National Alliance on Mental Illness Guest Post: Development of New Medications to Treat Serious Mental Illness

National Alliance on Mental Illness Guest Post: Development of New Medications to Treat Serious Mental Illness

10.10.12 | By Preet Bilinski

Michael J. Fitzpatrick is executive director at NAMI, the National Alliance on Mental Illness. Prior to taking that role in January of 2004, he served both as the director of NAMI's Policy Research Institute and as NAMI's national director of policy.

Since 1990, when Congress established the first full week of October as Mental Illness Awareness Week, mental health advocates have joined together to bring attention to this important issue. Today we hear from Michael Fitzpatrick, the Executive Director for the National Alliance on Mental Illness (NAMI). NAMI is the nation's largest grassroots mental health organization dedicated to building better lives for the millions of Americans affected by mental illness.

The past 20 years have witnessed enormous progress in development of medications to treat serious mental illnesses such as schizophrenia, bipolar disorder, major depression, borderline personality disorder and severe anxiety disorder. These medications - including atypical antipsychotics, SSRIs, SNRIs - have broad efficacy, proven benefits and are associated with improved delivery technologies. What's more, we now have a better understanding of how to use these medications along with non-medication treatments to improve overall health outcomes. Likewise, better imaging studies are facilitating developments of new treatments.

At the same time, we need to recognize the limitations of the medications we do have. The reality is that achieving a single medication "fix" for a complex disorder of brain and behavior such as schizophrenia is very difficult. Most major psychiatric disorders are multi-dimensional and have multiple causes which complicate finding precise medication treatments. New drugs are difficult and expensive to develop - with higher failure rates than other areas of medicine and an average of 2 years longer for clinical trials.

What is needed is a two track approach to drug development for serious mental illness:

  • Incremental improvement of existing therapeutic classes such as 2nd generation antipsychotics - new compounds with improved side effect profiles can be extremely valuable to patients unable to tolerate existing medications. These incremental improvements are essential if we are to improve adherence and advance recovery.
  • Breakthrough therapies that seek out entirely new targets and move us beyond the current paradigm of symptomatic treatment - We must have investment in research aimed at novel targets that have the potential to change the course of schizophrenia or bipolar disorder. To do this we have to reward investment in research that fail quickly and allow researchers to move on expeditiously to the next potential target.

We must also address current barriers in research on mental illness that create challenges for new drug development and venture capital investment. These include high placebo response and the difficulty of designing trials that can effectively blind side effects. More importantly, exclusion criteria in clinical trials too often keep patients with the most severe disorders from participating in research - medical co-morbidities, previous history of psychosis and suicide, adherence challenges, no active substance abuse, etc. We simply must find a way to integrate people with the most severe mental illnesses in the clinical trial enterprise. It is these patients that stand the most to gain from development of new therapies.

Despite these challenges, there is room for optimism. In June, President Obama signed into law the Food and Drug Administration Innovation and Safety Act. This important new law renews and expands the Prescription Drug User Fee Act (PDUFA) to ensure that FDA has the resources to quickly approve new therapies and make them available to patients. Among the key provisions in the new law that have the potential to advance research and development of new therapies:

  • New performance goals that will increase drug review efficiency and predictability and result in more consistent and transparent drug reviews - allowing the FDA to more clearly discern how efficacy and safety parameters are balanced as part of the FDA's risk-benefit analysis,
  • Enhanced Benefit Risk Assessment that should bring greater transparency to the benefit-risk assessments at the FDA,
  • Improvements on pharmacogenomics and biomarkers - The application of qualified biomarkers has enormous potential to accelerate drug development by helping to identify and predict which patient will respond to a particular medication,
  • Expedited Approval for Serious or Life Threatening Diseases or Conditions - NAMI is currently pressing the FDA to consider additional clarity with regard to defining surrogate endpoints for complex conditions such as schizophrenia where research has demonstrated irreversible morbidity related to acute episodes of psychosis, and
  • Breakthrough Therapies - NAMI is currently pressing the FDA to clarify what is necessary to demonstrate "substantial improvement" over existing therapies on clinically significant endpoints, such as substantial treatment effects observed early in clinical development. In moving forward, it will be important for this definition to integrate improvement over existing therapies not only with respect to clinical symptoms, but also serious side effects associated with existing therapies.

We have to make sure that this new law reaches its full potential for spurring investment in research and speeding access to new therapies as they are developed.

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