FDA Facilitates Expanded Access for Cancer Drugs

Lisa Henderson

Last month, I wrote about the disappointing Alzheimer’s drug pipeline. This month, I’m going to look at oncology-which is not disappointing in scientific possibilities, but certainly overwhelming. Oncology is never far from any headline, but the weeks leading up to the ASCO Annual Meeting are a never-ending stream of announcements and trial results. As it should be. Cancer continues to break hearts worldwide, and while we have seen definite improvements in certain cancer diagnosis and prognosis, there are clearly areas that suffer. Let’s not look at the pipeline science,

per se

, I want to bring everyone up to speed on the latest oncology stats and information most applicable to the clinical trials industry practice.

In late May, the IQVIA Institute for Human Data Science released its report “Global Oncology Trends 2019: Therapeutics, Clinical Development and Health System Implications.” Here are the highlights:

• The 2018 launch of 15 new active substances (NASs) bring the total NAS launches since 2013 to 57, with 89 approved indications for 23 different tumor types.

• Within the R&D oncologic pipeline, the most intense activity is for immunotherapies, with almost 450 in clinical development.

• A total of 1,170 oncology clinical trials were initiated in 2018, an increase of 27% from 2017 and 68%  from 2013.

• More than 700 companies have cancer drugs in late-stage trials, including 626 emerging biopharma companies and 28 large pharmas.

To date in 2019, the FDA has approved two drugs for cancer: Balversa from Janssen, for adult patients with locally advanced or metastatic bladder cancer, and Piqray from Novartis, for use in combination with approved endocrine therapy fulvestrant to treat postmenopausal women, and men, with hormone receptor (HR)-positive, human epidermal growth factor receptor 2 (HER2)-negative, PIK3CA-mutated, advanced or metastatic breast cancer.

FDA’s Information Exchange and Data Transformation Program (INFORMED) also inked a two-year research collaboration with COTA, a precision medicine technology company focused on real-world evidence (RWE), for a variety of breast cancer data initiatives. One that stands out for clinical trials is the agreement’s purpose to support FDA’s understanding of applying RWE into modern regulatory decision-making.

In a much-needed move to address the current disorganization around fulfilling expanded access requests for unapproved cancer therapies, the FDA’s Oncology Center of Excellence launched a new pilot program. A call center-called Project Facilitate-will be a single point of contact where FDA oncology staff will help physicians treating patients through the process to submit an expanded access request for an individual patient, including follow-up of patient outcomes. Prior to the pilot program launch, expanded access requests arrived at multiple places within the FDA and were forwarded separately to FDA oncology or hematology divisions.

The pilot program includes a central office for oncology requests so that FDA can follow up on individual requests and gather data. The phone number is 240-402-0004 and email is OncProjectFacilitate@fda.hhs.gov.

Lisa Henderson is Editor-in-Chief of Applied Clinical Trials. She can be reached at lhenderson@mmhgroup.com. Follow Lisa on Twitter: @trialsonline