11 ways clinical trial protocol design can improve speed and costs

Fewer than 1 in 10 new drugs reach the market and around 85% of potential drug therapies do not pass early clinical trials. Additionally, the cost of researching, developing and regulating a drug can be as high as $2.6 billion. It has been argued that drug developers are often losing promising products as a result of using development strategies based on outdated clinical trial designs. This assumption implies that alternative ways of developing new drug products must be implemented starting with the earliest phases of clinical trial.

Developing and implementing new drug development models requires the participation of companies, regulators, academic institutions, governmental agencies, and patient advocates. Here we look at 11 different opportunities to improve clinical trials [1] in terms of speed and costs by taking into consideration both protocol designs and contextual variables such as collaborations.

This is an extract from the whitepaper 'Innovative protocol designs in early phase clinical development' - download the full paper here.

1. Find new targets: The Human Genome Project has provided thousands of drug targets that can be considered. A precompetitive effort to assess which targets are the most likely to provide positive results could increase the success rate of drug development.

2. Use predictive toxicology and efficacy: Approaches such as pathway-based systems biology and organ-on-a-chip could deliver more efficient and accurate predictions of safety and efficacy.

3. Take advantage of existing drugs: Products that have already been approved for another disease can sometimes be used to target a different condition. The discovery of diseases that were thought to be independent but are mechanistically related provides the opportunity to treat several diseases with a product that has already been approved or is in development.

4. Use combination therapies: The development of methods to identify combinations of drug candidates with increased efficacy and reduced safety risks could increase the effectiveness of individual therapies developed or in development. This can work with dedicated technology development, testing, and clinical-development frameworks.

5. Use gene and cell-based therapies: The emergence of powerful new gene-editing techniques such as CRISPR-Cas9 and the increasing flexibility of stem-cell technologies offer many opportunities to provide transformational therapies complementary to small molecule and protein drugs.

6. Value precompetitive collaboration: More organized precompetitive collaborations involving companies, governments, and academic institutions can improve drug discovery, particularly in areas where substantial scientific background is missing. Examples of such developments include Accelerating Medicine Partnership and Alzheimer’s Disease Neuroimaging Initiative.

7. Develop qualified biomarkers: There is a limited number of qualified biomarkers or combinations of them that can fasten the drug-development and regulatory process. As such, there is a clear need for a biomarker-qualification process. Achieving this requires a good understanding of the context of use and a consideration of the benefits and risks of the market, as well as an understanding of the type of evidence standards required to approve the biomarker for use in preclinical and clinical testing.

8. Make use of real-world evidence (RWE): It is now possible to obtain data concerning the clinical efficacy and safety of drugs from real-world settings by establishing an appropriate size of cohort and number of observations that would reduce observational bias. RWE could be initially used for supplemental applications of approved medicine in order to reduce safety considerations. Later, this approach could also complement randomized controlled trials (RCTs).

9. Target precision medicine: Individualized treatments are likely to become feasible at a large scale in the near-future. Identifying groups that might benefit from a drug before clinical testing could make clinical trials smaller and shorter, leading to improved efficiency and reduce exposure of subjects who are unlikely to benefit from a specific intervention.

10. Aim for decentralized clinical trials: Bringing clinical trials in patient communities can reduce infrastructure costs and increase the participation of patients or providers who could not be reached otherwise.

11. Work with health care providers: Working with providers and information technologists can allow sponsors of clinical research to serve as catalysts for creating a learning healthcare system where health delivery is integrated with knowledge generation. For example, this could be achieved by integrating clinical trials with information technology systems such as electronic health records (EMRs).

Download the full 'Innovative protocol designs in early phase clinical development' whitepaper here.

References

1. ROSNEBLATT, M., AUSTIN, C. & BOUTIN, M. 2016. Innovation in development, regulatory review, and use of clinical advances. Perspectives.