- Drug approval is a costly and extensive process.
- Trials for drug approval include animal studies, safety studies, efficacy studies, and post-marketing research.
- A large number of drugs have gained new approvals in 2019. Some of the categories of these drugs include those within cardiology, dermatology, oncology, infectious disease, and neurology. This article introduces only a collection of the 2019 new drug approvals to date.
Gaining approval for a new drug is both a costly and lengthy process; however, each step is crucial in the development of safe and efficacious products. Research and development costs have been estimated at $2.8 billion, with the entire process lasting approximately a decade. Approval from the FDA is dependent upon an analysis of the target condition and current treatments, risk vs. benefit, and strategies for managing risks.
The development and approval process begins when a company or sponsor develops a new drug compound and would like to seek FDA approval. Animal studies are conducted to gather information on toxicity. After promising preclinical data has been collected from animal models, the sponsor may submit an Investigational New Drug (IND) application to the FDA. The IND contains all preclinical research data, information about the drug composition and manufacturing, and a plan for testing the drug in humans. After the IND has been reviewed and approved, testing in human subjects may begin. Clinical trials are divided into three phases. At the end of each phase, the sponsor and FDA review data to determine if the drug can safely proceed to the next level.
- Phase I: approximately 20-80 healthy volunteers
- Goal: determine safety, including side effects, pharmacokinetics, and pharmacodynamics
- Phase II: approximately 100-300 patients who have the disease/condition
- Goal: obtain preliminary data on efficacy and continue to evaluate safety
- Phase III: 1000-3000 patients who have the disease/condition
- Goal: establish safety/efficacy and compare against current standard treatments
If all clinical trial phases are successful, a New Drug Application (NDA) is submitted to the FDA for review. If approved, the drug becomes available on the market and is monitored over many years. Post-marketing surveillance is important as the full effects of treatment may still be unknown. Investigators are able to collect more information about the long-term effects, effectiveness, and safety of the drug.
In certain situations, other routes for approval are available to expedite the process. Accelerated Approval was implemented in 1992 for promising new therapies that provide therapeutic benefit over current treatments for treat serious/life-threatening conditions. Other expedited approaches have also been established that apply to drugs that treat serious conditions and fill an unmet need, drugs that demonstrate significant improvement over currently available therapy, and drugs that would substantially improve treatment, diagnosis, or prevention of serious conditions.
Since 2015, the average number of new approvals is 43 per year. Previously approved drugs may also receive approval for new indications or formulations. In 2019, the FDA has approved a variety of novel drug therapies, and expanded indications for others. This article contains highlights of notable 2019 drug approvals.5-7
Vyndaqel (tafamidis meglumine) and Vyndamax (tafamidis)
Polivy (polatuzumab vedotin)
Infectious Disease Agents
Dengvaxia (tetravalent live dengue vaccine)
Zolgensma (onasemnogene abeparvovec)
Women’s Health Agents
Previous Approvals with Expanded Indications/New Formulations
- Center for Drug Evaluation and Research. Development & Approval Process (Drugs). U.S. Food and Drug Administration. https://www.fda.gov/drugs/development-approval-process-drugs. Updated June 13, 2018.
- What are the phases of clinical trials? American Cancer Society. https://www.cancer.org/treatment/treatments-and-side-effects/clinical-trials/what-you-need-to-know/phases-of-clinical-trials.html. Published May 3, 2016.
- Joseph A. DiMasi, Henry G. Grabowski, Ronald W. Hansen. Innovation in the pharmaceutical industry: New estimates of R&D costs [abstract]. Journal of Health Economics. 2016;47:20-33. https://doi.org/10.1016/j.jhealeco.2016.01.012. Accessed August 26, 2019.
- Center for Drug Evaluation and Research. CDER’s New Molecular Entities and New Therapeutic Biological Products. U.S. Food and Drug Administration. https://www.fda.gov/drugs/development-approval-process-drugs/new-drugs-fda-cders-new-molecular-entities-and-new-therapeutic-biological-products. Updated February 02, 2018.
- Windle ML. FDA Drug Approvals — 2019 Midyear Review. Medscape. https://reference.medscape.com/viewarticle/916466_1. Published August 9, 2019.
- Lexicomp [database online]. New York, NY: Wolters Kluwer; 2019. http://online.lexi.com.eu1.proxy.openathens.net/lco/action/home. Accessed August 26, 2019.
- UpToDate [database online]. Wolters Kluwer; 2019. https://www.uptodate.com/contents/search. Accessed August 26, 2019.