Regulatory Challenges In Drug Approval: Balancing Innovation And Patient Safety

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Overview of drug approval processes
Innovation vs. safety: A regulatory balancing act
Evolving regulatory landscapes
Challenges in fast-tracking approvals
Patient safety and advocacy
Global perspectives on drug approval
Further reading

Navigating the complex landscape of drug approval presents significant regulatory challenges, requiring a delicate balance between fostering pharmaceutical innovation and ensuring patient safety.

Image Credit: Panchenko Vladimir/Shutterstock.comImage Credit: Panchenko Vladimir/

Overview of drug approval processes

The Center for Drug Evaluation and Research (CDER) within the Food and Drug Administration (FDA) is primarily responsible for evaluating the safety, efficacy, and potential toxicity of new drugs before that can be sold within the United States. Comparatively, within Europe, the European Medicines Agency (EMA) is responsible for authorizing medication approval through either a centralized or national route.

Worldwide, pharmaceutical and biotechnology companies, physicians, and academic professionals investigate tens of thousands of substances yearly for their potential to treat diseases. Only a small number of these agents will move beyond in vitro and in vivo testing to being evaluated in humans.

Within the U.S., companies, research institutions, and other organizations interested in moving their compounds to clinical testing in humans must submit an Investigational New Drug Application (IND).

After an IND is submitted, the FDA and a local institutional review board (IRB), comprising scientists and non-scientists in hospitals and research institutions overseeing clinical research, must approve the application before clinical studies can begin.

The pathway to clinical trials is similar in Europe, in which medicine developers must submit specific data on their novel drug to the EMA, which then conducts a thorough assessment of the data to determine the safety, efficacy, and quality of these medications.

Innovation vs. safety: A regulatory balancing act

The role of regulatory agencies like the FDA and EMA is to objectively assess a drug's benefits and known risks before it can be approved for marketing. However, these regulatory processes can positively and negatively impact the pharmaceutical industry, with one study citing reduced market innovation due to preapproval regulatory stringency.

Thus, regulatory flexibility that encourages creativity while ensuring the quality, efficacy, and safety of new drugs is needed to support innovation in drug development effectively.

To this end, various efforts have been made to modernize regulatory standards, some of which include amendments to the Federal Food, Drug, and Cosmetic Act, as well as providing special designations to accelerate the approval of drugs for treating serious conditions.

Evolving regulatory landscapes

Over the past several years, significant technological advancements have allowed for the development and introduction of digital health, targeted medicines, and regenerative medicine, including cell and gene therapies, into the clinic.

To accommodate these advancements and ensure their adequate evaluation, federal agencies like the FDA and EMA have implemented various approaches to modernize how medical drugs and products are reviewed.

To this end, between 2017 and 2020, FDA’s CDER implemented various initiatives to modernize the New Drugs Regulatory Program. The six primary objects of this modernization included advancing scientific expertise during the regulatory approval process, the integration of early assessments to ensure that new drugs meet both legal and regulatory requirements, systematically monitoring the benefits and risks of drugs both before and after their approval by the FDA, recruit new staff to support the New Drugs Regulatory Program, standardize both regulatory and business processes to support scientific efforts, and ‘knowledge management,’ to identify and distribute relevant information to improve decision-making processes surrounding the approval of new drugs.

The EMA has also proposed several new regulations to increase the availability, accessibility, affordability, and sustainability of drugs, as well as support drug development conducted by European pharmaceutical industries.

One key aspect of this proposal is to reduce the timeframe of the standard approval process from 210 days to 180 days, as well as adopt a “rolling review” system that will allow regulators to examine clinical trial data as it becomes available rather than waiting for all clinical trials to be complete.

The EMA has also proposed extending patent protection for non-orphan drugs from 11 to 12 years. However, this change will also reduce market exclusivity for a new drug from 10 to eight years.

​​​​​​Image Credit: Mongkolchon Akesin/​​​​​​Image Credit: Mongkolchon Akesin/

Challenges in fast-tracking approvals

From preclinical testing to approval, researchers estimate that it takes an average of 12 years for a new drug to be fully approved within the United States. However, in certain cases, ‘Accelerated Approval’ can be granted for a new drug to treat serious or life-threatening conditions, particularly when the agent offers a superior clinical benefit compared to currently available treatments.

Importantly, after a drug has been granted accelerated approval and enters the market, the drug manufacturer is required to conduct post-marketing clinical trials to verify the clinical benefits associated with the use of this drug. The FDA could withdraw the drug's approval if these trials fail to confirm the predicted clinical benefit.

In addition to this process, the FDA offers several other designations that can accelerate the approval of a new drug for treating serious conditions. These include ‘Fast Track,’ ‘Breakthrough Therapy,’ and ‘Priority Review’ designations.

The Fast Track process allows drug companies to accelerate the approval of drugs for the treatment of serious conditions that are meeting an unmet medical need.

Typically, there is no current treatment available to treat these conditions or the novel therapeutic has the potential to provide superior benefits as compared to any available therapy.

