In the Good Month category, FDA recently published “Novel New Drug 2014 Summary”, a glossy presentation of some truly great evidence of progress. Of 41 approvals considered novel new drugs (new molecular entities), nearly half were first in class, representing quite impressive validation of the attempt to find new approaches to old problems. Many of the new drugs were approved for rare orphan diseases, providing treatments for some true unmet needs but also with reason to be optimistic that some will prove over time to be more generally useful for other indications as well. Many were developed via priority/breakthrough/fast track programs that encourage pharmaceutical investment, and most were approved without regulatory delay. Also, of great benefit to industry, most of the novel new drugs (78%) were approved on the first cycle of review, with no delay to review time due to requests for additional information. FDA has previously had years it deserved to crow, but in our opinion 2014 stands out.
The new drugs approved in 2014 are defined as novel because they represent new molecular entities, but many are also novel in that their disease indications may be particularly well-served by these approvals when considering that existing therapy options may be limited. Of particular interest is Castleman’s disease, a form of lymph node hyperplasia that often acts like a lymphoma and in its multicentric form can create a serious condition of weakened immune system. Previously, treatments which included cancer therapeutics, radiation, or immune system modulators provided a therapeutic approach with well-recognized side effects and limitations; median survival is approximately 2-3 years. With the recent approval of a monoclonal antibody (Sylvant) which acts as an immunomodulator of the lymphoid hyperplasia, tumor and symptom responses are much longer than with best standard care. In contrast with the other treatment options, monoclonal antibody treatment appears to be well-tolerated, and patients rarely discontinue therapy because of drug side effects.
On the other hand, it was not a particularly great month for science and medicine when one reads the news headlines. Amongst the many difficult topics: measles is up in California and other states because parents can opt out of school vaccination requirements; vaccines of all sorts once again cause autism, that is, if you believe many otherwise intelligent individuals; health facilities are being used for target practice because they provide services not favored by all; mental health initiatives are still unrealistic if one is interested in durable outcomes; and you still don’t want to mess with Texas when it comes to health insurance law.
I recall many years ago being on a jury regarding a medically-related case, with opposing clinical experts being 1) an internationally acclaimed academic expert in the field with a 30 page CV, and 2) a prison doctor. Guess who most jurors believed? Based on the generally marginal faith many citizens appear to have with science and the opinions of scientists, I would expect there would be no difference today.
Robert Roth, M.D., Ph.D.
Vice President and Worldwide Medical Director