Interview: Partnering from the pharma perspective

In Partnering & Investment by Freya Smale


As part of a series of interviews conducted with leading industry experts in the run up to the World Orphan Drug Congress in Geneva, Total Orphan Drugs was delighted to sit down with Carrie Cook, Head of Licensing, Neurodegenerative Diseases, Global Business Development, EMD Serono, Inc. Total Orphan Drugs would like to take this opportunity to thank Carrie Cook for speaking to us.

Considering a pharma partnership in the future? Start preparing early, according to Carrie Cook, head of Licensing Neurodegenerative Diseases, Global Business Development, EMD Serono, Inc.

To get an orphan drug all the way to patients, you may seek to partner with a pharma for development, regulatory and commercial expertise. This may mean dovetailing differing priorities.

Carrie Cook conducts global business development and licensing for neurodegenerative diseases for EMD Serono. She looks for compounds that fundamentally address an unmet need and prefers compounds that have generated some clinical data. “The more data the better, obviously.” said Cook. She would consider a compound that is not yet at proof of concept but it must hold exceptional promise..

“There is a broad spectrum of orphan diseases.” Cook said. “We need to look at the risk associated with development as well as regulatory pathways—whether there is an existing pathway or some guidance behind that to help limit the overall development risk.” Development hurdles like understanding the underlying disease process, recruiting patients for clinical trials, defining inclusion and exclusion criteria —all of these are unique challenges for rare diseases.

Cook sources promising compounds via personal contacts and partnering and disease area conferences or prospects contact her directly. She and colleagues interact with key opinion leaders (KOLs), academic institutions or biotech companies. EMD Serono has advisory boards to discuss specific diseases, overall standard of care, unmet needs, and emerging therapy trends.

According to Cook, recently more companies have started delving into orphan drugs so the space is becoming more and more competitive. “In terms of the stage where the compounds should be, the later in development the better.” said Cook. “But again for example, in ALS, we’ve seen several late stage failures. If you look at it at the disease level it is going to be a lot of early stage investment at this point.”

According to Cook, it is difficult for biotechs to negotiate to retain commercial rights to compounds when there is a very centralized customer base. “There are a small number of centers, for example, that you as a company need to focus on in order to market the compound.” said Cook. “And when you are talking about sharing that it becomes very difficult.. For a pharmaceutical company partnering with a smaller biotech, both companies have to be very creative in how they come up with a solution that works for both.”

According to Cook, a model that works for both companies is where the compound addresses disease mechanisms that could affect multiple rare diseases. Normally, with just one disease one company would do the development and commercialization on its own. Where there are complementary interests and multiple potential indications, however, there is room for creativity on how to develop and commercialize a drug together.

1. Address risks early.

A biotech can raise its partnership attractiveness by de-risking its compounds as much as possible. “We do our diligence when, we see something promising.” said Cook. “It is important to have a realistic perspective on what the risks are for that compound, to be transparent about that, what makes it more challenging for us to move forward is when a company ignores those risks. We would like to see that the company has at least considered how to address them, has done the additional work in animal models or has identified or developed a plan to identify specific biomarkers.”

2. Clarify as much ambiguity as possible.

Demonstrate your overall understanding of development for a specific disease, having tested it in the right models, if animal models exist, and having sought external expertise and guidance such as KOL input and, regulatory advice. Consider how likely the regulatory agencies are to approve the compound based on the planned development path and how willing payers would be to pay for this solution

3. Think early in the process about how to expand indications.

There are different options around collaboration from the development and commercialization perspective if one thinks beyond just one rare disease. I think the earlier the biotech company can incorporate commercial considerations the better.”

About the Company

EMD Serono, Inc., a subsidiary of Merck KGaA, Darmstadt, Germany, has strong market positions in neurodegenerative diseases, endocrinology and in reproductive health. In addition, EMD Serono is growing its expertise and presence in the area of oncology, with more than 15 projects currently in development. EMD Serono’s US footprint continues to grow, with approximately 1,000 employees around the country and commercial, clinical and research operations in the company’s home state of Massachusetts.

image_thumb[4]Special thanks go to Wendy Wolfson who conducted the interview on behalf of Total Orphan Drugs.

Wendy Wolfson covers innovations at the intersection of medicine, science and healthcare as a columnist for Chemistry & Biology, a publication of Cell Press. She has contributed to magazines including Science, Nature Biotechnology, the Lancet, and Red Herring. Her work can be found at and she can be contacted at

Read more interviews in this orphan drug series
Partnering from the pharma perspective
How to deliver value to patients
Perspective from patient advocates in Sweden
Interview: Surmounting barriers to patient access
Unique opportunities to develop cures for orphan diseases in the Middle East shouldn’t be overlooked
Where do the current and future challenges lie in bringing new orphan drugs to market?
Bringing Biotech Spirit to a Non-Profit Environment
Paving the way for a whole new medical paradigm
Tailoring your reimbursement strategy, step by step, country by country
An Interview With Pfizer on the Orphan Drug Industry: Part I
An Interview With Pfizer on the Orphan Drug Industry: Part II
An Interview with the Chair of the IRDiRC
An Interview with an Orphan Drug Payer