The Breakthrough Therapy designation also accelerates the review of drugs to treat serious conditions based on preliminary clinical evidence demonstrating the benefits of this treatment over currently available therapies.

Comparatively, the Priority Review designation asks the FDA to evaluate drugs in six months, as compared to the standard 10-month period, that have the potential to significantly improve the treatment, diagnosis, or prevention of a serious health condition.

Like the FDA, the EMA also offers an accelerated assessment to reduce the time required for the EMA’s Committee for Medicinal Products for Human Use (CHMP) to review novel drugs from the standard 210-day period to 150 days.

To be granted accelerated approval by the EMA, applicants must justify that their novel product offers a significant therapeutic, scientific, or technical innovation for treating, preventing, or diagnosing a disease.  

Although these designations reduce the time needed for potentially life-saving therapeutics to reach patients, there are various concerns surrounding the expedited drug approval process. For example, the FDA expedited approval processes are largely based on surrogate endpoints, which include biomarkers that reflect health status.

However, these endpoints may not strongly predict the true efficacy of a novel drug nor their ability to confer survival in patients. In fact, one study found that many cancer drugs that have been approved based on surrogate endpoints did not lead to any significant survival benefits.

An additional limitation associated with accelerated approval processes is the lack of safety data, which may require these new drugs to be more closely monitored.

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​​​​​​​Patient safety and advocacy

Patient-focused drug development (PFDD), which is part of CDER, is a crucial approach that emphasizes the important role of the patient during the development and evaluation of novel drugs. PFDD often relies on patient and caregiver input through appropriate methods to inform drug development and regulatory decision-making.

Furthermore, PFDD is also responsible for identifying information that will allow physicians to effectively communicate the benefits and risks associated with new drugs to patients and support their ability to make well-informed decisions on their treatment.

Within the European Union, patients may represent their community through management boards or scientific committees, as well as various legitimate organizations, including the Patients’ and Consumers’ Working Party (PCWP), to get involved in regulating and approving new drugs.

Patients involvement at the EMA can also be in the form of a scientific expert to provide advice during the completion of protocols, review documents, and consultation with relevant scientific committees.

Global perspectives on drug approval

In 2015, the Chinese government implemented various regulatory reforms to improve the transparency and efficiency of their drug review process and accelerate the approval of new drugs for human use.

One recent study highlighted that it took the Chinese government about one year or less from the new drug approval (NDA) submission date to reach approval, which significantly differs from U.S. processes that typically take about one year to complete the NDA review.  

In 2018, China’s regulatory body, the National Medical Products Administration (NMPA), issued ‘Guidelines for Acceptance of Overseas Clinical Trial Data,’ which accelerated the approval of many drugs in China, particularly those with oncology indications.

The NMPA also recently revised ‘Administrative Measures for Drug Registration’ in 2020 to optimize the review and approval process for new drugs in the hopes that pharmaceutical companies will continue to develop innovative therapeutics.


  1. “Development & Approval Process – Drugs.” (Online). Available from:
  2. Joppi, R., BErtele, V., Vannini, T., et al. (2020). Food and Drug Administration vs. European Medicines Agency: Review times and clinical evidence on novel drugs at the time of approval. British Journal of Clinical Pharmacology 86(1); 170-174. doi:10.1111/bcp.14130
  3. “The FDA’s Drug Review Process: Ensuring Drugs Are Safe and Effective” (Online). Available from:
  4. “How EMA evaluates medicines for human use.” (Online). Available from:
  5. “Fast Track.” (Online). Available from:  
  6. “Accelerated assessment.” (Online). Available from:
  7. Frakt, A. B. (2018). The Risks and Benefits of Expedited Drug Reviews. JAMA 320(3); 225-226. doi:10.1001/jama.2018.8262.
  8. “Modernizing FDA’s New Drugs Regulatory Program” (Online). Available from:
  9. “EMA Overhauls the Medicine Legislative and Regulatory Framework” (Online). Available from:
  10. “CDER Patient-Focused Drug Development” (Online). Available from:
  11. “Getting involved” (Online). Available from:
  12. Honig, P., & Zhang, L. (2019). Regulation and Innovation: Role of Regulatory Science in Facilitating Pharmaceutical Innovation. Clinical Pharmacology & Therapeutics. doi:10.1002/cpt.1367.
  13. Ge, Q., Xu, L., DiMasi, J. A., et al. (2023). Impact of regulatory system changes on the availability of innovative drugs in China. Nature Reviews Drug Discovery. doi:10.1038/d41573-023-00058-0.

Further reading 

Last Updated: May 8, 2024

Benedette Cuffari

Written by

Benedette Cuffari

After completing her Bachelor of Science in Toxicology with two minors in Spanish and Chemistry in 2016, Benedette continued her studies to complete her Master of Science in Toxicology in May of 2018. During graduate school, Benedette investigated the dermatotoxicity of mechlorethamine and bendamustine; two nitrogen mustard alkylating agents that are used in anticancer therapy.


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