
- Applied Clinical Trials-02-01-2020
- Volume 29
- Issue 2
FDA Cites Expedited Programs in Boosting Review Process
Gains in FDA approval of new drugs and biologics last year are based on limited clinical trials and accelerated review programs.
FDA approved many new drugs and biologics last year based on limited clinical trials and utilizing accelerated review programs, notably to treat rare diseases and deadly cancers. In a report on Innovation in Drug Approvals of 2019, the Center for Drug Evaluation and Research (CDER) outlines these important gains, noting its use of expedited programs to advance access to 60% of 48 novel drugs
While last year’s numbers of new molecular entities (NMEs) falls below FDA’s record 59 approvals in 2018, the latest list includes numerous first-in-class therapies and advances in care across a range of conditions, from heart disease, macular degeneration, tuberculosis, osteoporosis, urinary tract infections, and migraine, to sickle cell disease, Parkinson’s disease, multiple sclerosis, and cancers and blood disorders. The report also cites numerous decisions to approve indications for expanded uses and additional patient populations, as well as new dosage forms for already marketed medicines, including the use of innovative adult therapies for children.
These gains reflect CDER’s more efficient and predictable review process, as demonstrated by its success in meeting all user fee goals for timely assessments, reviewing 90% of applications in the first review cycle, and approving nearly 70% of new drugs in advance of other countries. The report points out that advances by sponsors in developing more targeted therapies for vulnerable patient populations, often based on genetic characteristics, benefit from the agency’s increased use of innovative and accelerated review procedures over the last four years.
But while emphasizing gains in expedited drug development and review, CDER Director Janet Woodcock emphasizes that the agency’s “high standards for safety and efficacy have remained unchanged.” This responds to some fears that speedy approvals based on limited studies raise risks related to long-term safety and efficacy. Woodcock states that the agency is maintaining its high approval standards in utilizing innovative methods and new regulatory tools to
Advancing innovation
Support for innovative approaches to drug development also involves FDA public meetings and new guidances on a range of methods and policies for accelerating drug development and clinical studies. These include increased reliance on real-world data gleaned from electronic medical records, particularly for documenting additional indications of a medicine, for developing natural history studies, and to advance novel clinical trial designs for gene therapies.
A final guidance on
The agency also supports flexibility in considering when and how only one clinical trial may provide sufficient evidence on a new drug, as presented in a December draft guidance on
Meanwhile, FDA is taking steps to better monitor the safety and performance of both investigational and approved therapies. The agency has established a more efficient system for sponsors to report electronically adverse events involving study participants to
Ideally, FDA flexibility in clinical trial design and more efficient application reviews will reduce the cost of clinical research and yield less expensive therapies for patients. So far, high launch prices for many critical new drugs benefitting from expedited FDA programs may cloud the larger benefits of these regulatory innovations and ultimately support legislation setting price controls for drugs.
Jill Wechsler is the Washington Correspondent for Applied Clinical Trials.
Articles in this issue
over 5 years ago
Gene Testing: Future Problem or Just a Blip?over 5 years ago
Is Retraining on Good Clinical Practice Guidelines Effective?over 5 years ago
The Benefits of Advanced Imaging Management Systemsover 5 years ago
Trial Non-Reporting a European as Well as U.S. Problemover 5 years ago
Applied Clinical Trials, January/February 2020 Issue (PDF)over 5 years ago
Emerging Biopharma Find Ways to Persevere Strugglesover 5 years ago
Technology’s Role in Managing a Compassionate Use Programover 5 years ago
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