FDA's new Rare Disease Framework should include ultra-rare cancers. Cancers with one known driver need a clear path too.
I'm filing a comment asking for disease-specific guidance for these cancers, fibrolamellar included. If FDA spells out what it takes to get a drug approved, more companies will be willing to invest in these cancers.
My five asks are below. Comments are open through Dec 11.
@FDADrugs@RareDiseases@CancerResrch@FibroFoundation@reaganudall@KIDSvCANCER@FDA@FDA_KyleD
New study: HLRCC fibroids appear ~age 32, are nearly always multiple, and often lead to hysterectomy by 35. Early detection could unlock kidney screening in time. #HLRCC#WomensHealth https://t.co/OeOB7kmU1d
@eschwitz I’m so sorry to hear this and suggest you connect with my wife @lynnsdays_ who is not only fighting kidney cancer herself (HLRCC) but is a fierce patient advocate - there is a great network of doctors in SoCal who can offer a second opinion etc - praying for you!
I’m 37 and living with stage 4 HLRCC, a rare kidney cancer. Small patient populations make large trials difficult but our need for effective treatments is no smaller. Make rare cancers an explicit priority in this framework. Our voices and our futures belong at the table.
Not every clinical trial is right for every patient. That's why finding relevant options starts with understanding more about YOU!
The KidneyCAN Clinical Trial Navigator considers several important factors when identifying potential matches, including your diagnosis, your treatment history, your disease characteristics, and your location.
Using this information, the Navigator helps identify clinical trials that may be relevant to your specific circumstances, making it easier to explore your options and discuss them with your care team.
Ready to see what's out there? https://t.co/dq6eUswpbB
#AcceleratingCures
@Chubabubba I’m a caregiver to my wife, we heard crazy things early on - she’s on a clinical trial now for stage 4 HLRCC, and we don’t intend on giving up. Fight like hell and f the idiot doctors lacking bedside manners projecting
Tomorrow at AUSA, we’ll unveil something we held back at Ascend, built for the hardest ISR missions.
Streaming live 10/13, 5:45 PM EDT: https://t.co/wZkryLBDK5
@poteto@poteto - Awesome, signed up - but running into issues merging my accounts despite paying for the new subscription - had trouble merging my various accounts :(
@milesdeutscher It blows my mind that there are people out there who are close to tech and yet plainly refuse to even experiment and get some hands on time 🤦♂️
@AlecTPhD Congrats! I’m excited to see people like yourself get involved with @OpenEvidence. My wife @lynnsdays_ is fighting HLRCC (FH-deficient RCC) and we remain hopeful that resources applied in healthcare can better educate, prevent, and someday even cure a rare cancer like hers. 🙏