In the race to find effective COVID-19 treatments, scientists are setting up clinical trials that test multiple drugs simultaneously in the hopes of quickly determining which ones work best.

These "platform" trials have already led to significant breakthroughs in the race to identify effective and safe treatments to cure COVID-19.

Traditional trials evaluate whether a single experimental treatment improves patient outcomes as compared to a placebo or existing standard of care. This approach is scientifically sound but quite inefficient. That's because if multiple experimental drugs are ready for human testing at roughly the same time, those treatments must be evaluated individually. Each traditional trial requires its own funding sources, collaborating physicians, and patients. And since data collection techniques might vary across those trials, it's difficult to make head-to-head comparisons about which drug works best.

By contrast, in platform trials, researchers can test multiple interventions simultaneously. The most promising candidates move on to the next stage of the trial, while treatments that don't perform well get dropped. And researchers can introduce new potential treatments at each phase. This approach has many advantages. It's quicker, requires fewer participants, including a smaller control group to receive either a placebo or the standard of care, and allows for head-to-head comparisons.

Platform trials would prove especially helpful in rare disease research. Since these illnesses affect small to very small numbers of patients, traditional trials requiring large groups of participants aren't feasible. Rare diseases also tend to attract less funding. Platform trials can provide high-quality evidence without a large participant pool—and at a lower cost than standard trials.

Recognizing these benefits, the Children's Tumor Foundation, where I serve as president, is co-funding the INTUITT-NF2 trial together with Takeda. This multi-site innovative platform trial can evaluate treatments for multiple tumor manifestations in neurofibromatosis type 2 (NF2) simultaneously. NF2 is a rare genetic disorder that results in tumor growth on the nerves and in the brain.

While this promising approach is hopefully becoming mainstream, the biggest challenge of platform trials, especially in rare diseases, is the access to treatments.  For INTUITT, for example, we recruited the NF2 patients for the first drug arm in a record time, and although patients are waiting for the next drugs to come, we have so far been unable to access the drugs we need. These failures are not because of the ill-will of anyone but because the current operational, legal and social systems are outdated. The existing systems complicate drug repositioning and drug combinations, especially if the drugs belong to different companies.

Driven by our commitment to accelerate patients' access to treatments, our organization is very proud to play a leadership role in two projects: BRIDGE and EU-PEARL.

BRIDGE, driven by three mission-driven non-profit organizations (FasterCures, CureSearch, and the Children’s Tumor Foundation), is entirely focused on developing a framework to effectively exit effective and safe drugs - discontinued for operational, strategic, or economic reasons- from pharma and biotech.

The EU Patient-Centric Clinical Trial Platforms (EU-PEARL) project, a public-private partnership project from the IMI (Innovative Medicines Initiative), is developing platform trials for four very distinct diseases, including neurofibromatosis. The primary value of EU-PEARL is that this project will not only address the scientific challenges of platform trials, but this project will deliver a regulatory and business framework for the effective execution of platform trials.

All challenges are well worth overcoming. Patients' lives are at stake.