Biggest FDA Developments for 2017

As we begin the new year, it is time toassess the previous year and consider what really mattered when all is said and done. We’ve perhaps already done this on a personal level, but here is my take on an FDA level.

One thing is certain, it has been a busy year for FDA and on many levels, it was a game-changing time. There were many more approvals of new molecular entities during 2017 (46) than there were last year and the total number just exceeded that of 2015 which was a huge year for approvals (45). And it is not just the number of approvals, but there have been a number of “firsts” this year, including the first prescription app for use in treating opioid addiction.

The fact that more is happening has been reflected perhaps by the issuance of many more agency press releases this year by far over last year – 164 for 2017 compared to 122 for 2016. Many of the releases this year were special statements by the Commissioner to provide additional commentary on developments at the agency.

And so among all that activity, here comes my pick for the top five developments at/by FDA for this past year.

1. Approvals in Cancer Treatments – Reviewing the list of approvals of new molecular entities for 2015, FDA has approved 12 new cancer-related treatments which is 8 more than were announced by FDA in 2016 and nearly as many as were approved in the banner year of 2015. It is not just the number of cancer treatment approvals, but the scope as well. There were a large number of approvals aimed at types of blood based cancers – AML/leukemia as well as breast cancer and the approval of the first biosimilar for use in breast and stomach cancer. In addition, the agency granted accelerated approval for a treatment indicated for any solid tumor that has a specific genetic feature – the first time an approval was based not on the location of the tumor, but rather on a common biomarker.

2. CAR-T Approval – Related to the above, not a drug treatment but a cell-based gene therapy, the approval of the first CAR-T Therapy in August of this year merits its own category given the degree to which it evolves cancer treatment. A long-sought goal of cancer therapy has been to unleash the ability of the immune system itself to resist the cancer. CAR-T manages just that. While the earliest work has been successful in blood borne cancers, there is reason to hope that there might one day be used in solid tumors. Each dose is customized and is created using the patient’s own T-Cells, genetically modified to include a new gene that…