The past 20 years have seen a transformation in the field of precision medicine. Completion of the first human genome project marked the beginning of a new wave of knowledge, producing exciting findings that improved our understanding of the genetic underpinnings of disease and the discovery of new drug targets. The project was a key driver of today's modernized clinical trials that take advantage of a massive amount of molecular data that was not previously available. This has served as a blueprint for precision medicine.
Precision medicine has not only impacted drug development, but also drug discovery and disease diagnosis, allowing scientists to use molecular characteristics to define diseases and moving diagnosis away from clinical signs and symptoms.
Gaining new scientific insights through data analysis
The paradigm is clearly shifting away from one-size-fits-all for a given disease towards a much deeper understanding of the biology, disease and genome. Understanding the molecular basis of disease allows us to seamlessly repurpose a drug from treating one disease to treating multiple diseases, thereby maximizing its benefit.
The pace of innovation is accelerating. We are starting to see the integration of different types of biomarkers into clinical development to understand variation in clinical response. The ability to overlay biomarker data onto the clinical trial data allows scientists to gain important insights as to why patients have responded in different ways. The global regulatory authorities are strong advocates of precision medicine and are increasingly asking companies to use biomarker data to explain variation in clinical response.
For biotech companies, this new landscape holds tremendous promise for innovation – for new drug targets, newly designed drugs, new technologies for drug delivery, and engineered cell or gene therapies. Biotech companies often have the ability to be agile and flexible, at times giving them an advantage over their large-pharma counterparts. Indeed, the most critical and innovative new therapies for rare and untreated diseases are in the biotech pipeline.
One of the key advantages of precision medicine-guided drug development is that drug developers can generate critical data to demonstrate to a payer that the medicine will work well for a specified group of patients. This will help secure reimbursement and bring not only an important new medicine to patients, but value to the company.
Overcoming perceived hurdles
Some biotechs are still shying away from precision medicine approaches. One commonly cited reason is the perceived cost. For a small biotech company, the collection of biomarker samples in a clinical trial may seem an unnecessary expense. However, there is a very strong cost: benefit ratio, because the samples can be banked and frozen as an insurance policy. That way, if there are questions, the samples are at hand for experimentation and data generation that can help interpret the trial results, guiding further trial design or even rescuing the drug. Of equal concern is the lack of in-house translational medicine expertise. Indeed, generating large sets of biomarker data requires experts who can handle, organize and interpret them within the context of disease-specific knowledge. One solution is to partner with those who possess the skilled or specialized workforce necessary for precision medicine trial development and execution.
Understandably, some biotech companies are focused on reaching their next milestone, whether that be funding for their next clinical trial or selling the rights to their investigational medicine to another company. Yet in a broader sense, from our experience working with biotech companies, they also face the common challenge of effectively and efficiently translating their science, technology and potentially breakthrough drug products into clinical development.
Taking a strategic approach
That said, biotech companies are best placed to take advantage of opportunities in precision medicine when they explore this approach from the outset. Parexel Biotech can partner with companies to develop a strategy that encompasses science, medical need, patient perspectives, regulatory requirements and market access – including reimbursement and launch. To achieve this, we ask questions, such as:
- What is known about genetic variation or biomarkers in the disease, drug target and target pathway? Is there a good understanding of the target indication and target patient population? What biomarker-driven patient enrichment strategy should be used? What is needed to establish the biomarker assay validity and clinical predictability?
- How can the development program encompass the interests of key stakeholders, investors, regulators, health providers and payers? What evidence will be required for regulatory approval? What data will payers require to make reimbursement decisions? How can timelines be accelerated, risks reduced and resources best utilized?
- When assigning patients to treatment cohorts, is it ethically appropriate to randomize them to a treatment that is unlikely to be beneficial? How can adaptive trial designs be used to maximize patient benefit, focusing on treatment regimens that are more likely to be effective?
Clearly, opportunities abound for biotech companies to employ precision medicines in their quest to bring innovative new treatments to patients. At Parexel Biotech, our operating model provides dedicated leadership and resources, carefully selected and organized to provide the essential expertise biotech companies need to succeed. With strategic guidance based on years of industry experience, we can enhance biotech companies' capabilities through our lean operational structure and flexible processes.
As one of the world's top CROs, we offer services ranging from development strategy and meeting clinical development timelines to optimizing product and pipeline value and navigating competitive, regulatory and reimbursement landscapes. Designated centers of excellence in a wide range of therapeutic areas include medical, biomarker, regulatory and biostatistics experts to provide early-advisory consulting for biotech companies as part of their drug development program. These encompass precision medicine as well as the use of adaptive trial designs, real-world data - and more.