This was originally written as a comment on the new podcast about medical innovation called Hard Drugs by Works in Progress.
The last couple of days I've been thinking about how to solve a disease in general, and which levers there are for patients & policymakers. I wish there was a playbook!
I identify 3 major levers:
1. Research quantity/funding. This is the obvious lever that a lot of advocacy focuses on. Simple to understand, but hard to convince politicians of! What does the shape of the input-output curve look like, and when do diminishing returns really set in? (ME/CFS funding is like 10x below average, so obviously there's a lot of focus on funding)
2. Research quality. I have the impression that this matters greatly, and spans many factors or even orders of magnitude. You can have heaps of money, but with a toxic field you don't get far (think Alzheimer's). For ME/CFS, 89% of studies in the last decade did not even mandate subjects to have the defining symptom of the disease (Post-Exertional Malaise), which gives a general sense of how bad the field is. imagine a lot comes down to peer review quality (I'm excited for Al improving the process), and perhaps some diseases offer more prestige so attract more talent? think patients can affect this, but it's hard. I've also seen IFP innovating the grant-making process, and would like to add that patients have a pretty unique perspective and e.g. the Patient-Led Research Collaborative had a pretty good regranting program. I imagine a couple of separate entities, one run by patients, would be far more effective than trying to involve patients in in the legacy process where they're often not empowered.
3. Market incentives. Others have said a lot about this. FDA/ EMA processes have pushed the cost of drug development >$1 billion. Earlier market access while still forcing rigorous Phase 3 trials eventually seems like a good policy. In my uninformed impression, patient advocacy groups don't focus much on this, and don't understand the pharma industry well. I wish they would collaborate more to pressure reform here.
More generally, I wonder which theory of change is more accurate/cost-effective:
A) Understand first. Figure out how a disease works through non-drug research, then try to solve that
B) Trial-and-error. Run trials trying to understand and maybe coincidentally hit something that works.
I feel like A is often the assumed model, but B would probably work better, IF drugs were easier to get to market.
The last couple of days I've been thinking about how to solve a disease in general, and which levers there are for patients & policymakers. I wish there was a playbook!
I identify 3 major levers:
1. Research quantity/funding. This is the obvious lever that a lot of advocacy focuses on. Simple to understand, but hard to convince politicians of! What does the shape of the input-output curve look like, and when do diminishing returns really set in? (ME/CFS funding is like 10x below average, so obviously there's a lot of focus on funding)
2. Research quality. I have the impression that this matters greatly, and spans many factors or even orders of magnitude. You can have heaps of money, but with a toxic field you don't get far (think Alzheimer's). For ME/CFS, 89% of studies in the last decade did not even mandate subjects to have the defining symptom of the disease (Post-Exertional Malaise), which gives a general sense of how bad the field is. imagine a lot comes down to peer review quality (I'm excited for Al improving the process), and perhaps some diseases offer more prestige so attract more talent? think patients can affect this, but it's hard. I've also seen IFP innovating the grant-making process, and would like to add that patients have a pretty unique perspective and e.g. the Patient-Led Research Collaborative had a pretty good regranting program. I imagine a couple of separate entities, one run by patients, would be far more effective than trying to involve patients in in the legacy process where they're often not empowered.
3. Market incentives. Others have said a lot about this. FDA/ EMA processes have pushed the cost of drug development >$1 billion. Earlier market access while still forcing rigorous Phase 3 trials eventually seems like a good policy. In my uninformed impression, patient advocacy groups don't focus much on this, and don't understand the pharma industry well. I wish they would collaborate more to pressure reform here.
More generally, I wonder which theory of change is more accurate/cost-effective:
A) Understand first. Figure out how a disease works through non-drug research, then try to solve that
B) Trial-and-error. Run trials trying to understand and maybe coincidentally hit something that works.
I feel like A is often the assumed model, but B would probably work better, IF drugs were easier to get to market.
