Policy Issues In The Development Of Personalized Medicine In Oncology

One of the challenges in treating cancer is the disease's complexity and variation among patients. Cancer manifests differently in each patient, so treatments that are effective in one patient may not be effective in another. As cancer care becomes more personalized, subpopulations of individuals will be given preventive or therapeutic interventions based on their susceptibility to a particular disease or their predicted response to a specific treatment. However, before the use of personalized cancer care can reach its full potential, the health care system must resolve a number of technological, regulatory, and reimbursement issues. To explore these policy challenges, the National Cancer Policy Forum held the workshop Policy Issues in the Development of Personalized Medicine in Oncology in June 2009. Experts provided presentations on the current state of personalized medicine technology, as well as issues in the validation of, regulation of, and reimbursement for the predictive tests that underpin personalized medicine. Participants discussed the obstacles and possible solutions to further developing and using personalized medicine technologies. This document summarizes the workshop.

A long-held goal in oncology has been to develop therapies that target the specific abnormalities in each patient's cancer rather than simply treating cancers based on the tissue of origin. In the past decade, advances in technology have enabled researchers to relatively quickly and inexpensively determine, in minute detail, the genetic makeup of tumors. Although relatively few targeted cancer therapies are currently available in the clinic and it is not yet clear whether all cancers are driven by genetic changes that can be targeted, there is widespread optimism in the cancer community that this new ability to assess the genetic abnormalities in tumors will ultimately lead to better cancer treatments and improved patient outcomes. Policy Issues in the Development and Adoption of Biomarkers for Molecularly Targeted Cancer Therapies is the summary of a workshop convened in November 2014 by the Institute of Medicine's National Cancer Policy Forum to discuss recent trends in the development and implementation of molecularly targeted cancer therapies and explore potential policy actions to address specific challenges. This report highlights the presentations and discussions at the workshop.

Nowhere is the explosion in comprehensive genomic testing more evident than in oncology. Multiple consensus guidelines now recommend molecular testing as the standard of care for most metastatic tumors. To aid in the advancement of this rapidly changing field, we intend this Special Issue of JPM to focus on technical developments in the genomic profiling of cancer, detail promising somatic alterations that either are, or have a high likelihood of being, relevant in the near future, and to address issues related to the pricing and value of these tests. The last few years have seen the cost of molecular testing decrease by orders of magnitude. In 2018, we saw the first "site-agnostic" drug approvals in cancer (for microsatellite unstable cancer (PD-1 inhibitors) and NTRK-fusions (TRK inhibitors)). Research on targetable mutations, determination of genetic "signatures" that can use multiple individual genes/pathways, development of targeted therapy, and insight into the value of new technology remains at the cutting edge of research in this field. We are soliciting papers that present new technologies to assess predictive biomarkers in cancer, original research (pre-clinical or clinical) that demonstrates promise for particular targeted therapies in cancer, and articles that explore the clinical and financial impacts of this paradigmatic shift in cancer diagnostics and treatment.

Personalized and precision medicine (PPM)—the targeting of therapies according to an individual’s genetic, environmental, or lifestyle characteristics—is becoming an increasingly important approach in health care treatment and prevention. The advancement of PPM is a challenge in traditional clinical, reimbursement, and regulatory landscapes because it is costly to develop and introduces a wide range of scientific, clinical, ethical, and socioeconomic issues. PPM raises a multitude of economic issues, including how information on accurate diagnosis and treatment success will be disseminated and who will bear the cost; changes to physician training to incorporate genetics, probability and statistics, and economic considerations; questions about whether the benefits of PPM will be confined to developed countries or will diffuse to emerging economies with less developed health care systems; the effects of patient heterogeneity on cost-effectiveness analysis; and opportunities for PPM’s growth beyond treatment of acute illness, such as prevention and reversal of chronic conditions. This volume explores the intersection of the scientific, clinical, and economic factors affecting the development of PPM, including its effects on the drug pipeline, on reimbursement of PPM diagnostics and treatments, and on funding of the requisite underlying research; and it examines recent empirical applications of PPM.

This, the first of two volumes on personalized medicine in lung cancer, touches on the core issues related to the understanding of lung cancer—statistics and epidemiology of lung cancer—along with the incidence of lung cancer in non-smokers. A major focus of this volume is the state of current therapies against lung cancer—immune, targeted therapies against EGFR TKIs, KRAS, ALK, angiogenesis; the associated challenges, especially resistance mechanisms; and recent progress in targeted drug development based on metal chemistry. Chapters are written by some of the leading experts in the field, who provide a better understanding of lung cancer, the factors that make it lethal, and current research focused on developing personalized treatment plans. With a unique mix of topics, this volume summarizes the current state-of-knowledge on lung cancer and the available therapies.

Progress and Challenges in Precision Medicine presents an insightful overview to the myriad factors of personalized and precision medicine. The availability of the human genome, large amounts of data on individual genetic variations, environmental interactions, influence of lifestyle, and cutting-edge tools and technologies for big-data analysis have led to the age of personalized and precision medicine. Bringing together a global range of experts on precision medicine, this book collects previously scattered information into one concise volume which covers the most important developments so far in precision medicine and also suggests the most likely avenues for future development. The book includes clinical information, informatics, public policy implications, and information on case studies. It is a useful reference and background work for students, researchers, and clinicians working in the biomedical and medical fields, as well as policymakers in the health sciences. Provides an overview of the growing field of precision medicine Contains chapters from geographically diverse experts in their field Explores important aspects of precision medicine, including applications, ethics, and development

For this volume of Advances in Pharmacology we have brought together some of the foremost basic science and clinical researchers to discuss some of the new frontiers in the development of targeted cancer therapy. Although still in its formative stages, the development of targeted cancer therapies has already shown incredible promise in a limited number of cancer types. As basic cancer research and drug development continues, we expect this number to grow and more patients to benefit from these exciting advances. Through better patient selection and novel strategies to manage resistance, a future can be envisaged in which cancer can be reduced to the level of a chronic, manageable disease. Brought together some of the foremost basic science and clinical researchers Part of the renowned Advances in Pharmacology Serial Discussion of some of the new frontiers in the development of targeted cancer therapy

Motivated by the explosion of molecular data on humans-particularly data associated with individual patients-and the sense that there are large, as-yet-untapped opportunities to use this data to improve health outcomes, Toward Precision Medicine explores the feasibility and need for "a new taxonomy of human disease based on molecular biology" and develops a potential framework for creating one. The book says that a new data network that integrates emerging research on the molecular makeup of diseases with clinical data on individual patients could drive the development of a more accurate classification of diseases and ultimately enhance diagnosis and treatment. The "new taxonomy" that emerges would define diseases by their underlying molecular causes and other factors in addition to their traditional physical signs and symptoms. The book adds that the new data network could also improve biomedical research by enabling scientists to access patients' information during treatment while still protecting their rights. This would allow the marriage of molecular research and clinical data at the point of care, as opposed to research information continuing to reside primarily in academia. Toward Precision Medicine notes that moving toward individualized medicine requires that researchers and health care providers have access to very large sets of health- and disease-related data linked to individual patients. These data are also critical for developing the information commons, the knowledge network of disease, and ultimately the new taxonomy.