What Really Moves Drug Design Forward?
We’ve got CRISPR gene editors, AI that predicts protein structures in minutes, and gene therapies sprouting across pipelines—but bringing a new drug to market still takes 10 - 15 years and costs about $2 - 3 billion. It’s a phenomenon known as Eroom’s Law, shorthand for slow innovation despite tech progress. Why this disconnect? This post pulls back the curtain on the whole innovation machine: science, incentives, regulation, manufacturing, pricing, and trust. I’ll frame it with two starkly different cases:
Casgevy, the first CRISPR-based therapy that succeeded when everything aligned.
Aduhelm, an Alzheimer’s drug that flamed out despite early enthusiasm.
Then I’ll widen the lens to show how discovery science is actually speeding up, and whether other gears are keeping pace.
The Eroom’s Law Paradox
When Jack Scannell’s team coined Eroom’s Law, they meant it: drug R&D is slowing and becoming more expensive. Today, it typically costs $2.5 billion and takes 12–15 years per drug—and these figures double roughly every nine years . That’s science swinging forward against regulatory, financial, and systemic friction.
The Five Gears That Drive Innovation
Successful drug design happens only when all these gears engage:
Science: breakthroughs like CRISPR, AI-assisted biological models, programmable mRNA sensors, etc.
Incentives & Funding: public grants, patents, market exclusivity.
Regulation: pathways like fast-track, orphan, and RMAT designations.
Manufacturing & Delivery: capacity to produce and administer therapies.
Economics & Access: pricing models, payer coverage, and infrastructure.
Let’s see what happens when one or more gears misfire.
Casgevy: All Systems Go
Approved in December 2023, Casgevy (exa‑cel) treats sickle-cell and β‑thalassemia - 11 years after CRISPR’s discovery.
Science & Speed: Lab to patient in just 11 years.
Incentives: Received fast-track, orphan, and RMAT status from the FDA.
Manufacturing & Delivery: 45 centers in the US, with plans for ~75 globally.
Cost & Economics: Priced at $2.2 million per patient.
Casgevy shows the magic that happens when gears synchronize: platform tech, incentives, infrastructure, and pricing all in lockstep.
Aduhelm: A Gear in Crisis
Aduhelm (aducanumab) was approved in June 2021 under accelerated FDA guidelines, despite evidence of only modest amyloid plaque reduction and inconclusive clinical benefit .
Regulatory discord: Approved despite unanimous FDA advisory committee rejection, with allegations of unconventional FDA-Biogen dealings.
Pricing & payor pushback: Priced initially at $56,000/year, deeply contested by payers; Medicare restricted coverage to clinical trial participants.
Market collapse: Generated only $4.8 million in 2022; Biogen wrote off $233 million in inventory and took a $60 million charge.
Public trust crisis: Congressional and media outcry framed it as one of FDA’s worst decisions.
Aduhelm teaches that approval without system alignment can backlash.Evidence That Innovation Is Accelerating
This isn’t just hype, real progress is happening:
Gene Therapy
Approvals rose from 1–2/year to 7 in 2023, 6 in 2024, and 17 expected in 2025.
Therapies like Hemgenix and Roctavian report durable results.
AI in Drug Discovery
5.2 million AI-designed molecules released by SandboxAQ for accelerated binding models .
AlphaFold, the Nobel-winning tool, shrunk protein structure prediction from months to minutes .
Startups show speed gains:
Insilico: IND in 18 months at 10% cost
Exscientia: OCD drug in 12 months
AI‑identified COVID therapy in 48 hours
Are We There Yet?
Science
CRISPR drug in 11 years; record gene therapy approvals
AI & Computation
5.2 M molecules fueling AI models; Nobel-scale tools; accelerated drug timelines
Regulation
Casgevy’s fast-tracking; Aduhelm’s regulatory cautionary tale
Manufacturing & Delivery
45–75 Casgevy centers; CDMOs scaling—but still nascent
Economics & Accessible
$2M+ therapies emerging; payers catching up cautiously
Yes, the gears are beginning to spin in harmony. But as Aduhelm shows, one faltering gear can stall everything. The runway is shorter than before, but we still need to finish building while flying.
What’s Needed Next
To hit full speed, we must:
Expand funding models from discovery to delivery
Build manufacturing into R&D pipelines early
Align pay-for-outcomes pricing with curative potential
Strengthen trust between regulators, science, and public
Encourage pharma strategies centered on innovation ecosystems
Final Thought
Innovation isn’t just about clever molecules, it’s about orchestrating entire ecosystems. The data is accelerating. Now, we just need the rest of the system to catch up.
References:
Cost of drug development: https://pmc.ncbi.nlm.nih.gov/articles/PMC11214120, https://www.phrma.org/policy-issues/research-development
Casgevy: https://www.isct-cytotherapy.org/article/S1465-3249(24)00736-9/fulltext
Aduhelm: https://healthjournalism.org/blog/2024/02/adieu-aduhelm-biogen-pulls-plug-on-controversial-alzheimers-drug/, https://www.fiercepharma.com/pharma/biogen-files-233-million-aduhelm-write-wake-aduhelm-overhaul