Progress in cell-biology, genetics, molecular and systems pharmacology has led to a paradigm shift in medicines research. Novel systems therapeutic interventions (STI’s) are personalized treatments aimed at modification of the disease process. The introduction of STI’s has major implications for the therapeutic evaluation in the context of regulatory review and approval. First, as STI’s are typically highly individualized treatments, each patient is treated differently. As a consequence the evaluation has to focus on the efficacy of a procedure rather than a specific product with a fixed composition. Second, as STI’s are designed to modify disease progression, their clinical effects are delayed and may not become manifest until several months to years after the initiation of the treatment. Traditional statistical techniques for the demonstration of efficacy are invalid in these situations. Finally, STI’s typically target multiple foci in a biological system to overcome resilience. As a result the interactions in the biological system need to be taken into account when designing STI’s.
The focus of this session is on the development of novel systems pharmacology concepts as the scientific basis for therapeutic evaluation and regulatory approval of novel systems therapeutic interventions. As these systems pharmacology concepts have a mechanistic basis, their application may be particularly useful to obtain substantial evidence of effectiveness.
The 21st century brings the introduction of therapies that can restore health or prevent prematurely fatal conditions (chronic hepatitis C infection, leukemia, lymphoma, rare retinal conditions, immunotherapy, gene and cell therapies. At the advent of this new area, the challenges are what is the potential impact of curative therapies might be, whether HTA bodies and payers are ready for them, what aspects of evaluation may need to be modified or expanded and how we might pay for them.