Literature-Based Submissions: Using Publicly Available Data to Support Drug Development
16 Jan 2026
Before a new pharmaceutical product can be brought to market, comprehensive drug approval application packages—such as New Drug Applications (in the United States), New Drug Submissions (in Canada), or Marketing Authorization Applications (in multiple countries)—must be prepared for evaluation by a regulatory body.
These packages comprise extensive documentation on the efficacy, safety, and quality of the new drug—typically a unique, novel chemical entity—based on proprietary data from numerous clinical, safety, and toxicology studies conducted with the proposed new drug. The preparation of these packages can be both time and resource intensive. Fortunately, drug development companies have an attractive option available when developing a new drug that contains an active ingredient that has been extensively studied: companies can prepare what are known as literature-based submissions, repurposing publicly available data for incorporation into the drug approval application so long as there is no patent infringement.
What is a Literature-Based Submission?
Literature-based submissions allow data to be leveraged from an approved drug with a different indication than the new drug, from a drug that may have a substantial body of publicly available literature available, or from an existing drug that will be developed as a new formulation. These types of submissions rely on published scientific and medical literature to provide efficacy, safety, pharmacokinetics, and metabolism data for a drug without the need for a sponsor (i.e., drug development company) to conduct its own studies to support a marketing application. Simply put, published peer-reviewed literature can fulfil some or all of the typical efficacy and safety endpoints required in a drug approval application. Where gaps exist in a sponsor’s own dataset (e.g., if nonclinical reproductive or developmental toxicology studies are missing), publications with data from thorough, well-conducted studies with appropriate endpoints can be used to bridge the gap. Furthermore, in situations where a sponsor has conducted a small clinical trial for its drug approval application but needs to address additional endpoints typically covered by larger studies, published literature can help fill the gap.
The applicability of the literature-based approach will depend on various factors including the drug type, the quality of the identified publications, the relevance of the publications to the drug, and even the targeted jurisdiction.
Regulatory Health Agencies That Accept Literature-Based Submissions
Various agencies, including the United States Food and Drug Administration (U.S. FDA), Health Canada, the European Medicines Agency, and Australia’s Therapeutic Goods Agency (TGA), accept literature-based submissions in support of new drug approvals. Brazil’s Agência Nacional de Vigilância Sanitária (ANVISA) also has an abbreviated development pathway that allows for literature to be used in certain scenarios. Some agencies offer guidance on the preparation of literature-based submissions: Health Canada offers guidance in Drug Submissions Relying on Third-Party Data (Literature and Market Experience) (Health Canada, 2015), while the TGA has a Pre-submission guide for literature based submissions (LBS) and provides “how‑to” guidance (TGA, 2014, 2021, 2025). While not all regulatory agencies have formal guidance documents for literature-based submissions, many allow for the use of existing high-quality peer-reviewed published data to support part, or most, of a drug approval application. Regulatory authorities also offer pre‑submission consultations that can help companies determine whether the literature-based approach is appropriate for their new drug product.
As a real-world example, the U.S. FDA accepted a literature-based submission for Iloprost (Aurlumyn™), approving the drug as a treatment for severe frostbite in February 2024 (U.S. FDA, 2024). Efficacy data for the approval was based on a published clinical study from a thesis, along with supportive clinical evidence of efficacy from other existing literature. Additional endpoints were addressed by reference to the FDA’s findings of nonclinical and systemic safety for Ventavis™, an approved drug, and safety data from studies conducted by the sponsor for a different indication.
Key Considerations for Literature-Based Submissions
When it comes to literature-based submissions for drug approvals, a key consideration—and one that is common among all regulatory health agencies—is the need for comprehensive, robust evidence to support the new drug’s safety and efficacy. It is important for drug development companies to show that the publications referenced are relevant and clearly apply to the new drug and its proposed indication(s). Drug development companies need to critically review the information in the publications for its applicability, including the purity of the product tested, the dosing regimen, and the patient population.
Another key consideration pertaining to literature-based submissions is the approach used to prepare the submission, acknowledging that the approach may differ slightly between regulatory agencies. A sponsor will need to adopt an appropriate search strategy to obtain relevant publications and will need to document it. For example, a systematic review must be used for literature-based submissions submitted to Brazil’s ANVISA, while other authorities may give more flexibility.
It is also important to note that the type of data that can be provided in a literature-based submission will depend on what is publicly available. If there is a lot of published information, companies may try to only focus on select studies (e.g., randomized, placebo-controlled studies); however, if the data available are scarce, lower quality studies (e.g., open-label studies or case series) may also be considered.
