Many clinical trials fail not because the science is wrong, but because of operational issues like patient recruitment and retention. These problems often stem from overly burdensome and rigid trial designs that deter participation, a preventable error.
Many startups focus only on reaching the next clinical milestone, creating operational "islands." This leads to post-approval crises when they haven't considered formulation, payers, or pricing. Integrating commercial strategy from Phase 1 is essential for long-term survival and a successful launch.
For companies with multiple similar assets, like ASOs for related genetic disorders, umbrella studies offer a streamlined path. By getting FDA buy-in on a master protocol, companies can add new drugs as "baskets" without filing full, separate INDs, accelerating development and reducing paperwork.
Scientists often design trials to answer every possible academic question, which adds complexity and patient burden. Drug development trials should be ruthlessly focused on two things only: safety and efficacy. All other extraneous research can wait for post-approval studies.
The "time is lives" mantra also applies to the companies themselves. For single-asset biotechs with short financial runways, trial delays can bankrupt the company before the drug has a chance. "Time to first patient" is a critical business milestone, not just a clinical one.
Designing a lean $25M trial instead of a $100M trial makes a startup unattractive to large VCs who won't write small checks. However, this capital efficiency opens funding opportunities from family offices and smaller groups who prioritize impact alongside returns and can be more patient investors.
The highly personalized, N-of-1 approaches developed for rare diseases are not a niche field. With advanced genetic sequencing, it's becoming clear that every disease is effectively rare and unique to the individual. The lessons from rare disease are creating the foundation for all future medicine.