A Discussion with Michael Kornhauser - A Rare Disease Ally in Clinical Research

About Michael Kornhauser

For over 15 years Michael has skilfully navigated assets through the entire clinical development lifecycle, concentrating on rare diseases, neurological, and mental health disorders. As a rare360 team leader with the Rare Advocacy Movement (RAM), Michael excels at aligning the lived experiences of the rare disease community with a product’s intended use. His clinical operations expertise, strategic post-registration insights, commercialization acumen, and holistic perspective drive the successful development of innovative therapies in the competitive rare disease market. Michael's input facilitates and enhances optimal outcomes for patients, providers, and pharmaceutical companies.

Driven by a profound commitment to improving patients' lives and backed by a track record of accomplishments, Michael continues to devote himself to advancing healthcare and nurturing meaningful collaborations between the community and the biotechnology/pharmaceutical industry.

A Discussion with Michael Kornhauser

Q. What motivates you to work in clinical research, particularly in the areas of enhancing patients' lives and driving advancements in healthcare? 

A. My twenty plus years of experience in clinical management and therapeutic treatment development have impressed upon me the impact of expediting solutions to patients. I've seen firsthand how countless small errors can accumulate, significantly delaying the clinical development process and, in severe cases, obstruct a much-needed breakthrough from reaching patients. Through my work as an industry-based consultant, it is a privilege to be able to facilitate the acceleration of recruitment timelines, rescue clinical studies, manage crises, and proactively avert future obstacles, so that new therapies can become available for people who need them without delay.

Q. The most significant impact on rare disease families comes with speedily delivering life-changing therapies to patients. In your experience, what is the single most important factor for expediting the clinical research process? 

A. The most commonly known challenge is matching patients to the right studies, which is crucial given the potential outcome for both patients and trial sponsors. The lesser-known challenge is the patient experience and that of their support network. Regrettably, many patients and their families endure significant burdens: travel, lifestyle disruptions, and potential adverse events, all for uncertain benefits. I believe in navigating the process with the families via simple, clear, and timely communications, while proactively minimizing potential pain points. This approach has helped in the acceleration of timelines across numerous studies and a dramatic decrease in trial dropout rates due to logistical or technical difficulties. Because retaining participants is just as important to expediting a trial as to finding them, it is essential to create a good experience for patients and their families.

Q. What is crisis management and trial rescue, and why is it important to have good partners to help patients through?

A. Unforeseen issues tend to arise in all human affairs, and clinical trials are no exception. While it may be a small matter to a trial protocol, we know that even a small hiccup can be disruptive for patients and their families if not immediately and effectively addressed. I believe having the right partner they can rely on for timely, reliable communication can save the patients and their families from an immense amount of undue stress and a subsequent emotional roller coaster. One of the reasons I founded my current organization, with some like-minded colleagues, was to be this trusted partner for patients.

Q. Collaboration between pharma, academia and the patient community is crucial for the success of a clinical trial and all the more so rare disease trials. How do you balance the priorities of all parties to ensure that projects move forward efficiently while still considering the patients' well-being? 

A. Clinical trial research navigates the delicate interplay between scientific rigor and the unpredictable nature of human experiences. My colleagues and I balance the precision required, by academic institutions and pharmaceutical sponsors, with patient needs to achieve a seamless integration of trials and therapies into the lives of rare disease patients. In recognition of the unique challenges in each patient’s life, we embrace innovative approaches such as home clinical trial nursing to bring the trial directly to patients. In so doing, we align interests of both sponsors and rare disease families to benefit everyone involved. This focus on empathy and personalized care leads to enhanced participant engagement and better outcomes.

Q. What is the cost to study sponsors, patients, and their families?

A. When it comes to clinical studies, patients and their families should bear no cost.

For trial sponsors, a patient-centric approach could significantly diminish the risk of the following, each of which carry both monetary and opportunity consequences: a) exceeding trial recruitment timelines and delaying crucial milestones, b) compromising data integrity, c) loss of essential rare disease community support, crucial for FDA and regulatory submissions.

I seek to mitigate these risks and offer a therapeutic program the best chance of success with our clinical and community expertise.

Q. And lastly, what gets you out of bed each morning?

A. Besides making sure my kids get up for school and packing their lunches, it’s knowing that I will get to have authentic conversations with real people, and help them in meaningful ways.

I’ll always remember this one gentleman. He still had his family, but his friends had stopped visiting. No one said it was because his condition made them confront their own mortality, but one suspects. We spoke often throughout the trial. He used to speak fondly of his work (helping those in need) and children, and worried that his grandchildren wouldn’t remember him because they were so young. If they did, he hoped they would remember his warm embrace. Once, pain struck him so hard that he froze mid-sentence; that was the day I learned how to sit with someone and their pain, and be with them in silence.

Due to the nature of the work, the time I have with participants is short and finite. The connection between us, however, is deeply real. I carry them all with me still, and they make me look forward to waking up and meeting the next person.

Abbreviation Key

• Q= Question

• A= Answer