Are we in the golden age of orphan drug development? As I’m recording this on Groundhog Day, the FDA groundhog must not have seen his shadow, he’s come out of his hole and continues to move full speed ahead to approve new drugs, especially for rare and orphan diseases.
In 2018 the FDA set an all time record with 59 new drug approvals, and over half (34) were for orphan drugs. And also a new record for novel medical devices, as 106 were approved. Thanks to Jim Westerkamp of Cliniceval Solutions for posting this information and for Regulatory Focus for publishing their summary.
I’m pleased to say that of the 34 orphan drugs approved, we have patient panels for 28 – that’s 82% coverage. This includes our panels for HIV, ITP, PKU, Dravet Syndrome, Lennox Gastaut Syndrome, Melanoma, , Fabry, Hereditary Angioedema, PNH, cancers such as AML, CLL, FL, SLL, NSCLC, ALL, and GEP-NET. No, we don’t have panels for River Blindness and Malaria!
Of the 25 non-orphan new drugs approved, we have patient panels for 19, that’s 76% coverage. That includes HIV, Prostate cancer, Psoriasis, Migraine, RA, endometriosis, Breast Cancer, and COPD.
And finally, of the 13 selected potential new drug approvals for 2019, we have panels for all 13, 100% coverage! These include breast cancer, MS, thrombocytopenia, depression, Psoriasis, AML, SMA, peanut allergies, bladder cancer, RA, osteoporosis, and osteopenia.
So if you are looking to get patient and caregiver reactions to these new and upcoming treatments, we’d be happy to provide the patients for your qual and quant studies.
We’re posting our counts for our panels for each of these new drugs. As always, check out our counts on our website: rarepatientvoice.com, go to the For Researchers tab and select Patient/Caregiver Panels.