Explainer: Drug development, the regulatory process and the COVID-19 vaccine race

Athena Kolivos, Chief Scientific Officer CRC & Farmaforce

The vaccine discovery and development experience unfolding for COVID-19 serves as a timely reminder that researching, developing and commercialising therapies to prevent, treat or cure life-threatening diseases is an extensive process; one that is very different from bringing everyday products and commodities to the marketplace.

Across the globe, collaborations among pharmaceutical and biotechnology companies, medical research institutes, governments and academia are working to accelerate research and development efforts so an effective vaccine can be available as soon as possible. The reality is that it needs to undergo the same multiple-step evidence generation, evaluation and regulatory processes as any other therapeutic product to gain registration approval and market entry. 

New drugs and medical devices must undergo several rigorous phases and stages to bring them to market, which include:

  • Discovery and design
  • Development and manufacturing
  • Laboratory testing
  • Pre-clinical (animal) studies
  • Clinical trials
  • Regulatory technical dossier creation
  • Evaluation and registration approval.

Importantly, these phases and stages involve various validation, quality management system and process control activities. These are critical to ensuring compliance with strict regulations which govern the safe and ethical research and development, and registration of new therapeutic products that are of acceptable efficacy, safety and quality.

Typically, it is the unmet clinical needs that drive new drug or medical device development. For example, it may be a new drug therapy or device that could potentially save many lives and/or substantially improve the quality of life of people living with a serious chronic disease.

Whatever the driver, the underlying premise of new therapeutic product development is that the benefits of the new drug or device must outweigh the risks. This means the focus is on maximising the benefits and minimising the risks of a new drug therapy or device in ensuring optimal human health and safety. It starts from pre-clinical activities, i.e. before human studies, and continues through to registration and post launch ongoing product safety monitoring.  

Meeting Key Regulatory Requirements

The regulatory approval pathway to register new drugs and medical devices is mostly controlled by government regulatory authorities around the world. By way of illustration, the US Food and Drug Administration (FDA) – a leading regulatory authority with a global profile – requires these key activities be conducted sequentially to approve a new drug therapy:

Pre-clinical Testing is conducted before human trials, which involves conducting laboratory and animal studies to show the proposed new drug’s biological activity against the target disease and with particular focus on its safety and potential for toxicity.  

Investigational New Drug Application (IND) is filed with the FDA for authorisation to begin human studies, which contains various technical details about the new drug, results of animal pharmacology and toxicology studies, and other previous experiments such as the proposed clinical trial development plan.

Phase I Clinical Studies is to analyse the new drug for the first time on 20-80 humans to assess its;

  • Pharmacokinetics (i.e. what the body does to the drug – how it is absorbed, distributed, metabolised and excreted)
  • Pharmacodynamics (i.e. what the drug does to the body)
  • Safety profile (i.e. the safe dosage range)
  • Adverse effects. 

Phase II Clinical Studies is to evaluate the new drug’s efficacy (how well it works in those with the disease/condition), on hundreds of people, the minimum and maximum dosages, and to further assess its safety.

Phase III Clinical Studies is conducted, usually, on several hundreds to thousands of people with the disease/condition to further study the drug’s efficacy by comparing it with other treatments and/or placebo, as well as to monitor adverse effects and collect additional safety information.

Phase IV Studies is also known as ‘post-marketing studies’ because they are performed after registration approval, when the new drug is being used in the marketplace, to monitor its effectiveness and safety in large populations over a long period of time, to answer further clinical questions, to as well as address other issues such as years of life saved, quality of life and cost-effectiveness.   

Other Clinical Trials, namely exploratory studies called ‘Phase 0 trials’ or ‘pilot studies’, are sometimes performed before Phase I trials to test the body’s response to an experimental drug. Here, small or subtherapeutic doses of the drug are given once or for a short time to a very limited number (10-15) of people.

All pre-clinical and clinical study data, alongside manufacturing information and many other technical details, are then included in the regulatory dossier submitted for evaluation by the regulatory body in each country or jurisdiction across the globe for registration approval. 

Whether or not clinical trials are needed for a new medical device depends on its regulatory risk class based on the level of harm it may pose to users. For a device requiring clinical evidence because it is in a higher risk class, a similar clinical study pathway for drugs may be undertaken, which consists of “stages” rather than “phases” because the device needs further monitoring and examination.   

Urgent Unmet Clinical Needs

Importantly, where there is an urgency for individuals with a life-threatening or serious disease to access a new therapy that can address an unmet medical need, the FDA and other regulatory authorities have several “fast track” or expedited registration approval mechanisms, which enables it to be available in the marketplace sooner than under the standard pathway. These mechanisms are typically applied when the new therapy is the first available treatment option or has shown superior clinical outcomes over other therapies. They are designed to expedite development of the new therapy, as well as accelerate regulatory authority review and approval. These pathways have typically been applied to certain cancers, blood disorders and rare diseases, as well as other serious or life-threatening conditions where there are very limited or no viable treatment options.

Investing in the development and commercialisation of new drug therapies and medical devices is therefore quite a different proposition from investing in commodities.

Time, money and patience are key to a successful life science sector investment, which has the potential to yield massive long-term returns due to the various evidence-based activities, quality controls and other regulatory processes designed to protect human health, well-being, rights and safety.

As modern technologies advance scientific capability in life sciences, the potential for innovations to drastically improve medicines is extraordinary. Without biotechnology, the development of medicines such as vaccines to stop sickness on a global scale would not be possible. As we face an unprecedented modern era of pandemic, this link between scientific innovation and basic healthcare is becoming clearer by the day. With the right investment in life sciences, and an understanding of the quality control measures required to bring medicines safely to the people who need them, investors have the chance to significantly impact the future of healthcare.

Athena Kolivos

Chief Scientific Officer, CRC and Farmaforce

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