Doctors Welcome Informed Creativity

Siqi Chen shares his experience navigating the medical system for his daughter's rare brain tumor, revealing how motivated individuals can go beyond standard care by becoming deeply informed and proposing alternative treatments.

• The gap between standard medical care and frontier treatments is enormous

  • "What is available as standard of care... and what is available at the frontier, there's a huge gap"
  • For rare diseases, the gap is even wider due to limited research and economic incentives

• Motivated individuals can become more knowledgeable than specialists

  • "Because this disease is so rare, I am like more knowledgeable about the disease than anyone in the room because they have to like study 50 different cancers"
  • This knowledge allows patients/caregivers to propose viable alternative treatments

• Doctors may privately acknowledge limitations of standard care

  • "I straight up said I do not care what the standard of care is, I think the standard of care is crap and she basically said yeah I think so too, I just can't say that"
  • Some clinicians welcome well-informed partners who think outside the box

• Finding one doctor willing to prescribe off-label is the key

  • "You do have to like show enough research and be well informed enough that someone is willing to prescribe it off label on a compassionate use basis"
  • "You just need to convince a doc, a one doctor... and then you're in the clear"
  • "It's a lot easier if it's like a fairly serious rare disease"

• The medical research system has structural problems

  • IP issues prevent research on potentially effective existing drugs
  • "There's been no clinical trials for it because there's no money for it because you can't patent a pinworm drug"
  • Research funding flows to patentable molecules even when existing treatments might work

• Highly motivated individuals can create their own solutions

  • "There's no one more motivated than a dad with a sick kid"
  • Some founders have gone as far as manufacturing their own drugs for rare diseases
  • "End of one cures exist" for those sufficiently motivated