Set the Course from Idea to Clinical Development for Innovative Therapies: Key Steps
When developing new medicinal products, the journey from the first spark of an innovative idea to the start of the first stages of clinical development is a complex and demanding process, especially for highly specialized, cutting-edge biopharmaceutical products. Each of the following steps are critical in eventually gaining regulatory approval to start the first-in-human (FIH) study.
1. Initial Funding
Securing initial funding is the cornerstone of any successful drug development project. This financial backing supports the early stages of research and the procurement of essential resources. Additional funding must be ensured throughout the entire development process.
2. Lead Candidate Selection
Selecting a lead candidate is a pivotal decision in the drug development process. This involves identifying the most promising compound that demonstrates the desired biological activity and has favorable safety and efficacy profiles based on preliminary research and nonclinical data.
3. Proof of Concept (PoC)
The nonclinical proof of concept, supported by both in vitro and in vivo studies, is essential for demonstrating the potential efficacy of the lead candidate. This step establishes a scientific basis for further development, supporting subsequent phases and possibly attracting investors.
4. Contract Development and Manufacturing Organizations (CDMOs)
Finding reliable and cost-effective CDMOs is crucial for the development and manufacture of clinical trial materials. These organizations should provide in-depth technical expertise and the necessary infrastructure for process development and GMP-compliant manufacturing.
5. Manufacturing Process Development
Developing a scalable and reproducible manufacturing process for the drug substance is crucial. This step ensures that the drug substance can be manufactured consistently and in sufficient quantities for the first-in-human clinical trial. The manufacturing process also should be scalable for late-stage clinical development and eventual commercialization. In case of future manufacturing process changes, comparability between the materials before and after the change must be demonstrated.
6. Drug Product Formulation
The formulation of the drug product entails creating a stable and effective form of the drug that can be administered to patients. This includes considerations of dosage, route of administration, delivery method, and stability. Extensive formulation development studies can only be initiated once sufficient amounts of the drug substance are available. The intellectual property situation for formulation candidates should be carefully assessed to avoid patent infringements.
7. Analytical Methods
Developing robust analytical methods is necessary to ensure the quality and consistency of the investigational medicinal product. These methods are used to test the drug substance and the drug product at various stages of development and manufacturing, typically for release and stability testing as well as for in-process testing during manufacture.
8. Good Laboratory Practice (GLP) Toxicology Studies
GLP toxicology studies are performed to assess the safety profile of the lead candidate. These studies are meticulously designed to comply with regulatory standards and provide critical data on the potential adverse effects of the compound in animals. The GLP toxicology studies should be conducted with study material representative of the material to be used in the first-in-human study.
9. Clinical Study Design
The clinical study of a first-in-human study must be designed to primarily determine the safety and tolerability of the investigational medicinal product in humans in compliance with GCP. If the IMP is tested in patients, preliminary efficacy can also be assessed. The clinical study design should be meticulously planned by the sponsor and be aligned with regulatory authorities during a scientific advice meeting.
10. Clinical CRO and Study Sites
Finding a suitable clinical contract research organization (CRO) and selecting high-recruiting study sites are critical steps in conducting a clinical trial. The CRO provides essential services for successful study conduct, including study management and clinical monitoring.
11. Manufacture of Investigational Medicinal Product (IMP)
Once process development and analytical method development have been completed, the IMP can be manufactured under GMP. The IMP must be released using data from quality-control tests and must be subjected to stability testing.
12. Clinical Trial Application
Before initiating a clinical trial, a Clinical Trial Application (CTA) must be submitted to the concerned regulatory authorities and ethics committees for their approval. The CTA includes detailed information about the drug, the study design, and the manufacturing processes, among other things, and the requirements are defined by ICH and regulatory guidelines.
13. First-in-Human Study
The initiation of the first-in-human study marks the beginning of the clinical phase of the drug development process.
Additional Considerations
For highly innovative biopharmaceutical development programs, additional development efforts and iterations may be necessary throughout the development process. The planning should take into account potential failures, setbacks, and major risks and should allow for sufficient resources and time for improvement and optimization of the drug candidate, addressing any challenges or issues that arise.
By following these structured steps, developers can navigate the complex path from an initial idea to the start of clinical trials, laying the groundwork for potential breakthroughs.
MF BIOTECH provides comprehensive project solutions throughout the entire product development cycle. With our extensive development experience in biopharmaceuticals, we will help you to establish your development strategy, avoid pitfalls, and reach your company goals. Contact us for more information.