FDA IND Requirements by Indication: Standard, Oncology, Rare Disease & Pediatric — Transcript

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1. 0:00Did
2. 0:00you know that the FDA reviews your IND based on the indication
3. 0:05that you're submitting it for? And not every program is the same.
4. 0:08They all depend on what patient population you're going after.
5. 0:11So this chart here, we're going to go through the different indications that we
6. 0:14have and how the FDA has you measure up.
7. 0:18So for a standard indication like metabolic or immunology,
8. 0:25your safety margin expectation is more than 10x of your clinical AUC.
9. 0:29Your study duration has to meet or exceed your clinical regimen.
10. 0:33Your safety farm has to be, you're going to have a couple of standalone
11. 0:37studies for your CV, your CNS, your respiratory usually. You also have integrated endpoints.
12. 0:38Your genotox, you're going to require the full battery before phase one.
13. 0:41And your risk tolerance is low. You must explain all your findings,
14. 0:43that safety margin, that exposure margin has to be adequate enough.
15. 0:47You have to have everything pretty much right on the dot.
16. 0:49And your clinical clinical package, your non-clinical program is going to be strong,
17. 0:52robust, and a lot of work. For serious and life-threatening indications,
18. 0:57oncology, terminal, or post really advanced,
19. 0:59very sick patient populations,
20. 1:01less than a 10x safety margin might be acceptable.
21. 1:04For oncology, sometimes we go 1, 3, or 5x,
22. 1:07but it has to be justified as well. Your study duration,
23. 1:10you can have a 14-day study that may support a phase one in terminal
24. 1:13indications, depending on how sick the patient population is.
25. 1:16And within phase one, you're not going to go into healthy volunteers,
26. 1:19you're going to go into patients. Your safety farm,
27. 1:21we can integrate this into GLP talks a lot,
28. 1:23and you can waive standalone studies. Sometimes you might need a cardiovascular standalone a
29. 1:28safety farm study to understand cardio toxicity But most of the time we can
30. 1:32integrate this into GLP talks have some endpoints like ECGs FOBs
31. 1:36within those studies and then histopath to correlate those findings.
32. 1:39Your genotox, you're going to require at least an AMES test before phase one,
33. 1:43and you're going to require the rest of the battery throughout the rest of
34. 1:45development. Your risk tolerance is a lot higher,
35. 1:48you have a little bit more wiggle room. If your benefit outweighs your risk
36. 1:51for that indication, then you're going to have a much easier time getting through
37. 1:55your IND clearance and getting into humans. Rare disease,
38. 1:59you have a lot of flexibility here, you can see the green here.
39. 2:03So your safety margin expectation is a lot more flexible if your benefit risk
40. 2:06argument is strong. Your study duration will vary.
41. 2:09You'll get flexibility on that but it depends on the disease.
42. 2:12Your safety farm is case-by-case.
43. 2:14It depends on your target findings, how your pharmacology adds up,
44. 2:17and what you're going to need to be able to justify that you have
45. 2:19a safe drug scientifically. Genotox,
46. 2:22your standard battery is usually required. drugs,
47. 2:26sometimes pediatric or genetic disorders, so show
48. 2:29the genotoxicity risk or lack thereof.
49. 2:31Risk tolerance is moderate. Again, these are diseases where severity is considered in a
50. 2:35benefit risk scenario why you should or should not get your
51. 2:40clearance. and pediatric programs, these are a lot more strict programs,
52. 2:43these are usually first in children or an adult pedi combo.
53. 2:46Your safety margin is a lot more higher of a standard.
54. 2:49Your study duration, Juvenile animal studies are usually required on top of typical
55. 2:54standard tox studies as well. Safety pharmacology strongly standalone
56. 3:00studies as well as integrated endpoints. Genotox,
57. 3:03same as adult, standard minimum, doing the battery,
58. 3:06uh, before phase one. And your risk tolerance is very,
59. 3:08very low. there's a higher standard for a vulnerable population.
60. 3:12Pediatrics and going into children, you have a lot more riding on it.
61. 3:16The programs are weighted a lot more because you're going into children,
62. 3:19there's a bigger risk there. So here's the full makeup.
63. 3:22You can see all of the different medications that we have and all the
64. 3:25different things that the FDA is going to require. Save this,
65. 3:28pin it, bookmark it, and if you want to go into more depth about
66. 3:32what the FDA is going to require and how to do a non-clinical regulatory
67. 3:35strategy that will pass your IND, Check out my course the complete guide to
68. 3:38non-clinical development. It'll be pinned in the comments below as well as in the
69. 3:42description of the video