What you need to know about regulatory submissions for a successful CTA or MAA

Regulatory submissions are essential for obtaining the necessary approvals to conduct clinical trials, bring a drug to market or make changes to a previously approved product.

These submissions ensure your pharmaceutical product, whether you’re in the early stages of clinical development or close to market approval, meets the required safety, efficacy and quality standards set by regulatory authorities. Amongst the most important types of regulatory submissions are clinical trial applications (CTA) and marketing authorisation applications (MAA), which play pivotal roles in the journey from research to commercialisation.

Regulatory submissions can occur at various stages of the drug development process and are subject to the specific requirements of the regulatory bodies in each region, such as the European Medicines Agency (EMA) and the US Food and Drug Administration (FDA). The goal of each regulatory submission is to demonstrate that your drug is safe, effective and compliant with regulatory standards.

In this blog, we’ll explore the different types of regulatory submissions — particularly CTAs and MAAs — and what you need to know for a successful submission. 

What are the key types of regulatory submissions?

There are several types of regulatory submissions that you’ll need to navigate throughout the life cycle of a drug. The two most significant types are clinical trial applications and marketing authorisation applications.

Clinical trial application (CTA)

A clinical trial application is one of the first regulatory submissions you’ll make during the drug development process. The CTA is submitted to the relevant regulatory authority before initiating a clinical trial. Its purpose is to gain approval to conduct human trials for a new investigational drug or device.

In the EU, CTAs are submitted to national regulatory authorities or the EMA if the trial is to be conducted in multiple EU member states. A CTA typically includes:

• Pre-clinical data: evidence from laboratory and animal studies showing the safety and potential efficacy of the drug.
• Clinical trial protocol: detailed plans for the study design, objectives, methods and data analysis, along with how patient safety will be ensured.
• Informed consent forms: documents explaining to participants the nature of the trial and their rights.
• Investigational medicinal product (IMP) information: details about the drug being tested, including manufacturing and quality control measures.
• Ethics committee approval: proof of approval from an ethics committee or institutional review board (IRB).

The clinical trial application must be carefully prepared to meet regulatory requirements, as approval is needed before any human trials can begin. If the CTA isn’t approved, the clinical trial cannot proceed.

Marketing authorisation application (MAA)

Once clinical trials have demonstrated that a drug is safe and effective, the next step is submitting your marketing authorisation application to the relevant regulatory authority to gain approval for marketing the drug. In the EU, the MAA is also submitted to the EMA (or to national regulatory authorities if the drug will be marketed only in a specific country).

An MAA regulatory submission includes a comprehensive package of data collected during the drug development process, including:

• Clinical trial results: data showing the safety, efficacy and overall benefit-risk profile of the drug in human trials.
• Pharmaceutical data: information on the drug’s formulation, production methods and quality control processes.
• Labelling and packaging: proposed labelling information, including dosage instructions, indications, side effects and other relevant details.
• Pharmacovigilance plans: strategies for monitoring the drug’s safety once it’s on the market, including post-market surveillance and adverse event reporting.
• Risk management plans (RMP): plans to mitigate any identified risks associated with the drug.

The marketing authorisation application is a critical step toward commercialisation, as approval will allow you to market and sell the drug within the EU. However, approval can be complex, and regulatory authorities may request additional information or clarification before granting authorisation.

Other key regulatory submissions: IND, NDA and BLA

While the CTA and MAA are the primary regulatory submission types in the EU, other submissions may be necessary depending on the jurisdiction:

• Investigational new drug (IND) application. In the US, you must submit an IND to the FDA before conducting clinical trials. The IND includes data similar to that of a CTA but is tailored to meet the FDA’s requirements.
• New drug application (NDA). The NDA is the US equivalent of the MAA and is submitted to the FDA once clinical trials are complete. The NDA contains data on the safety, efficacy and quality of the drug. It’s necessary for market approval.
• Biologics license application (BLA). This is similar to an NDA but is used specifically for biological products, such as vaccines or gene therapies.

