By Karoline Bechtold-Peters
The roles were hitherto clear: the pharmaceutical industry wants to market its new and innovative research products as quickly as possible with the highest possible profit potential after the shortest possible review time, and the approving authority sets the bar of quality, efficacy, and safety profile requirements as high as possible, leading to a dramatic increase of R&D costs in the past 20 years. But this situation has also led to a significant decrease (PDF) in the efficiency of the R&D pipeline as well as a growth in the size of clinical trials. Some drugs, in spite of good efficacy, have been dropped due to lack of marketing potential (“blockbuster hype”) and therefore have unfavorable net present value, or at best the drug would be licensed out. Innovative but expensive therapies and medications have also failed due to the third player in the game: the health insurance companies who consider their existing therapies as sufficient (e.g., fast-acting insulin analogues in type 2 diabetics).
Both the U.S. and the European Union (EU) regulatory bodies became aware of this dilemma and began to try to counteract with new guidelines and initiatives, including:
- introduction of the Office of Regulatory Science and Innovation at the Food and Drug Administration,
- a road map to 2015 (PDF) by the European Medicines Agency, which states, “There is thus a need for debate on how best to support and translate the new science into regulatory requirements,”
- acceptance of risk-based approaches,
- implementation of a break-through therapy designation,
- the possibility of accelerated assessments (PDF) in case of major interest for public health, and
- establishment of guidelines on advanced therapy medicinal products to increase regulatory clarity inclusive of installing the Committee for Advanced Therapies to consult companies with R&D in this area.
On the other hand, incidents such as with TGN1412 when healthy volunteers experienced a cytokine storm, led to the introduction of MABEL studies before starting phase 1 clinical trials for certain medications and sequential dose escalation. Finally, rather rare side effects and the complete safety profile of an innovative drug or therapy are often only fully seen after launch, and this must also be accepted by patients. Here in Europe, the critical-only reporting about the profit-oriented pharmaceutical industry and the lack of supervision of the regulatory authorities, or the disproportionate compensation in the U.S. in case of health damages after use of drugs due to unwanted side effects, is not necessarily helpful. The alternative would be the refusal by the authorities to accept any innovative medicine if a zero-risk strategy is applied.
The regulatory agencies are therefore always in the dilemma of not letting innovative therapies reach the patients quickly enough, who are then not adequately treated, versus being corrective to ensure that only products with reasonable quality and sufficient risk-benefit enter the market and therefore get to ill or future ill patients.
If you want to hear more about this subject, please visit the Regulatory themed roundtable session Common Pitfalls in Recent Applications of Biologic Drugs, Including Biosimilars and Cell-Based Products—The Dilemma between Innovation and Protection from Innovation. Regulators from the U.S. and the EU as well as industry representatives will speak and discuss as panelists.