Why the US Lags in Drug Development
The US clinical trial system is failing, with only 4% of patients enrolled. High costs, fragmented services, and outdated processes hinder life-saving drug development.
The successful clinical trials that gave us COVID vaccines and medicines were tested in the UK, not the US. The US lacks a well-functioning system to share data or recruit clinical trial patients. Only 4% of U.S. patients are enrolled in clinical trials, though now (as of a few months ago), the US government’s ARPA-H program aims for 90% of eligible patients to have access.
The lack of easier clinical trials is becoming a main bottleneck for life-saving drugs. A cancer center director says ideas are everywhere. This is without considering advances in AI that could find new molecules 1,000 times faster than existing models.
CRISPR, a groundbreaking gene-editing technology, will soon move from technical improvements to testing treatments. Stalling from lack of clinical trials could hinder groundbreaking advancements in common diseases like heart attacks.
Another issue: clinical trials also test how safe a drug is. In 2004, several drugs made headlines for safety concerns. Today, we’re likely not finding out about safety concerns because those clinical trials are no longer common.
Cost per patient in clinical trials can now reach up to $500,000 per patient, while the number of patients in each study has also increased. Costs are high for a variety of reasons. The fragmentation among service providers (i.e. medical services, technology solutions companies) prevents contract research organizations from reaching economies of scale. The tedious, stressful nature of the work and low career advancement in clinical research organizations lead to high turnover rates and burnout. Participant recruitment costs are high too, partly because clinical trials aren’t allowed to prominently highlight financial incentives. Also, payments for low-income participants are not tax-exempt, meaning low-income participants could lose income-based benefits.
Instead, drug companies focus on rare diseases, which use smaller trials and command high prices. This is likely why average new medicine prices rose from $2,115 to $180,007 from 2007 to 2021. The proportion of drugs costing over $150,000 increased from 9% to 47%.
Under pressure to speed up drug approvals, Congress is leaning toward provisional approval instead of simplifying clinical trials. Provisional approval relies on real-world evidence, which is often less robust than randomized trials. Even the conservative Manhattan Institute recommends against the current Accelerated Approval standard because follow-up studies often don't get done, delaying the detection of ineffective drugs.
What Would Actually Make America Great Again:
Clinical Trials Focusing on How Drugs Work
The Nobel Prize winning scientist who helped develop immunotherapy medicines wants a system where promising smaller studies are done, where patient samples are collected to understand how the immune system reacts to each new drug combination. Right now, clinical trials often combine whatever medicines that company happens to own. Drug giant Merck is doing 211 clinical trials of its drug Keytruda, the third best-selling drug in the world.
High-Quality Accessible Data Ecosystems
In the UK, two scientists used existing health data infrastructure and minimal data collection to determine that the cheap drug dexamethasone reduced ventilator deaths by 35% in COVID patients. The UK and Israel’s organized data systems confirmed that COVID vaccines worked.
The US needs simpler, larger trials with better data systems. Alphabet’s life sciences division is exploring this, but Congress hasn’t acted toward this. To advance, the US must streamline trial processes and enhance data infrastructure.
High-quality, accessible data ecosystems could also reveal why diseases occur. So far, intentional studies are the only way to discover this. A well-functioning system could even conduct randomized trials of existing treatments automatically.
Clinical Trials Shouldn’t Solely Be From Pharma Companies
Clinical trials shouldn’t be limited to just pharmaceutical companies. Insurers and healthcare systems also want to see effective, safe drugs be available. A blood cancer survivor noted that his CAR-T treatment, a therapy that modifies a patient's own cells to fight cancer and saved his life, is not available today due to pharmaceutical companies’ shifting priorities toward new better drugs focusing on blood cancer. The National Cancer Institute is also calling for more stakeholders and communities to be involved in clinical studies in new ways.
Inspiration: This extensive 2022 STAT News piece that stayed on my mind constantly. I wanted to see what parts of the paper had been applied, updated, and make it more accessible by highlighting the most salient parts.