4 Recurring Risks to Consider in Early Phase CNS Research

April 3, 2019 Mike Cioffi

Early phase is the discrete period of time from first-in-man through proof-of-concept in which the challenges and risks of clinical research are amplified for both sponsors and volunteers. In the early phases of investigation, sponsors aim to determine the safety and efficacy of a new chemical entity. A critical question to ask in early phase development is: are we engaging the target? 

Early phase CNS research is plagued by a higher than average failure rate and complicated by a lack of discrete endpoints. As science evolves, hope arises. Today, we are able to improve our decision-making by increasing the number of objective data points collected in early phase research.  
 

Demanding Innovation

As the understanding of the central nervous system increases, so does the success in identifying a number of biomarkers and genetic components associated with neurological and psychiatric diseases. In an effort to increase the probability of success for investigational compounds, it is becoming more prevalent to incorporate alternative methods of identifying biological targets in order to more effectively screen new drugs. The use of functional magnetic resonance imaging (fMRI) in schizophrenia is giving companies the ability to assess target engagement and provide insights into the biological processes associated with the symptoms of schizophrenia. In fact, many biopharmaceutical sponsors try to de-risk their research portfolio by adding more biomarker data to early phase trials. If biomarkers indicate target engagement and hint toward efficacy, then companies will likely continue development.

Because decision-making in early phase clinical research is so important, many biopharma sponsors invite third-party experts to advise them on endpoint selection, trial methodology and design, and trial implementation strategies. WCG MedAvante-ProPhase helps sponsors bridge the gap between scientific integrity and operational excellence of their CNS research. We bring 25 years of experience in CNS and trusted data on 95% of all industry-sponsored protocols. Our CNS expertise, coupled with the scope of our data-driven insights, enables us to partner with biopharmaceutical sponsors who want to ensure that they are in the best possible position to succeed.

Having worked with every major sponsor in the industry, including emerging biopharma companies, we base our recommendations on unsurpassed clinical and scientific expertise, deep operational knowledge, and access to the highest volume of CNS-specific trial data in the industry. We’ve seen what works, and more importantly, what doesn’t.

So, what’s the takeaway?

Success means demanding innovation, especially in early phase CNS trials. Our industry is slow to change; we all know this. But we keep doing the same thing over and over again, expecting a different outcome. That’s the definition of insanity.

4 Recurring Risks to Consider in Early Phase CNS Research:

  1. Protocols are becoming more complex, and highly specialized. Many protocols now involve a genetic component, which introduces its own risks and necessitates certain considerations.
  2. Sponsors don’t often pay enough attention to site selection in CNS, especially in early phase. Often, they fail to consider which sites (if any) can actually execute the desired level of complexity.
  3. As opposed to late phase research, Phase I and II research is conducted in comparatively small populations. With smaller numbers, the quality of data becomes increasingly important.
  4. Identifying the perfect patient is like finding a needle in a haystack. Virtual trials are the wave of the future. This brings clinical research to the patient and provides greater access to untapped patient populations.

Confident Decision-Making 

When biopharma sponsors bring WCG MedAvante-ProPhase in to support early phase research, we help them to distinguish the “must-haves” from the “nice-to-haves’” in a given protocol design. This is particularly important because having the right requirements means finding the right patients to meet the proper enrollment criteria.

Based upon previous conversations with our CNS Scientific Leadership Team, we know that Principle Investigators (PI’s) often struggle with patient enrollment, particularly in CNS trials. By choosing the right sites and providing them with the right resources, we help clients to achieve enrollment 33% faster than average. This sets up sponsors for success from the very beginning of the clinical trial process.

Just like how a patient can go to a doctor for a second opinion, sponsors look to us for confident decision-making, especially our emerging biopharma clients, for whom we are an integral part of their process. By helping sponsors to accelerate the discovery of new treatments and therapies, we’re giving hope to the millions of patients and their families who are living with CNS disorders.

About the Author

Mike Cioffi

Mike Cioffi is the Senior Vice President, Clinical Solutions and Strategic Partnerships at WCG MedAvante-Prophase. Mr. Cioffi brings more than 20 years of pharmaceutical industry experience to his role leading MedAvante-ProPhase’s operational business units in support of project delivery and implementing global enterprise solutions that will drive continuous improvements in efficiency, output, and quality.

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