Getting patients access to 'precision' medicines is crucial

• "Precision" medicines have the potential to bring highly effective therapies to patients and lower the overall cost of treating serious diseases.
• Precision medicines are dramatically changing the treatment landscape for deadly cancers — increasing survival rates and reducing the need for costly procedures and hospitalizations.
• In order to realize the promise of precision medicines, we must work to make sure patients have access to them and bring down the high out-of-pocket costs to these patients.

A decade ago, receiving a medicine designed for your specific genetic makeup or modifying your own immune cells to fight cancer may have seemed like something out of a science fiction novel. But today, "precision" medicines — tailored therapeutics based on a patient's distinct genetic characteristics — are turning fiction into fact for many patients.

Since every person is unique, not only do precision medicines have the potential to bring highly effective therapies and high-value care to patients, they can also lower the overall cost of treating many of the most serious diseases. The investments needed to discover and develop these medicines can substantially improve health outcomes, and reduce the cost of failing to appropriately target treatment, estimated to be tens of billions of dollars every year.

Tailoring medical treatment to the profile of each patient can enable physicians to identify the best course of treatment and often avoid or reduce adverse drug reactions and the toxic effects of medicines that may not be necessary.

For instance, according to a recent study published by JAMA Oncology, genetic profiling can predict which women with early-stage breast cancer have a lower risk for their cancer coming back after surgery, allowing up to 15 percent of patients to avoid unnecessary chemotherapy.

Most importantly, precision medicines can help patients live longer, healthier lives. Already, the first wave of precision medicines have entered mainstream clinical practice, including targeted therapies that now make it possible for patients with a once incurable form of leukemia, chronic myelogenous leukemia, to live close-to-normal life spans. Similarly, precision medicines are dramatically changing the treatment landscape for deadly cancers like non-small cell lung cancer and metastatic melanoma, not only increasing survival rates but also reducing the need for the costly procedures and hospitalizations that are now part of the standard of care for these diseases.

As a case study, consider acute myeloid leukemia (AML), one of the most serious and challenging blood cancers. Progress understanding and developing effective and safe therapy for patients with AML has been modest, and overall survival for patients with this terrible disease is measured in months. According to a study published by the journal, Biology of Blood and Marrow Transplantation, the average cost for the chemotherapy and stem cell transplantation involved in treating many patients with AML has been estimated to be between $280,000 and $500,000. Discovering why this disease occurs and developing targeted medicines to treat it are really the only alternatives to help these patients and to reduce the cost of treatment failures.

Yet, to realize the promise of precision medicines, we must act collectively across the health-care ecosystem to ensure that patients who desperately need these transformational therapies have access to them.

A problem that too many Americans face when prescribed specialty medicines to treat complex or rare conditions is high out-of-pocket costs. Many patients with the most serious illnesses face high deductibles and coinsurance requirements, which often put the latest, safest and most-effective treatments out of their reach. These patient cost-sharing barriers are one of the reasons half of the medicines used to treat chronic diseases are not taken as prescribed, contributing to the estimated $100 billion to $290 billion of unnecessary costs to the U.S. health-care system from medication non-adherence, as cited by the Annals of Internal Medicine and the New England Journal of Medicine.

We must do better. We need to work together to ensure access to these medicines and reduce the financial burden on patients. Towards this end, Celgene is proactively working with major commercial U.S. health-care payers on arrangements designed to give eligible patients access to our most recently approved medicine — a precision therapy with an accompanying diagnostic test — without deductibles, co-pays and co-insurance. By partnering with payers to offset and even eliminate patient cost-sharing as an obstacle to treatment, our hope is to prevent some of the financial burden that leads to many of the problems currently impacting patient care.

Our action is just one step in what will be needed to ensure access to the medicines Americans grappling with devastating diseases need. As health-care stakeholders, it is up to all of us to work together to develop market-based solutions to ensure that medical innovation continues to be valued, and that patients have affordable health care. We're not there yet, but we are getting closer. Celgene is working with U.S. commercial health-care payers to step up to that challenge. We are also committed to engaging with policy-makers on finding ways to develop innovative contracting strategies that can benefit patients with government insurance as well. We encourage others in the health-care ecosystem to join us in finding solutions to these challenges.

Commentary by Mark Alles, CEO of Celgene.

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