by admin | August 2021 | Uncategorized
There is nothing worse than handing out a diagnosis for a debilitating rare disease which has no treatment. At RxMD we find it gratifying to work on rare diseases and have worked/are working on Huntington disease, Gorlin syndrome, recessive dystrophic epidermolysis...
by admin | June 2021 | Uncategorized
The risk to the mother and the unborn child from maternal exposure during pregnancy is part of every risk management plan for a newly approved small molecule. Here we look at the causality assessment of birth defects. Birth defects are generally classified as...
by admin | June 2021 | Uncategorized
What should one look for when prioritizing cell therapy assets? The phenomenal success of CARs in hematological malignancies has been driving the push towards development of CARs against solid tumors. We are often asked to help a biotech prioritize its CAR T assets....
by admin | May 2021 | Uncategorized
In post-marketing surveillance, proportional reporting ratio (PRR) is a useful first step for signal detection. PRR is the ratio of the proportion of adverse events of interest with the drug versus other drugs. The image above depicts the derivation of the PRR....
by admin | May 2021 | Uncategorized
A few years ago we were requested by a Sponsor to undertake an analysis of outcomes of maternal exposures to a small molecule – a popular immunosuppressant. Of the different safety reports we author, these are particularly meaningful for the risk from maternal...
by admin | May 2021 | Uncategorized
An exposure response analysis is perhaps the best way to establish Proof of Concept (PoC). It is equally effective to establish causality for safety and to demonstrate proof of efficacy. It is surprising how it is seldom used to establish efficacy or causality for...
