Beyond borders: biotechnology industry report 2013

Patient-centric innovation

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Patient-centric innovation, by Husseini Manji, MD, Global Therapeutic Head for Neuroscience, Janssen Research & Development 

The challenge of brain diseases

Husseini Manji, MD, Global Therapeutic Head for Neuroscience, Janssen Research & Development, A Johnson & Johnson Pharmaceutical Company

Brain disorders are among the most devastating ailments affecting society — something we are increasingly starting to appreciate. On one end of the age spectrum, neurodegenerative diseases such as Alzheimer’s diminish and ultimately destroy lives of older people, disrupt families and threaten to bankrupt health care systems.

In our rapidly aging population, the number of Alzheimer’s patients is predicted to triple or quadruple by 2050, and costs could increase to over US$1 trillion in the US alone. Today, Alzheimer’s is 100% incurable, 100% fatal and 100% of patients require some sort of full-time care. To change that, we need to move from just treating symptoms to actually slowing disease progression.

At the other end of the age spectrum are serious mental illnesses. These diseases are very disabling and costly because they often emerge early in life — among adolescents or young adults — but are lifelong. They have a hugely disproportionate impact on individuals’ productivity, which is increasingly important in today’s knowledge-driven economy.

Last year, a World Economic Forum report found that mental illnesses are projected to cost society more than cancer, diabetes and chronic respiratory diseases combined. Severe depression is predicted to be the number one cause of disability worldwide by the year 2030. In the US, it’s estimated that there are more than 35,000 deaths a year from suicide, which means that there are only three forms of cancer which have a higher annual death rate.

Tackling these diseases presents some significant challenges. The brain is both the most complex and the most inaccessible human organ. As a result, the understanding of molecular brain networks is considered to be more challenging than what’s required for other diseases.

Our diagnostic classification is often based on signs and symptoms that aren’t directly linked to a molecular path or physiology. For example, it’s much easier to induce cancer in a mouse than to induce psychotic symptoms or the learning and memory problems of Alzheimer’s. Our translational models need deeper scientific exploration and greater refinement.

As with a lot of R&D, neuroscience is highly specialized and can be siloed, and it will be critical to collaborate broadly. We need good biomarkers to help us measure the progression of Alzheimer’s years before it is manifested in visible symptoms — but this is not something any individual entity needs to own.

It’s therefore gratifying to see people collaborating in precompetitive spaces. For instance, the Alzheimer’s Disease Neuroimaging Initiative (ADNI) brings together the NIH, FDA and 15 or so companies along with various university experts to identify biomarkers that predict the progression of Alzheimer’s.

Patient-centric innovation

Payers and society at large are moving toward rewarding value and improved outcomes, which will encourage the adoption of more holistic solutions. The field of neuroscience could benefit tremendously from this, because brain disorders are complex and affect everything from genes to behavior to relationships, making this an area where we are more likely to need multimodal interventions that go “beyond the pill.”

To succeed in this endeavor, we have to think of innovation not just in terms of scientific breakthroughs but also in terms of patient centric innovation. At Janssen, we have realigned to create end-to-end therapeutic areas, encompassing the entire cycle of care. Even our discovery scientists need to be aware of what payers are going to reward. We try to project how payers will regard a future product, even if it’s in the earliest phases of discovery.

This drives us to concentrate on genuine unmet needs. To the extent we focus on things that are already partially addressed, we have to know from the start how we might identify the subpopulation in which a new treatment will be superior — making biomarkers inevitable. Very early in clinical trials — once we’ve identified dose and possible side effects — we bring in an active comparator to see how our treatment compares with the standard of care in a real-world setting.

This also involves collecting data about real-world outcomes. Increasingly, society will want evidence not just of improved symptoms at three weeks, but of getting people back to work faster or improving their ability to engage in physical activities or making them less dependent on caregivers.

So far, it hasn’t always been easy to get payers to focus on longer term, real-world measures. Organizations concentrate on the budgets for which they are responsible, and gravitate to things such as minimizing short-term hospitalization costs. In the fragmented US market, broader, longer-term measures get less traction than in European countries, where interests are typically better aligned.

But things are changing, and we are going to see things shift in this direction in the US as well. New mobile health technologies are streaming behavioral and physiological data on an unprecedented scale and will play a huge role in this outcomes-driven future. In neuroscience, this is very powerful, since it can help identify when someone is about to have a relapse or stroke. After all, patients spend only a small amount of time in a physician’s office — they live in the real world.

We are working on technologies that would enable the integration and interpretation of different streams of data from devices and technologies, to help us move from a paradigm of “diagnose and treat” to one of “predict and preempt.”

For instance, we are developing technologies to improve adherence. Schizophrenia is one example where going off your medication for as little as 10 days dramatically increases the likelihood of re-hospitalization. The problem is compounded by the fact that some individuals with these disorders often don’t have full insight into their illness. So we’ve developed and now market a one-month injection for schizophrenia medication and are currently developing a three-month formulation. Moving to only four injections a year could dramatically reduce relapses caused by non-adherence.

To bring all of these solutions together, we are experimenting with integrated care models. Once again, Europe has led the way. In Germany, for instance, we have a holistic care model for schizophrenic patients. Patients receive whatever care the physician deems appropriate, regardless of whether it involves our medication or a competitor’s drug. The integrated care provided includes education to facilitate adherence, family counseling, rehabilitation services and more. We have similar programs in the UK as well.

Over time, we are going to have to work together to marry these new technologies with integrated care models and streaming data to inform decision-making in real time. That’s not to say that technological advances at the molecular and cellular level aren’t important. They’re exceedingly important. But we need to think of innovation more broadly.