By Chris Molloy, CEO of RSA, the world’s largest Talent Consultancy dedicated to the life sciences industry.

It’s easy to be patient-focused when you’re developing drugs for a single patient population; anyone would agree with that. It sounds simple, but it would be underestimating the skills needed and the value that it adds to clinical development. The laser-like focus of the CMOs and CSOs developing Orphan drugs goes much further, putting them right out in front when it comes to living their passion and creating a corporate culture that enables successful outcomes.

The second of our new series of RSA Talent equity® Reports looks at the R&D and clinical development leadership of European and North American speciality pharma companies with Orphan drug indications. Orphan drugs are part of the market for speciality pharmaceuticals and refer to products used for diagnosis, prevention and treatment of rare diseases. Working with EvaluatePharma and hundreds of contacts around the world, we have used our research to better understand the profiles of this talented generation of scientific and clinical leaders.

Our analysis identifies the leading scientific and clinical talent in Europe and in the US. Through our study of the recent regulatory wins – Orphan drug designations and regulatory approvals – we have identified 10 Chief Medical Officers and five Chief Scientific Officers who drive their companies’ success in the Orphan drug field. To ensure that we only looked at the most focused of these companies, we excluded those working in oncology and haematology.

Driven by talent and a favourable environment

Orphan drugs are both the products of a changing pharma industry and are helping to lead that change in the hands of talented scientific and medical leadership. Worldwide Orphan drug sales are forecast to total $176bn (CAGR 2014 to 2020: 10.5%): almost double overall prescription market growth (excluding generics). The national Organisation for Rare Disorders (NORD) estimates that 30m Americans suffer from 7000 rare diseases and in 2014, spurred on by favourable legislation, 468 indication designations covering 373 drugs had been approved.

Tectonic shift

The ability to target focused, engaged patient populations and Key Opinion Leaders has had a tectonic effect on the industry. Biotechs that might once have licensed their assets out to big pharma are now choosing to “hold”. They’re able to do this because Orphan drugs are seen as lower risk and higher reward. Lower risk because clinical development costs less than for traditional drugs. Study populations are smaller and more rapidly recruited from more motivated populations and clinical investigators dedicated to helping their patients. Higher reward as Orphan drugs command higher prices when they launch, their cost per patient averaging six times that of non-Orphan drugs.

Given this high growth environment, it’s easy to see how talented individuals who have gained experience in mainstream pharma companies are attracted to work in this arena. No wonder that megapharma is adopting the ‘Orphan’ approach as it reassesses its viability and business model.

Zeroing in on the top talent

By studying 63 European and North American pharmaceutical and biotech companies, 1002 Orphan drug designations and 157 marketing approvals, we have arrived at our top 10 performing companies. We then looked at the CMOs who have driven focused clinical evaluation and, for the earlier stage leaders, the CSOs who have brought clinical candidates through to designation. What we found is shared characteristics amongst a group of highly focused CSOs with a passion for the diseases they’re targeting and a tight grip on the technologies they employ to craft their candidates. Coupled to these are the seasoned CMOs, most of them with megapharma experience and training, who demonstrate a passion for helping patients and a need to engage with them.

Innovative and pioneering

Working with small and disparate patient populations has meant that most of the featured CMOs have had to pioneer new approaches to clinical development, regulatory, approval and medical affairs. Most of the CSOs have succeeded in translating science into first-in-class therapies for previously untreatable rare diseases.

Team builders

Teambuilding is critical in these circumstances and many of our identified leaders have a remarkable track record of building successful clinical development or research teams across the product development life-cycle towards marketing approval.

Big pharma experience

This was a common factor, perhaps not surprisingly, for our CMOs. The experience gained in big pharma has given our young generation the breadth of skill to be focused when it matters and adaptable when new challenges arise.

Multiple therapeutic area expertise

For the CMOs we found that experience of working across many therapeutic areas at different stages of development gave them the required breadth of skills whereas many of the CSOs had gained their experience in early stage biotechs.

Scientific leadership

All of our featured CSOs have made remarkable contributions to their field of study; publishing extensively and with many patents to their names.

How can this report help the pharma and biotech industry?

Through this analysis, we have developed a high-definition understanding of what makes successful CMOs and CSOs in the field of Orphan drugs. This has enabled us to develop an insightful model of what it takes to be successful. When it comes to searching for the right executive for your firm, or to valuing an investment, nothing is more important.

We are first and foremost an advisory firm and our advice is based upon our in-depth knowledge of what ‘good’ looks like. The research we’re publishing in our report is the tip of the iceberg of our knowledge about this sector and the traits that make people successful within it.

If you want to know more about our analysis, or about how successful people drive successful companies, talk with us.

RSA’s Talent equity® report is the second of an ongoing, evidence-based series of reports about the life sciences sector.

CEO Talent Report
Orphan Pharma Report


Further reports will offer insight into many different sectors of the life sciences on an international basis.


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