The business case for more robust pre-clinical research

16. july 2017 at 11:53 | Veronika Valdova, ARETE-ZOE
In the high consequence environment of pharmaceutical development, any assumption made earlier in the process can prove extremely costly if uncorrected once more information becomes available.

From a business perspective, it is essential to create a safe avenue for communication of concerns regarding the drug candidate's efficacy, safety, toxicity or pharmacological function immediately as the researchers become aware of them.

Compliance, IND submissions, and business incentives
The Food and Drug Administration (FDA) requires researchers to provide specific data about new drug candidates before the drug can to proceed to human clinical trials. The IND submission package includes preclinical data on animal pharmacology and toxicology as a proof that the drug candidate is reasonably safe for human trials; manufacturing information to ensure consistent quality of the product; and clinical protocols and investigator information. The application also includes information on clinical professionals and a commitment to obtain informed consent and a review by the institutional review board (Food and Drug Administration, 2017).

The pharmacology/toxicology package contains pharmacology studies, acute, subchronic and chronic toxicity studies, special toxicity, carcinogenicity, reproduction toxicity, mutagenicity and absorption, distribution, metabolism and excretion (ADME) studies (Food and Drug Administration, 2017). This information generally comes from in vitro tests, computer modeling, and from experiments on laboratory animals. All experiments on animals in the U.S. have to conform to Part 58 of Title 21 of the Code of Federal Regulations Good Clinical Practice for Nonclinical Laboratory Studies (eCFR - Code of Federal Regulations, 2017).

From a business risk perspective, compliance and due diligence are the easy parts. The hard part is the predictive power of this information whether or not the drug candidate has the potential to show safety and efficacy in clinical trials and obtain approval. There is a universal pressure to conduct these tests in the timeliest manner practicable, in order to pass the IND submission landmark. This step typically indicates the company's willingness to undertake a huge investment in clinical trials, without any guarantees of success. The impact of IND submission on stock price is typically neutral (Picardo, 2017). The dropout rate is especially high for new molecular entities with the potential of becoming first-in-class. The dropout rate in phase III is especially worrying.


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