The typical timeframe between the discovery of a substance with potential therapeutic effects and its approval as a registered medicine is about 10–15 years. This lengthy process is often attributed not only to technical hurdles but also to the social and institutional requirements involved. In fact, only about 1 in every 5,000 to 10,000 discovered compounds ultimately becomes an approved drug. However, as demonstrated during the COVID-19 pandemic with the rapid development of mRNA vaccines, it is possible to expedite this timeline while maintaining rigorous safety and efficacy standards.

The Standard Process

Discovery & Preclinical Research (3–6 years)

• Discovery: Identifying a potential therapeutic compound and conducting initial lab-based screens.
• Preclinical Testing: Assessing efficacy and safety (toxicology) in laboratory and animal studies. If results are promising, the sponsor files an Investigational New Drug (IND) or Clinical Trial Application (CTA).

Phase I Clinical Trial (1–2 years)

• Objective: Determine safety and dosage in a small group of healthy volunteers or patients (20–100).
• Outcome: Data on side effects and how the drug is metabolized or excreted.

Phase II Clinical Trial (1–2 years)

• Objective: Test effectiveness in a larger group (usually a few hundred) with the target condition.
• Outcome: Early insights into therapeutic benefit, refined dosage, and a more detailed side-effect profile.

Phase III Clinical Trial (2–4 years)

• Objective: Confirm effectiveness in a large population (often in the thousands), comparing it to existing treatments.
• Outcome: Comprehensive data used for regulatory approval.

Regulatory Review & Approval (1–2 years)

• application: Submission of a New Drug Application (NDA) or Biologics License Application (BLA) in the U.S., or a Marketing Authorization Application (MAA) in the EU.
• Outcome: Agencies like the FDA or EMA review the data, inspect facilities, and decide if benefits outweigh risk

Phase IV (Post-Marketing Surveillance) (ongoin

• Objective: Monitor real-world safety and effectiveness.
• Outcome: Identification of rare or long-term side effect

While this procedure is acceptable when a new medicine offers only incremental improvements over existing treatments, it can be far less tolerable for diseases lacking any effective therapy. One example is Parkinson’s disease, a progressive loss of dopaminergic neurons that currently has no cure. Research increasingly points to alpha-synuclein fibril buildup and neuronal uptake as key mechanisms in Parkinson’s. Over the last three years — especially in the last three months — tremendous progress has been made in understanding these processes. Substances that inhibit alpha-synuclein oligomerization or fibril uptake are now entering, or are already in, Phase I clinical trials. Unfortunately, most such trials are geographically limited, leaving participation open only to those in specific regions.

Introducing the “Pioneer Tester” Concept

I propose establishing a “Pioneer Tester” program that would permit individuals from anywhere in the world to gain access to new substances entering Phase I testing. Participants would sign a waiver exempting the company or study group from liability, and in return receive the investigational substance at no cost. Their only obligation would be to provide regular reports on their experience. Although likely only a few people would choose this path, those willing to assume the risk should have the opportunity to do so.