Clinical trials, market authorization and product launch
To support you in the planning of a pharmaceutical project, we here describe the different steps in the drug development process.
In order to carry out clinical trials in Sweden, it is necessary to obtain permission from both an ethical committee and the Swedish Medical Products Agency, or other equivalent European regulatory body. Clinical studies are generally carried out in three phases.
- Phase I has focus on tolerability and safety in healthy volunteers.
- Phase II involves demonstration of efficacy in a limited patient population
- Phase III is performed in a larger group of patients, often on a multi-centered basis.
In order to register the new therapy, it is vital that all studies are planned and performed so that they fulfill all the documentation requirements for regulatory submission and acceptance. This documentation should also include data supporting relevant health economics calculations associated with the treatment.
Market authorization and launch
The process of development of a novel therapy is not fully complete with the granting of market authorization. There are many more steps to take to gain market access, which, among other things, involves the eventual subsidisation of the treatment based on an appropriate Health Technology Assessment (HTA). It is even necessary sometimes to seek assurance that the targeted health system is actually amenable to use and subsequent vigilance of the therapy. Indeed, for certain products, such as cell-, gene- and immunotherapies, special demands for post-marketing vigilance can exist.
The final step involves following up treatment in register studies or in register-based, randomised clinical studies, in order to collect real-world data (RWD). Experience from such studies can be both useful to optimizing treatment regimes, and in terms of providing a strategic planning document moving forward.
Target product profile (TPP)
The above general description of the treatment development generally focuses on the preclinical phase, GMP manufacture, and clinical studies and therapeutic effects. There are of course many other factors to consider early on in order to ensure the development of an economically viable product that reaches the marketplace and has benefit to patients. One useful tool which can assist in planning in all of these factors in the overall scheme of development is the target product profile (TPP) document. This is not required regulatory documentation, but it is very useful and important in efforts to strategically plan the overall development process for a particular new therapy.