Evolving World of Pharma: Addressing real-world challenges in personalized medicine

Earlier this year I wrote about a new model for healthcare – one of ‘personalized medicine’ – in which all patients receive exactly the right diagnoses and treatments, personalized to them as individuals. So it was particularly interesting to hear, at the recent BioIntegrates conference in London, that the pharma biotech industry, at least, is now unified by the goal of "providing the right therapy to the right patient at the right time".

As a result, we now see biomarker-based approaches being integrated in more than 40% of new prospective drug development programmes. This concept of ‘biomarker-enabled drug discovery’ is helping drug developers to maximize efficiency and minimize risk, as it increases success rates for new drug approvals thanks to higher response rates in specific, selected populations.

Amgen, Gilead and GSK are said to be maximizing on the potential for biomarker-enabled drug discovery, and there is a strong focus on oncology, with Roche, Janssen and Merck & Co all showing strong pipelines of late stage candidates. Due to the natural heterogeneity of different types of cancer, which can be biomarked, the field of personalized oncology has seen the most rapid advancement.

As explained by Maria Antonietta Cerone, Precision Medicine Portfolio Manager at Cancer Research UK, the second-largest funder of cancer research in the world, “Instead of looking at one drug for one type of cancer patient, new molecular profiling technologies allow a granular patient stratification with subgroups of patients within one cancer type potentially benefiting from more targeted drugs.”

Jeff Settleman, PhD, Pfizer’s new Senior Vice President and Group Head of Oncology R&D, said in a recent press release, “There has never been a more exciting time in cancer research,” referring to Pfizer’s “highly promising pipeline that includes a variety of targeted therapies.”

So far so good – clearly, advances in genetics and genomics a playing a significant role in enabling the identification and implementation of biomarkers in medical research. However, how does this R&D translate to market?

In fact, the manufacturing of personalized treatments would be prohibitively expensive due to, for example, the necessarily small batch sizes, were it not for other technological advances that are permitting the growth of this sector in a more affordable manner. Reducing the cost of manufacturing is key for the success of personalized medicine.

According to Thomas Theelen, Business Development Manager at Univercells, commenting specifically for this column, “We have observed that personalized medicine, such as cancer vaccination, is enabled by platform technology developments. Several companies have invented biological platforms allowing rapid integration of cancer neoantigens in viral vectors. Given the relatively small batch sizes and the short timelines to obtain the personalized vaccine, a suitable small-scale equipment platform is needed. Univercells’ manufacturing technology allows [us] to mimic a standard vaccine manufacturing process at bench scale with sufficient yield for one patient. A high volumetric productivity is key since one facility should enable manufacturing personalized vaccines for multiple patients simultaneously. Therefore, the full process fits into a biosafety cabinet, which allows setting up multiple small units in a relatively small area. The downsizing combined with single use equipment also helps to contain costs.”

Another challenge is that the application of biomarker-driven therapies requires the assessment of biomarkers in clinical practice.

“There is a risk of identifying biomarkers that cannot be widely used in clinical practice due to the complex assays required for their identification,” explains Cancer Research UK’s Maria Antonietta Cerone. “Clinical trials allow testing of biomarkers in wider populations but often the assay used in those settings are either too complicated or too expensive to be easily translated into routine care. However, in several cases accompanying in vitro diagnostics test are also developed to facilitate implementation.”

The FDA has begun approving companion diagnostics that are packaged with an approved drug, and the number of these tests has increased dramatically in recent years, but the hope is that we will eventually have one affordable test for multiple drugs, rather than just one test for one drug. The development of effective, affordable (and reimbursable, where relevant) diagnostic tools is vital to the success of personalized medicine, and it represents an important growing area of medical technology.

It is also big business, as evidenced by recent news, for example, that a pioneering blood test developed by Johns Hopkins researchers in the US, that makes cancer detection a part of routine medical care has received the largest investment ever for a Johns Hopkins-licensed technology ($110 million). The new technology, called ‘CancerSEEK’, is designed to be used in routine medical care with the goal of identifying multiple cancer types at earlier stages, and it comes with an integrated service that provides support in result interpretation, confirmatory diagnostic testing, and guidance for additional clinical care.

In summary, personalized medicine has the potential to revolutionize the way in which healthcare is delivered to individuals across the world.

“If properly implemented and with enough research into the biology of treatments/diseases, precision medicine could drastically improve healthcare of future generations by allowing better patients stratification and identifying the subset of patients that will respond to specific drugs/drug combination and reducing negative side effects on those patients unlikely to respond to certain drugs,” says Cancer Research UK’s Maria Antonietta Cerone.

However, identifying risk factors, responders and developing treatments appropriate for those populations is only the first step in delivering this new model. In order to implement this new model, we need to overcome challenges such as those associated with new methods of manufacturing (on a small scale) and the practicalities of diagnosing and selecting patients in the clinical setting. These and other challenges associated with the advent of personalized medicine offer tremendous opportunities for companies working in this space.

Author biography

Sarah Harding, PhD

Sarah Harding worked as a medical writer and consultant in the pharmaceutical industry for 15 years, for the last 10 years of which she owned and ran her own medical communications agency that provided a range of services to blue-chip Pharma companies. In 2016, she began a new career in publishing as Editor of Speciality Chemicals Magazine, and has more recently taken up the role of Editorial Director at Chemicals Knowledge. She continues to also provide independent writing and consultancy services to the pharmaceutical and speciality chemicals industry.

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