What Orphan Drugmakers Seek From CROs

By Roger Garceau, MD, is senior VP and CMO, NPS Pharmaceuticals

Regardless of size, CROs with the right mindset can flourish in specialty niches.

Many small and midsize CROs have struggled in the past few years as pharmaceutical companies slashed their research budgets. There’s no question size and scale are a big plus for a CRO. However, smaller contract organizations that follow best practices and pay close heed to clients’ needs can also be attractive partners in the fast-growing area of orphan drugs.

To seize the opportunities, it helps to understand the challenges orphan drugmakers face. Many of these players are themselves small companies working on just one or two products — each of them a one-off indication. In the case of NPS, which develops orphan therapies for GI and endocrine disorders, virtually all aspects of our preclinical and clinical studies must be outsourced, and finding the right partner is paramount.

Let’s begin with the assets a large CRO brings to the table. First, they have breadth of experience, from writing protocols and handling toxicology in multiple animal models all the way to putting together advisory panels. Greater scale also means large players may be better prepared in a crisis. What happens, for example, if one of the CRO’s trusted field monitors on a project quits or is taken out of commission mid-study? This can be catastrophic for the sponsor — especially for an orphan drugmaker that has groomed relationships with an academic research center, or a particular scientist or thought leader. A large CRO with a half dozen projects at the same research center is better positioned to substitute a competent monitor and transfer institutional knowledge.

Small Can Be Beautiful
Acknowledging these large-company advantages, there are still aspects in which a small or midsize CRO has a fair chance, as long as it can provide certain assurances to the sponsor. For one thing, the CRO may have focused, specialized experience in the area being studied. In addition, the smaller client will have more clout with a CRO of comparable size than with a giant whose corporate culture closely mirrors that of Big Pharma. Such a client is bound to receive more personalized attention from a like-sized CRO. 

Staff turnover also will probably be lower at a small CRO, sparing the client unnecessary interruptions and obstacles. By the same token, if a big contract organization is accustomed to dealing with Pfizer, Merck, or Novartis, it may not share the small drugmaker’s sensitivity to time and budget constraints. A small to midsize CRO is more likely to recognize the immutable truth that time is money. 

In summary, I see the following six core competencies for a CRO targeting the orphan space:

Track record. Even though each orphan drug is unique, clients want evidence that you have successfully tackled compounds or proteins resembling theirs. CROs should obtain permission from former clients to share success stories.

Performance metrics. For smaller specialty pharma, cost is key. The CRO should assign a project manager up front who can help the client keep costs under control in the face of unexpected delays or setbacks in the study.

Academic relationships. The CRO should show that it has experience in the timely resolution of contractual issues common in academic settings, e.g., disputes over intellectual property and publishing rights. Also, the CRO should prove that it can extract rigorous, consistent work from thought leaders who may have little experience with FDA registration studies.

Regulatory competence. As far as the FDA is concerned, the CRO is an extension of the sponsor. That means the client must own any mistakes the CRO makes.  Every step of the process must be completely transparent to the client.

Honesty. Too many CROs roll out the A-team just once, during the initial meeting. Few deceits are worse than bait-and-switch. If you promise a client that you’ll assign a particular study manager, don’t back away from the commitment. Similarly, changing field monitors mid-study needs to be minimized.

Crisis Control. In the negotiating phase, the client must be assured that the CRO has fail-safes and backup plans if key personnel assigned to the project are suddenly put out of commission. Understand and compensate for liabilities related to your size.

Roger Garceau, MD, is senior VP and CMO of NPS Pharmaceuticals. He has more than 20 years of experience in the pharmaceutical industry.