From Concept to Commercialization: Pushing the Envelope

Over the past 30 years, I’ve been fortunate to have a front-row seat to the transformation and growth in the biotech and pharmaceutical industries—first as a physician, then as an investor, and now as chairman and CEO of Marinus Pharmaceuticals, a commercial-stage pharmaceutical company with a focus on rare genetic epilepsies and refractory seizure disorders.

The rare disease space is a particularly challenging area for drug development: it is rapidly evolving, and (historically) decisions have been based on very limited clinical evidence.¹ On top of that, neurology and epilepsy can be especially difficult areas in which to find new therapies due to their complexities. But the need and urgency remain. Patients in this community are often faced with very limited options and time.

As the industry and treatment landscapes continue to rapidly evolve, it’s imperative we turn our heads away from more traditional approaches and think about tactics that topple the status quo, explore more revolutionary ways of working, and always understand and incorporate the patient voice.

Thinking outside the box

The most successful breakthroughs in medicine are often the result of thinking outside the box and challenging established norms. Pioneering first-in-class medicines requires a willingness to embrace new and bold ways of thinking, especially in the rare disease space where there are few solutions available to patients and unique challenges associated with drug development—such as regulatory hurdles, limited patient populations for clinical trials, and a lack of established treatment standards.

At Marinus, we set out to bring the first treatment option to patients with CDKL5 deficiency disorder (CDD) knowing we would need to push the boundaries of what we thought was possible and consider new approaches and perspectives. For example, we changed the size of our pivotal Phase III Marigold trial from 70 to 100 patients, which added nine months to the study; but it also gave us and FDA the data needed to move the medicine forward. In doing so, we successfully enrolled the largest double-blind, placebo-controlled clinical trial in CDD to date and received the first and only FDA approval in this indication.

Collaborating with patient advocacy groups to maximize impact

In addition to scientific innovation, successful drug development in the rare disease space requires a commitment to patient centricity and collaboration with patient communities. Engaging patients and their families as well as local, regional, and national patient advocacy organizations early and consistently throughout the drug development lifecycle allows researchers to gain a better understanding of the unmet medical needs and treatment preferences of those affected by rare diseases, leading to more effective therapies and improved outcomes.

Advocacy organizations at the local level are especially vital conduits between patients and companies that are looking to recruit for clinical trials. It’s essential to partner with these groups to help break down barriers for patients and give them a voice as well as instill trust that our intentions behind creating these solutions are in fact true.

Furthermore, patients and caregivers are accelerating disease understanding by closely aligning with the biopharma industry and providing their insights, which can drive real-world perspectives that optimize outcomes all the way from clinical trial design to commercial launch strategies.

Spearheading organizational transformation while bringing new medicines to market in this landscape takes agility, tenacity, and ingenuity. Companies and their leaders must step out of their comfort zone and embrace a multifaceted approach that combines scientific excellence, patient centricity, and a willingness to take calculated risks in pursuit of improving the lives of patients. There have been tremendous breakthroughs just in the past year alone, and I cannot wait to see how far we can go.

Reference

1. Kempf, L.; Goldsmith, J. C., Temple, R. Challenges of Developing and Conducting Clinical Trials in Rare Disorders. Am J Med Genet A. 2018. 176 (4), 773-783. https://pubmed.ncbi.nlm.nih.gov/28815894/