Skip to main content

Changing Rules of Drug Development Should Benefit Patients

By March 23, 2011December 18th, 2023Awareness, Science & Research

The panel discussions at this week’s annual meeting of the Massachusetts Biotechnology Council that I attended on behalf of the Children’s Tumor Foundation included leadership from many drug giants: Millenium, Sanofi-Aventis, Novartis and AstraZeneca to name a few. From all of them the message was the same: the rules of drug development are changing – and in a big way.  We are watching these issues as they will be of growing importance in neurofibromatosis as we see more drugs getting into clinical trials.

First and foremost, we are seeing the emergence of personalized medicine. This is based in understanding an individual’s genetic makeup, and then identifying why a person develops cancer or heart disease or any other malady. This information helps doctors to determine which patients are likely to benefit from specific molecularly-targeted medicines. Though this seemed like science fiction just a few years ago, the first results of personalized medicine are already emerging specifically from cancer trials. The result we hope will be faster  and more meaningful clinical trials and a faster path of getting drugs to the market.

In response to this, drug companies are realizing that if they are developing a new drug they need to be thinking about developing a ‘companion diagnostic’ – e.g. a blood test so they can identify, prior to prescribing the drug, the patients that will most benefit from the treatment. This is revolutionizing the way drug companies are planning their drug development strategies and excitingly encouraging partnering between drug and diagnostic companies early on to ensure that no promising drug is left behind due to lack of a diagnostic.

This partnership extends to considerations about how the Food and Drug Administration will co-approve drug and diagnostic (there is noise about a new office opening at FDA to aid with this). And finally … will insurance pay for this?  Drug companies are realizing that once they have a promising new drug, conversations with those overseeing reimbursement need to be a priority.

Endnote: our poster ‘Neurofibromatosis Networking’ presented Monday night garnered a significant amount of attention from attendees and made some helpful new connections for NF in the drug world. Attendees were give three ‘$1 million dollar’ bills to ‘invest’ in the posters and though we did not win, our envelope was quite full!