Beyond the Ice Bucket

Five years ago, I was unexpectedly struck by amyotrophic lateral sclerosis, or ALS. I have dedicated my life to moving mountains in what was, at first, an unfamiliar field. My entrepreneurial career included real estate, flowers, oil and shipping. But the past five years had challenged me with the steepest learning curve I’ve ever faced: my disease.

During my crash course on ALS, often called Lou Gehrig’s disease, I noticed that regulatory review and approval seriously hindered the process of drug development and results in missed deadlines.

Although I am an academic drop out, I’ve learned to appreciate science as the backbone of evidence-based theories. However, being a patient with a killer disease means never ruling out miracles – the desire for a cure drives everything I’ve come to appreciate the passion and compassion of people within the ALS community – the patients, researchers and companies that underpin that drive.

I’ve also learned to apply the skills I developed in my previous careers to drive innovation in drug development. This has led to the development of what is currently one of the largest genetic datasets of ALS patients on the globe. The shift in focus from technology-driven to disease-driven drug development is necessary in an industry ruled by science but governed by non-disclosure agreements. The heart of next generation drug development lies here – with innovative research approaches that include the experiences of the patients that the industry is focused on helping.

Today, real innovation increasingly comes as a result of patients’ involvement in drug development. Admittedly, most pharma and biotech companies have patient-centric advocacy embedded in their strategy and mission statements, but patients have traditionally formed just a small part of the drug development equation. The patient voice should echo in all parts of the development path, including governing bodies, ethical committees and expert panels. I’d go as far to say that if that was done 50 years ago, we probably would not have been able to increase fivefold the time it takes for a drug to come to market. Fortunately, this is starting to change.

Patients are increasingly aware of their ability to play a significant role in the biotech R&D paradigm. Social phenomena like the Ice Bucket Challenge and increased patient involvement to accelerate drug approvals have served as good examples for both the industry and the patient community. We should not omit science and data from advocacy campaigns. R&D should remain a scientific process, not one driven solely by popular vote. However, placing patients at the center of the drug development process can help spur changes and direct attention to unmet medical needs.

Industry and government also have responsibility to recognize this shift. Pending U.S. legislation like 21st Century Cures Initiative and industrywide conferences such as the BIO International Convention provide opportunities for patients to make themselves heard, and for stakeholders to recognize these new roles.

I founded a biotech company that aims to address a deadly disease with innovative, transparent research, genetic data analysis and accelerated therapy development. Our for-profit status allows us to apply general business rules to attract capital and advance our mission. We still face the boundaries obstructing fast development tracks, but this generation of patients, “patients 3.0,” will eventually break down traditional boundaries and help streamline and expedite drug development. With more than 5,600 people diagnosed with ALS each year, the sooner, the better.

Bernard Muller

This blog was published as the Congress Blog on The Hill on June 10th 2015