If Orphan cancers were a disease category by itself, it would be one of the top 5 largest killers in the USA just behind Heart Disease, Cancer, and #COVID19. I have myeloma and had the opportunity to go to ASH 2019, and it happened to be one of the best opportunities to see the future of blood cancer treatments. For many of the meetings you will see some of the most significant developments in cancer treatment like CAR T, ADC(Anitbody Drug Conjugates), AWC(Antibody Warehead Conjugates), BiTE’s, etc. and they are held in rooms which can seat hundreds or sometimes thousands of people.
However, the one thing all of the drugs under development have in common is they all must obtain FDA approval. The FDA had two meetings scheduled. It was for this reason I wanted to make sure I attended the meeting titled “FDA Dialogue with Patient Representatives and Advocates”. Myeloma clinicians and advocates had just gone through a very difficult approval process for Selinexor, and I wanted to find out what I could learn at this meeting. I almost missed the meeting because most are in large halls, and this meeting was in a meeting room, so I kept walking past it thinking this little room could not be the venue for a meeting as important as it seemed to me. As you can see in the following image the advocates meeting was in OfficeW206, and the other FDA meeting and most meetings in a hall the size of the grand Hall D.
At this meeting the FDA was well represented with 38 in attendance, and they seemed to outnumber the advocates by a factor of two to one. I was frankly shocked at the poor turnout of advocates, in that these FDA officials held the final judgment on approval or rejection.
We were able to provide inputs to the group, praised them for final approval of Selinexor, asked for more representation specific to the disease to be at the the Oncology Drug Advisory Committee meetings to discuss the FDA concerns, and to recognize this is a last chance at life for late stage patients. For example if there were just one more myeloma specialist (or a total of two) of the 13 members on the ODAC(oncology drugs advisory committee), Selinexor would have been approved months earlier.
Small Biotech companies are at a significant disadvantage in the universe of drug companies. They are small and find it hard to fund themselves as they work through a 8 to 10 year FDA approval process. They seldom profit during these initial development years and find it impossible to borrow money, so they must depend on the capital markets for funding. As I had written before, the SEC has few tools to protect these baby biotechs from Capital Vultures(CLICK HERE for the article) who manipulate the stock, an illegal tactic driving the stock price down. This makes it even more difficult to fund their trials and these baby biotechs are forced into bankruptcy. I would love to see the SEC with the enforcement power to put these animals in jail, as well as the government provide a guaranteed loan program as another funding avenue, and prevent the use of viscous short sale attacks designed to profit from the drastic decline in a vulnerable biotech stock price. Because of these disadvantages we need all the allies we can get and must have more protections for small biotech firms.
One way as advocates and disease representatives to champion your orphan disease would be to attend any of these FDA/Advocate programs. Or send this article to your Senate and House representatives (CLICK HERE for contact information for your senators and representatives), @secazar, and @SteveFDA .
Good Luck and may God Bless your family's cancer journey. You can see more information on myeloma at my site www.myelomasurvival.com.