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Last month, executives from leading international biopharma companies came together at the World Orphan Drug Congress 2020 to discuss how they are evaluating new opportunities in the rare disease space. The panel discussion was moderated by Premier Consulting’s Dan Duffy, and panelists included:

• Joana Granado, External Innovation Director, Actelion;
• Weiyong Sun, Senior Director, Specialty Medicine Search, Evaluation and Global Business Development, Daiichi Sankyo;
• Brian Bronk, Head of Business Development, MS/Neurology, Rare Blood Disorders, Rare Diseases and Innovative Technologies, Sanofi;
• Daniel Curran, Head, Rare Disease Therapeutic Area Unit, Takeda; and
• Thomas Kassberg, Chief Business Officer, Ultragenyx.

The panelists brought diverse perspectives on the future of orphan drug development, how their companies evaluate new potential products and partnering opportunities, and how they are working to address challenges like clinical study design and patient recruitment. Four common threads carried through the discussion.

The rare disease space is experiencing exciting and robust innovation.

The amount of interest and investment in rare diseases and orphan drugs has grown tremendously in recent years, leading to continuous innovation in a space that was once largely overlooked. In the past decade, new orphan drug legislation has encouraged pharma companies to invest in the space, and more and more clinical programs exploring various targets and modes of action have enabled a better understanding of the diseases themselves, clinical trial design for small patient populations, and biomarkers for diagnosis and monitoring of patients being treated. The result is new modalities and new technologies that enable a broad range of biotech and pharmaceutical companies to work together to bring therapies to rare disease patients.

What’s exciting is the potential for this wave of innovation to deliver treatment to some of the orphan diseases where currently there is nothing. —Joana Granado, Actelion

Biopharma companies seek products that will have the biggest impact on patients.

Biopharma companies look for partners who are driving medical innovation and who have a clear path to demonstrating the profile to bridge an unmet medical gap, especially in the rare disease space. Given the limited patient populations, the panelists agreed that a rare disease treatment candidate needs to:

• Be first-in-class or first-in-treatment,
• Fill a meaningful unmet medical need, and
• Modify the disease (not just treat the symptoms).

Often, only the first treatment available will make it to the market given the small patient populations, so it is important for developing partners to understand fully the natural history of disease progression, the indication, and the current standard of care. Only then can they truly know the impact that the product will have on the disease.

The patient voice is critical.

In the orphan drug space, direct involvement from patients and patient advocacy groups is essential to building a complete picture of the treatment needs for any given disease.

You can easily be misled by relying on what’s reported in the literature or talking to certain experts. I think you really need to get the feedback directly from patients and then incorporate that feedback as you think about how you’re going to develop the product to be successful. —Tom Kassberg, Ultragenyx

Takeda in particular has taken unprecedented steps to incorporate the patient perspective in the development process by including patients on its global project teams.

We can’t possibly know what these patients are experiencing. And while it’s important and we talk to the KOLs all the time, they’re not the ones living with the disease. —Dan Curran, Takeda

Collaboration is key.

Drug discovery and development is an immensely complex undertaking, and moving through the process alone can be extremely challenging. Partnerships between players that have complementary strengths, knowledge bases, and goals can be the keys to success. Often, that means combining the innovation and specialized knowledge of a small biotech with the resources and experience of a large pharmaceutical company.

It’s really about understanding the partner’s strengths and Daiichi Sankyo’s strengths, then we decide on responsibilities. I think we share a common goal, and that’s delivering drugs to the patient as quickly as possible. —Weiyong Sun, Daiichi Sankyo

All of us have a role and a place in this ecosystem. We want to connect patients across the globe to the right therapy, and that ultimately is what is important to Sanofi, to anyone who is in the business of discovering, developing, and commercializing therapies. —Brian Bronk, Sanofi

Author:

Taylor Anderson
Content Marketing Associate