What are the steps for a successful clinical trial application? 

To achieve a successful clinical trial application, you need to follow these key steps:

1. Prepare pre-clinical data. Before submitting a CTA, you must gather extensive pre-clinical data from laboratory and animal studies. This data should demonstrate the drug’s safety profile, including its potential for toxicity or side effects, as well as its mechanism of action.
2. Develop a robust clinical trial The clinical trial protocol is the backbone of your CTA regulatory submission. The protocol must include detailed plans for study design, inclusion and exclusion criteria for participants, endpoints (primary and secondary) and methods for data collection. The protocol must also align with international guidelines, such as good clinical practice (GCP), and demonstrate a clear pathway for patient safety.
3. Submit ethics approval. Ethical approval is required before any clinical trial can proceed. An independent ethics committee or institutional review board must review your trial’s design, the informed consent process and the safety precautions taken for participants.
4. Submit the CTA to regulatory authorities. The CTA, complete with all necessary documentation, is then submitted to the regulatory authorities. In the EU, this can be done to either national authorities or the EMA, depending on the scope of the clinical trial.
5. Address feedback and obtain approval. Regulatory authorities will review your clinical trial application and may request additional data or modifications to the study protocol. Addressing their feedback promptly and thoroughly is key to obtaining approval and moving forward with the clinical trial.

What are the steps for a successful marketing authorisation application?

For a successful marketing authorisation application, you need to follow these essential steps:

1. Ensure robust clinical trial Before submitting an MAA, you must have completed the required trials demonstrating the safety and efficacy of the drug. The results must be statistically significant and show a favourable benefit-risk profile.
2. Compile comprehensive manufacturing and quality data. The MAA must include information about the drug’s formulation, manufacturing processes and quality control measures. Regulatory authorities will assess whether the product is consistently manufactured to meet quality standards.
3. Prepare risk management and pharmacovigilance plans. The MAA regulatory submission must include detailed plans for monitoring the drug’s safety once it’s on the market. These plans should outline how you’ll report adverse events and manage ongoing safety.
4. Submit the MAA and address regulatory feedback. Once you’ve submitted the marketing authorisation application, regulatory authorities will review it and may request additional data or clarification. Responding to these requests in a timely and thorough manner is critical to obtaining approval.
5. Market authorisation and post-approval surveillance. Upon receiving approval, you can market the drug within the EU. However, post-approval surveillance will continue to monitor the safety and efficacy of the drug in the broader patient population.

Accelerate your progress through the regulatory pathway with TMC

Regulatory submissions are a vital part of the drug development process, ensuring new medicines are safe, effective and compliant with regulatory standards. Clinical trial applications and marketing authorisation applications are two of the most significant submission types you need to be aware of when bringing your drug to market.

For a successful CTA and MAA submission, you must gather high-quality data, prepare comprehensive documentation and work closely with regulatory authorities to address any feedback or requests for additional information.

By understanding the regulatory submission process and preparing thoroughly, you can significantly improve your chances of success in bringing a new drug to market.

TMC can help you bring treatments for rare diseases to patients swiftly through our flexible, integrated and tailored regulatory services. At TMC, we’ll guide you, as your partner, throughout the drug development process — using our expertise in regulatory processes for rare diseases to help you design effective clinical trials and secure early-stage approvals.

We can also act as your marketing authorisation holder (MAH), if you don’t have a presence in Europe, to ensure compliance with all local regulations. As an established EU/EEA-based marketing authorisation holder with SME status, TMC can be your EU/EEA representative — simplifying regulatory submissions and helping you secure substantial discounts on EMA fees.

Find out more about TMC’s regulatory services or contact our team today at regulatory.services@tmcpharma.com to see how we can accelerate your progress through the regulatory pathway and help you secure market authorisation approval — efficiently and cost-effectively.