Let us begin this article recently published in MedCity News by starting with the ending: in order to help millions of rare disease patients waiting for rare disease treatments, a variety of new strategies are required for clinical trials. We cannot rely solely on technology. Drug companies must collaborate, not compete.

The following opinion is that of Andrea Bastek Ph.D., Director of Innovation at Florence, a Remote Site Access platform. Ms. Bastek has prioritized finding workflow efficiencies to improve clinical trial execution.

An Overview

Simply stated, millions of people in the world have a rare untreated disease. They face a devastating lack of treatment options for life-threatening and degenerative diseases. Ms. Bastek suggests that a new approach for clinical trials is needed.

It is true that technology is important. Technology helps rare disease trials by:

• Increasing the rate of recruitment
• Improving communication
• Speeding workflow
• Optimize funding

But it is not the only answer. New methods of patient recruitment such as patient recruitment software, telemedicine, and eConsent are needed by sponsors and research sites.

They should form partnerships and consider adopting the accelerated approval pathways used by the FDA. Note that any regulatory process that expedites treatment is called an accelerated approval path.

Technology can only go so far by quickly activating trial sites and helping trial sponsors set up the clinical trials. But that is only a small part of the overall path to accelerated approval.

An example of the efficiency of accelerated approval programs together with surrogate endpoints is the reduction of clinical trial costs from about ninety million dollars to between twenty-eight and forty million dollars per trial. Surrogate endpoints involve data showing that treatment can ‘combat’ a disease despite the fact that the researchers have not yet made a determination about the outcome. It has been proven that using surrogate endpoints gets more treatments to market.

Rare Disease Clinical Trial Challenges

• Locating enough patients to conduct a meaningful trial
• Finding the even smaller number of people that have the disease the clinical trial is studying
• Rare disease patients generally need to be recruited from around the world, creating difficulty in coordinating sites in multiple locations worldwide.
• Data and documents must be collected involving complex privacy regulations
• It is more difficult to acquire funding

And if this list is not challenging enough, the COVID pandemic has been delaying many trials. Currently, eighty percent of clinical trials cannot complete enrollment on time.

Recruiting Patients for Clinical Trials

Software that uses AI is able to match patients with clinical trials that are suited to their diagnosis or genetic profiles. Patients should be aware that if they submit their genetic profile to the database they are providing valuable information because most rare disorders are genetic.

The distance to the clinical trial sites and the cost of transportation have kept many patients from participating in clinical trials. Although the cost of the treatments is covered, the transportation is not.

Telehealth calls and mobile apps would greatly increase trial participation. It is estimated that ninety-five percent of research sites offer telemedicine visits. Ninety-one percent are on target to use electronic informed consent documents.

It is now possible for patients to go to local pharmacies or doctors’ offices rather than traveling long distances to medical centers.

Collaboration vs. Competition

By thinking in terms of collaborating rather than competing with each other, researchers can pool their funding and pool rare disease participants for improved study results.

The Rare Disease Clinical Research Network was created by the National Institutes of Health. The Network shares data about disease progression and examines the best treatment options.

Looking Forward

Sponsors and sites must work together without being limited by international borders. They should participate in accelerated approval programs. A variety of innovative strategies must be put in motion to help the millions of people with rare diseases waiting for their life-changing treatments.