Literature Review Methods
As mentioned, some regulatory agencies stipulate the methodology to be applied in the literature review process. Thus, the guidelines of the applicable regulatory agency must be thoroughly reviewed before proceeding with a literature search to assess the availability and quality of the information for use. The three common literature review methods are outlined below. Regardless of the approach, companies will need to ensure that reputable and appropriate literature databases are searched.
- Systematic review: A systematic review is structured, is usually designed to answer a specific question, and follows a reproducible process to identify, select, and evaluate studies. The screening and selection of publications follows pre-defined criteria and is often done by two individuals in parallel. A systematic review also includes a formal assessment of publication quality that focuses on the question.
- Meta-analysis: A meta-analysis pools the results from the publications identified in the systematic review using statistical methods.
- Narrative review: A narrative review involves providing summaries of publications. The quality of publications identified will also need to be discussed, but the evaluation may be more subjective. It is a more flexible approach.
Once the literature is collated, an assessment should be conducted to determine the likelihood of building a persuasive drug approval application using the literature on its own or combined with proprietary studies. Once publications of interest have been identified, it may also be helpful to consider a pre-submission consultation with the regulatory authority of interest. Additionally, a consultation with legal counsel is highly recommended to ensure that patents or intellectual property rights are not infringed upon.
Challenges and Benefits of Literature-Based Submissions
One of the challenges with literature-based submissions is making sure, once the high-quality studies are located in the literature, that those studies are clearly relevant to the new product being developed; it is important to be able to justify why they support the new application. Another challenge is the size of the search, as literature searches can become quite large. Well-defined inclusion and exclusion criteria are necessary from the start to keep the process manageable.
Despite the challenges, there are benefits to literature-based submissions. This approach can significantly reduce drug development time since new studies do not have to be conducted, making the whole approval process move faster. Using Iloprost (Aurlumyn™) as an example again, the critical published study that supported its approval recruited patients over a 12-year period, since severe frostbite is so rare. While this may be an extreme case in terms of study length, and most clinical studies are shorter, it illustrates how this approach could save time: the drug development company did not have to conduct a study over several years but instead leveraged existing data. Furthermore, if a company can make a case for its drug using published studies, the overall drug development program becomes less costly, as clinical trials with the proposed new drug can be avoided.
How Intertek Can Help
Intertek has the expertise to help drug developers with literature-based submissions through the following service offerings:
- Feasibility Assessments and Gap Analyses: Intertek can conduct literature searches and evaluate the available information at a high level to identify key gaps in information to support the efficacy and safety of a new drug.
- Authoring Support: Intertek can review publications, assess publication quality, and write the application sections in Common Technical Document format according to the regulatory health agency’s expectations for safety and efficacy data. Working with our clients, we provide high-quality, organized summaries of information to meet regulatory expectations.
- Regulatory Support: Intertek can prepare and submit meeting requests to regulatory agencies and prepare meeting information briefing packages for sponsors wanting to discuss their drug development strategy with regulatory agencies prior to the formal application.
Why Choose Intertek?
Intertek’s scientists have extensive experience evaluating original data and published information and preparing regulatory applications for a variety of therapeutic modalities and disease indications. We are proud of our collaborative approach that supports our clients’ needs.
References
Health Canada. Drug Submissions Relying on Third-Party Data (Literature and Market Experience). Health Canada; 2015. Available at: https://www.canada.ca/content/dam/hc-sc/migration/hc-sc/dhp-mps/alt_formats/pdf/prodpharma/applic-demande/guide-ld/srtd_pfdt_gd_ld-eng.pdf.
TGA. Systematic Literature Searches for Literature Based Submissions. Therapeutic Goods Administration; 2014. Available at: https://www.tga.gov.au/resources/resources/user-guide/systematic-literature-searches-literature-based-submissions.
TGA. Pre-submission Guidance for Literature Based Submissions (LBS). Therapeutic Goods Administration; 2021. Available at: https://www.tga.gov.au/resources/resources/user-guide/pre-submission-guide-literature-based-submissions-lbs.
TGA. Literature based submissions. Therapeutic Goods Administration; 2025. Available at: https://www.tga.gov.au/how-we-regulate/supply-therapeutic-good/supply-prescription-medicine/pre-submission-planning-prescription-medicines/literature-based-submissions.
U.S. FDA. Clinical reviews: Addendum to address substantial evidence of effectiveness for NDA 217933, Iloprost for the treatment of severe frostbite to reduce the risk of digit amputation. In: U.S. Food and Drug Administration; 2024. Available at: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2025/217933Orig1s000MedR.pdf.
