Desenvolvimento de Novos Medicamentos - Slides de Apresentação

Look, you've got discovery and preclinical testing first - that's where you find your compound and prove it works in labs and animal studies. Takes about 3-6 years, which feels forever but trust me on this. Don't rush the preclinical stuff or you'll get burned later with failed Phase I trials. Quality preclinical data makes your IND application so much smoother. FDA won't give you weird looks in pre-IND meetings either. Honestly, just bite the bullet and do rock-solid preclinical work upfront. Yeah it sucks waiting, but you'll save years and ridiculous amounts of money later.

So the FDA is basically watching over your shoulder through every step of drug development. Before you can even start Phase I trials, you need their green light. Same goes for each phase after that. They're reviewing your protocols, safety data, manufacturing details - the whole nine yards honestly. If they think the risks outweigh benefits, they'll shut you down or make you jump through more hoops. Here's what saved me headaches: book those pre-IND meetings early. Trust me, finding out about issues later costs way more than dealing with their feedback upfront.

So preclinical trials are where you test everything in labs and on animals first - basically the "will this actually kill people" stage before human testing. You're checking if the drug is toxic, how the body processes it, whether it even works. Pretty unglamorous but you can't skip it. The FDA absolutely won't let you touch humans without solid preclinical data first. It's also how you figure out what doses to start with later. My cousin works in drug development and she says this phase takes forever but saves lives down the road.

So Phase I is tiny - like 20-100 people - just checking if it'll kill you basically. Phase II bumps up to a few hundred to see if the thing actually works. Then Phase III is massive, thousands of people, your drug vs whatever's already out there. That's where companies either strike gold or lose millions, honestly. Think of it like testing a recipe on yourself, then your friends, then opening a restaurant. If you're looking at drug stocks or whatever, Phase III results are what really matter since that's when you find out if doctors will actually prescribe it.

So basically they partner with community health centers and advocacy groups to reach different populations. Transportation and translation services help remove barriers too. The FDA now requires diversity action plans which is actually pretty cool - about time they addressed those historical gaps. Some trials are going decentralized so people can participate from home, opens things up big time. Oh and flexible scheduling helps a lot. Start planning your diversity strategy early though - trying to fix recruitment issues mid-trial is a nightmare. Way easier to get it right from the beginning.

So basically three things kill most drug candidates - safety issues, the drug just doesn't work, and crappy pharmacokinetics. Safety problems alone wipe out like 30% of them (makes sense, nobody wants to get sued). Then efficacy failures hit another 40-50% because surprise, your miracle compound actually does nothing. PK issues are brutal too - drug gets broken down too fast or your body won't even absorb it properly. Oh and lately? Commercial viability is becoming huge. Companies won't touch anything that won't make bank. My advice - do your safety and PK homework early or you'll burn through millions in Phase II trials on something that was never gonna work.

Look, money literally controls everything in drug development - it's honestly depressing how true this is. Solid funding means you can hire the best people and run trials with enough participants to actually prove your drug works. Without it? Companies cut corners on Phase III trials or can't afford proper regulatory help, even when their science is solid. Most teams blow their budget estimates by like 30-50% too (learned that the hard way). Get your funding locked down early and pad those numbers way more than you think you need to.

Dude, AI is seriously cutting drug development time by like years - they can predict if compounds will work before labs even make them. Pretty wild. CRISPR's obviously the big one for genetic stuff, but I'm really seeing digital twins and organ-on-chip tech replace a lot of animal testing. Way more accurate for human responses anyway. Plus all those wearables and patient apps are feeding real data into trials now instead of those annoying monthly check-ins. Honestly, if you're starting any new projects, you'd be crazy not to look into how these could speed things up.

So pharmacogenomics is totally changing drug development - companies can now look at your DNA and predict if a drug will work for you or mess you up before you even try it. Pretty crazy, right? Clinical trials now start with genetic screening instead of just throwing medications at random people and seeing what happens. Drug companies are pairing new treatments with genetic tests from day one. My advice? Don't treat genetic testing like some fancy add-on anymore. It's becoming standard practice, so factor it into whatever strategy you're building from the start.

Okay so patient safety has to come first, obviously. Be super transparent about all the risks and side effects when you're getting people to sign up. The paperwork is honestly insane but there's a reason for all of it. Don't go after vulnerable groups unless your drug actually helps them specifically. Independent ethics committees need to review everything before you can even start - and honestly, they can be pretty picky. My biggest advice? Factor in time for ethics reviews right from the beginning. I've seen people try to rush this part and it always comes back to haunt them later.

So biotech companies team up with big pharma because they need the cash and expertise to actually get drugs to market. Biotechs are great at the creative stuff - taking risks on weird new targets that might not work. But then pharma swoops in with their massive clinical trial networks and regulatory teams. Plus all that manufacturing infrastructure that costs insane money. The milestone payments usually tell you who's really in control of the deal. Smart biotechs negotiate decent royalty splits upfront because that's where the real money is later. It's actually pretty brilliant how they divide up the work - each side does what they're best at.

So post-marketing surveillance is like your backup plan once drugs are actually out there. Clinical trials miss stuff - they just do. Real people using these meds reveal side effects that never showed up in those perfect lab conditions. Honestly, it's kind of wild how much we don't know until millions start taking something. You'll see rare reactions, weird drug interactions, all sorts of things. Document everything you notice though. That feedback literally changes warning labels and dosages. Sometimes drugs get pulled completely if things go sideways.

Patient advocacy groups are surprisingly powerful. They lobby the FDA directly for faster approvals and fund research for rare diseases that big pharma ignores. Honestly, they're probably one of the most underrated players in drug development. These groups also help design trials with patient-friendly endpoints and recruit participants - some even run their own research foundations now. The smart move? Engage them early, especially for rare diseases. Their voice carries serious weight with regulators, way more than most people realize.

Oof, drug trial failures hit pharma companies HARD. We're talking hundreds of millions down the drain, sometimes over a billion per compound that doesn't work out. All those R&D costs, clinical trials, regulatory stuff, staff salaries for years - then nothing. That's honestly part of why successful drugs cost so much. The winners have to pay for all the losers, and like 90% of late-stage trials fail anyway. Pretty depressing stats tbh. Companies basically have to spread their bets across multiple compounds so they don't go under from one bad result.

So basically WHO, Gates Foundation, and Doctors Without Borders step in where big pharma won't go. Companies don't want to develop malaria or TB drugs because those patients can't afford crazy Western prices, right? These organizations fill that hole through grants and partnerships. Sometimes they'll even do research themselves. The regulatory stuff and distribution - they help with that too once drugs get approved. Oh, and their funding is way more accessible than you'd think. If you're doing anything with tropical diseases or rare conditions, definitely check them out.