Article updated January 2025.
Pharma is one of the most regulated industries and employs regulatory affairs professionals to ensure the quality, safety, and efficacy of therapies patients use. Regulatory affairs departments within companies that produce medicines, medical devices, food supplements, and veterinary medicines ensure that their organizations comply with all regulations and laws concerning their business and products. These regulatory affairs professionals must stay current with complex regulations and often have expertise in legal, regulatory, and clinical trial environments.
The state of regulatory affairs in pharma has been dominated by two topics of late: drug pricing and AI.
Price pressures on drugs, both those currently on the market and those nascent in clinical trials, will not abate. This places a greater emphasis than ever on drug development and commercial teams to achieve milestones with great efficiency and develop high levels of proficiency in launch excellence. While this has been true for years, it has intensified with regulatory pressures and data that show 90% of new drugs fail to make it to market, and of those that do successfully achieve regulatory approval, commercialization and adoption, 40% fail to achieve launch goals after 24 months.
On the technology side of pharma innovation and regulatory affairs, the first drug candidate generated with the use of AI recently entered phase 2 studies. As reported by Kirkland & Ellis. “Along with that development, the FDA announced new initiatives on artificial intelligence and machine learning in drug development. As part of the announcement, the FDA issued discussion papers to communicate with stakeholders and to explore relevant considerations for the use of AI/ML in the development of drugs and biologic products.
In late May 2024, Regulatory Focus reported, “The US Food and Drug Administration (FDA) plans to release a draft guidance this year on the use of artificial intelligence/machine learning (AI/ML) to support drug development. The guidance will be informed by the agency’s experience in reviewing submissions containing AI/ML elements, as well as feedback it received on two discussion papers on the topic released last year, said Tala Fakhouri, associate director for policy analysis in the Office of Medical Policy (OMP) at FDA’s Center for Drug Evaluation and Research (CDER).”
“The AI Act, a landmark rule that aims to govern the way companies develop, use and apply AI, was given final approval by EU member states, lawmakers, and the European Commission — the executive body of the EU — in May.”
This news has strong potential implications for combination products as drug delivery systems and bioresorbable therapies continue to blur the lines between drug and device.
When it comes to AI, proceed with caution, but do indeed proceed.
While pharma companies need to proceed with caution in their implementation of AI tools, especially “generative AI,” the technology has high potential to identify new candidate compounds, improve recruiting, design and implementation of clinical trials, which is encouraging.
Caution and vigilance remain important when implementing AI in healthcare including pharma (especially including pharma regulatory affairs teams) as inherent or unintentional bias is a top of mind concern, and generative AI remains prone to uncomfortably high levels of hallucination in medical summary generation.
“The most frequent hallucinations were related to symptoms, diagnosis and medicinal instructions, highlighting the fact that medical domain knowledge remains challenging to the state-of-the-art language models.”
AI also has the potential to accelerate milestone achievement especially for medical affairs teams by streamlining moderation, engagement and reporting needs in KOL and other stakeholder asynchronous engagements.
Note: Within3 excels in this capability. Our clients regularly report 100% participation rates in advisory board programs run on our platform. Additionally, the Within3 insights management platform, including industry-leading AI-enabled insights reporting capabilities, helps teams achieve 3-7x the amount of actionable insights while eliminating up to 90% of the workload associated with analysis and reporting.
What is regulatory affairs in the pharma industry?
Pharma regulatory affairs professionals are key players in drug development, maintaining a vital communications link between the pharmaceutical company and regulatory agencies like the FDA in the United States, TGA in Australia, MHRA in the United Kingdom, and MCC in South Africa, to name a few.
More high-quality feedback vs. typical ad boards in a 100% compliant platform.
Regulatory affairs departments interact with worldwide, federal, state, and local regulatory agencies to assure that the following activities involving pharmaceutical and medical products are conducted in compliance with all applicable rules:
- Licensing
- Registration
- Development
- Manufacturing
- Marketing
- Labeling
Most countries differentiate between pharmaceutical legislation and pharmaceutical regulations as follows:
- Legislation refers specifically to the creation of laws that are usually written in fairly general terms to meet present and possible future needs. They have language that enables the government to issue regulations based on the law. Passing new laws requires a lengthy process and involves a country’s legislative body.
- Regulations are the rules established by an agency that interprets the laws to facilitate their practical implementation. They can be passed more quickly and simply than laws. For example, the US FDA has the rule-making responsibility for the “Food, Drug, and Cosmetic Act” of 1938 in the United States. Regulations have a way of expanding far beyond the size of the enabling law.
What is the role of regulatory affairs professionals?
According to the Organization for Professionals in Regulatory Affairs, regulatory affairs professionals are responsible for:
- Keeping track of the ever-changing legislation in all the regions in which a company wishes to distribute its products
- Advising on legal and scientific restraints and regulatory requirements
- Collecting, collating, and evaluating scientific data
- Presenting registration documents to regulatory agencies and carrying out any subsequent negotiations necessary to obtain or maintain marketing authorization for the pharmaceutical product concerned
- Giving strategic and technical advice at the highest level in their companies, making an important contribution both commercially and scientifically to the success of a drug development program and the company as a whole
- Helping the company avoid problems caused by badly kept records, inappropriate scientific thinking, or poor presentation of data from clinical development and clinical trials
What are the risks of regulatory non-compliance in pharma?
Regulatory non-compliance is among the most significant threats to the pharmaceutical industry and can have a profound negative impact on a pharmaceutical company’s finances, reputation, and customers. Still, major pharmaceutical firms are challenged to innovate while balancing that business goal with risk mitigation. Therefore, most of the world’s leading drug companies also face similar types of risks around regulatory compliance in the pharma industry.
Rather than being satisfied with siloed views of regulatory affairs – in which individual teams are responsible for ensuring regulatory compliance – pharmaceutical organizations should instead strive for an enterprise-wide view of what’s happening with regulatory issues. Drugmakers can also look to tech-enabled solutions to improve regulatory compliance. For instance, the use of big data in the pharma industry is increasing across the organization, from research and development to marketing. Regulatory affairs should explore whether this type of disruptive innovation in pharma can decrease the risk of pharma compliance issues.
How can insights management impact regulatory affairs?
Within3’s insights management can help pharmaceutical firms reduce overall concern about running afoul of a regulatory body. For example, engaging key opinion leaders in a virtual advisory board environment allows medical affairs teams to maintain vigilance when soliciting scientific information. Commercial teams can also rely on insights management technology to properly engage patients or other audiences.
Within3’s insights management solution provides pharmaceutical firms with a better way to preserve an enterprise-wide view of insight-gathering activity so that any potential regulatory issues can be identified and addressed proactively. To learn more about how insights management reduces business risk for pharma organizations, read our blog post.
