2015 Approvals Herald Rapid Progress, Growing Diversity in Precision Medicine

The Personalized Medicine Coalition (PMC) reports that 13 of 45 new molecular entities or therapeutic biologics approved by FDA in 2015, or 28%, were personalized medicines. The number of approved personalized (precision) medicines increased from 9 in 2014. While completing the transition to precision medicine will take years and require addressing major challenges, the momentum is undeniable and building.

Oncology and Rare Indications Lead the Way

As expected, oncology indications and rare diseases dominated the list of newly approved precision medicines. However, there is a temptation to interpret the dominance of these two categories in the light of traditional broad indications that predate the era of precision medicine and contradict the realities that drive it. Oncology and rare disease are categories that encompass many indications. Cancer is not one disease but many, with variants within each indication that profoundly effect therapeutic strategies and patient response.
Beyond oncology, every therapeutic area includes its own variety of rare indications that affect health and quality of life in many ways depending on the biological processes compromised. In short, there is nothing inherent in oncology indications and rare diseases that makes them more suitable targets for development of precision medicines. The prominence of these categories in precision medicine simply reflects greater understanding of disease processes and greater progress in identification of biomarkers and development of related diagnostics.

Precision Medicine Extends Its Reach Across Therapeutic Areas

Precision Medicines by Therapeutic Area 2015Viewing precision medicines approved in 2015 through the lens of traditional therapeutic areas reveals greater diversity than many recognize. Of the 13 approved new precision medicines, 5 were in oncology, distributed among four indications (NSCLC, melanoma, CRC, breast cancer). However, the remaining 8 precision medicines were distributed among neuroscience indications (schizophrenia, major depressive disorder) and indications in three other therapeutic areas: respiratory (asthma, cystic fibrosis), endocrine & metabolism (hypercholesterolemia, bile acid synthesis disorders) and infectious-disease (hepatitis C).
The therapeutic distribution of newly approved precision medicines reminds us that once disease processes are understood and biomarkers identified, indications in all therapeutic areas make equally good targets for precision development. Advancing the state of knowledge across the therapeutic spectrum will increase the scope of precision medicine accordingly.

Prioritizing Precision Medicines for Major Chronic Diseases

The Precision Medicine Initiative’s Million Person Cohort as well as disease advocacy organizations should drive much more vigorous activity in major chronic diseases such as diabetes mellitus, arthritis and heart disease in their many distinct forms. Inevitably, we will learn that these major chronic diseases are collections of more numerous distinct indications than are currently recognized. In addition, each indication will require its own precision medicines. Researchers have tools of unprecedented power but success in development of precision medicines will require identifying and understanding a much greater range of disorders and understanding disease mechanisms in greater depth. As the Precision Medicine Coalition states, “Substantial progress has been made, but there is still much work to do.” Precision medicine is going to be transformative but it is not going to be easy.
We look forward to great progress in precision medicine in 2016 in oncology indications and rare diseases. But let us also make 2016 the year in which we commit ourselves to achieving substantial progress in development of precision medicines effective against the other major chronic diseases that, like cancer, are the leading causes of death and disability in the United States and the world.

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