Interview with Anita Nelsen of PAREXEL

Anita Nelsen has more than 20 years of experience in the pharmaceutical industry and 29 years of experience in pharmacogenetic, human and molecular genetics research in roles including bench scientist, manager and function leader. At PAREXEL, she leads a global team of scientists who are recognized thought leaders and influencers in quantitative clinical pharmacology, modeling and simulation, biomarkers and genomic-based drug development to help clients strategically leverage and seamlessly integrate technologies across all phases of development. Read her full bio.

Interview with Anita Nelsen of PAREXEL

Q: There are various new, emerging technologies that bring us closer towards a cure for life-threatening disorders such as cancer, HIV, or Huntington’s disease. Prominent examples include the popular gene editing tool CRISPR or new and improved cell and gene therapies. By when can we expect these new technologies being part of routine clinical care?

A: Today’s emerging technologies are making the promise of individualized treatment a reality. We are able to edit genes, customize treatments down to a molecular level, and more. These technologies are exciting and hold a lot of potential, but they must be brought to clinical care in a compliant manner with well-designed and ethical clinical trials that showcase patient outcomes. With this testing, there is also a need for education so providers and consumers understand the impact and power of the technology, choose to participate in the clinical trials, and ultimately make informed treatment decisions.

Many precision medicine clinical trials, including those that leverage cell and gene therapies, are in early phases, so even with emerging and new regulatory guidance and pathways, it will likely take some time for these types of treatments to become mainstream for clinical care. Further, pathways to reimbursement also need to be established for these novel therapies. That said, these types of trials are promising – recent research conducted by The Economist Intelligence Unit (EIU) and commissioned by PAREXEL found that drugs developed using precision medicine trials were 10 percentage points more likely to be launched.

Q: What other emerging technologies will have a significant impact on patient care in the near and far future?

A: In the near and far future, we see patient data and analytics continuing to have a significant impact on care. The information, quality and consistency of it and how it’s analyzed are the keys to unlocking true precision medicine where patients receive customized treatments based on their genetic makeup, lifestyle, and other factors. Although patient data has been available for some time, the types of data we are accessing are getting more complex. In the future physicians may need to consider an individual’s genome, their immune biomarkers, and even their microbiome when helping to assess treatment options or recommend lifestyle changes. Not only are we collecting new types of data but there are still new ways to track it (e.g. wearables and sensors, smartphones, direct to consumer tests); however, there are challenges to understanding, aggregating and analyzing the quality of this data. If successful, the data holds the potential of providing powerful new algorithms and machine learning to predict outcomes and uncover new treatment avenues.

Additionally, we continue to see growth in adoption of patient-centric trials, which are designed specifically around patients’ needs to make it as easy and as convenient as possible to participate in research. There is more direct involvement of patients and/or their caregivers and/or patient advocacy groups in the initial design of studies. We’re also seeing increasing use of technology, such as wearable sensors, to enable the capture of clinical data in the home setting.

These advances are likely to improve the delivery of new drugs to market. Recent research findings from the EIU showed that patient-centric trials enrolled patients faster and are more likely to succeed, taking an average of four months to enroll 100 patients in patient-centric trials versus seven months for non-patient-centric trials. These types of trials also had a higher probability of success progressing from Phase II and III to market launch.

Q: In order to maximize the potential of the aforementioned technologies in the clinic, what strategies need to be adapted?

A: There are three main things that need to happen to maximize the potential of these technologies and ultimately precision medicines in the clinic.

  • First, payers need to cover these medicines. To do so, they need new and different models to assess these therapies versus therapies developed via traditional drug development paradigms.  They must understand the target patient profile, potential benefits, and risk of harm associated with these novel approaches as well as the cost impacts. Some have suggested that models for reimbursement need to be more innovative and there may be cause for risk-sharing models with pharma sponsors. Real world data and advanced analytics will likely play a critical role in these helping pharma convince payers of that targeted patient groups that are most likely to benefit from precision medicines and therapies.
  • Second, in the clinic, healthcare providers (HCPs) need to be equipped to analyze and interpret the data and translate it into treatment decisions that lead to better patient outcomes. They need the tools and infrastructure to obtain, analyze, and translate the data into treatment decisions.  At some level, they need to understand the data science and technology.
  • Finally, we need to translate the information to the patient in a way that is understandable and contributes to patients making informed decisions about testing, treatment, and lifestyle.
Q: What are some of the major challenges that need to be overcome before we can see widespread applications across the clinic?

A: Data and information sharing are major challenges that many in the industry are currently working to overcome. We have seen many potential solutions such as electronic health records, patient wearables, etc. to gather and share data but there are still roadblocks ahead. It’s going to take continual out-of-the-box thinking and communication to ensure that providers, trialists, and other industry stakeholders have the full picture of the data, how it better helps us understand diseases, and how to treat them.

Another challenge that has surfaced with the rise of precision medicine: human sample management. With an increased amount of these trials, scientists need certain biological resources to support discovery and development of precision medicine therapies, making biobanking and exploratory analyses a routine part of research. However, there are perceived challenges that come along with biobanking in global clinical trials, such as varied regulations and cultural perceptions, concerns about cost, potential delays in clinical trial recruitment and conduct, and ownership of and access to data and privacy risks for participants. All of these can be overcome but they are real challenges.

Q: How can and should the community work together to get those various technologies safely into the clinic?

A: Working together is critical and more and more I think the precision medicine community is working together. It takes a long time to drive culture change necessary to move from traditional, highly secretive drug development models to collaborative or cooperative models where data sharing fuels understanding of disease and identification of new drug targets to companies that work collaboratively to execute novel trial designs that can assess the efficacy of multiple drugs in parallel or develop a diagnostic test to use with multiple medicines. Early engagement with regulators and payers is encouraged and even expected to help pave the way for smooth review. We have finally realized that we all face the same challenges—having the tools and experience to make sense of the complicated new data types and drive decisions. We can solve for these challenges more efficiently if we work as a team.

Q: What solution is your organization providing to address what need in precision medicine?

A: PAREXEL is working closely with sponsors to implement innovative clinical trial designs leveraging these emerging technologies. Our experts are partnering with clients across the drug and diagnostic development continuum to design and implement biomarker and genomic strategies that support their assets from pre-clinical to post-market. Our precision medicine experts provide commercial, regulatory, medical, and scientific consultation to help sponsors understand the therapeutic and commercial benefit to leveraging diagnostic tools to make sure the right patients receive the right treatments at the right time. The Company is also hosting a series of closed session workshops with experts across the healthcare industry to identify barriers as well as enablers of adoption that can improve the future of drug development, related to precision medicine and other innovations.

Q: Is there anything you would like to share with the PMWC audience?

A: As a community, we have worked hard to demonstrate the value proposition of precision medicine strategies. As recognition of this value increases we must now demonstrate that we can overcome the challenges that come with the approach, and that we can build the experienced workforce, the data analytic technologies, and the collaborative partnerships to move from development to treatment and potentially cures.