah™) and axicabtagene ciloleucel (Yescarta™), chimeric antigen receptor (CAR) T-cell therapies have changed the treatment paradigm for patients with certain hematologic malignancies. Since those initial approvals, three other CAR T-cell therapies have been approved for various hematologic malignancies, but many challenges still limit the therapeutic efficacy of…
Chimeric antigen receptor (CAR) therapies use CAR T cells, a patient’s own immune cells that are programmed to recognize and kill cancer cells throughout the body. Beginning in 2017 with the approvals of tisagenlecleucel (Kymriah™) and axicabtagene ciloleucel (Yescarta™), CAR T-cell therapies have changed the treatment paradigm for patients with certain hematologic malignancies. Since those…
Oncology & Hematology
New FDA Draft Guidance Provides Insights on Use of PROs in Oncology Trials
Incorporating patient-reported outcomes (PROs) into clinical trials can help sponsors better understand patients’ symptoms and how a therapy will affect their quality of life, and these insights can be particularly valuable in oncology research. However, there has been a lack of guidance to help sponsors ensure consistency and quality when incorporating PROs into cancer drug…
Clinical Development
ANDA or 505(b)(2): Choosing the Right Abbreviated Approval Pathway for Your Drug
For developers seeking to obtain approval for previously approved drug products in the United States, the U.S. Food and Drug Administration (FDA) offers two abbreviated approval pathways — an abbreviated new drug application (ANDA) and a 505(b)(2) application. Both of these applications rely on the agency’s finding of safety and effectiveness for a listed drug,…