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Good news in Rx: Treatment options keep growing

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When chain drug store customers are waiting in line at the pharmacy, odds are they have at least one thing on their mind: relief for their disease or condition.

Stephen Ubl_PhRMA

Stephen Ubl, PhRMA

The good news is that at the start of 2017, America’s chain drug store customers will see more available treatment options than at any other time in recent history, providing relief for more of America’s patients.

And there’s reason for continued optimism. After all, since its peak in the early 1990s, the cancer death rate in the United States has fallen by 23% and the five-year survival rate has increased 41% across all cancers.

Meanwhile, the global medicines pipeline has grown to more than 7,000– 70% of which have the potential to be first-in-class therapies, meaning they use a completely new approach to fighting a disease. And 42% of these medicines in development have the potential to be personalized medicines.

Yet more needs to be done.

Every two minutes, three American adults die of a stroke, heart attack or related vascular disease, many of which have the common risk factor of high cholesterol. While statins can be prescribed to help lower cholesterol levels, they don’t work for everyone. Further, cardiovascular diseases and stroke are responsible for a greater portion of overall U.S. health expenditures than any other disease area — currently accounting for 17%.

Offering hope to patients and the health care system is a new class of cholesterol-lowering medicines, known as PCSK9 inhibitors, which address a significant unmet medical need for patients unable to control their cholesterol levels with traditional statin therapy.

The progress we’ve made in treating diabetes is also encouraging, but there is still a great deal of work to do today — more than 3,000 years after the disease was first described in writing.

A recent Pharmaceutical Research and Manufacturers of America report highlights the more than 170 medicines in development for treatment of diabetes and related conditions.

cdr-filler-opinion-750These advancements are not only changing the way researchers and physicians think about the disease but also the way patients think about the condition and its potential impact on their lives. For example, there are now new insulin formulations that last a full day and others that are more fast-acting, depending on individual patient needs.

Across these and many other disease groups, much of the progress flows from a deeper, molecular-level understanding of each disease — especially the broader knowledge gained through research into personalized medicines, which use diagnostic tools to help assess the medical treatments and procedures that may be best for each patient. These tools can include testing a tumor or testing the blood to determine if a patient has a particular mutation, which then guides what therapy is chosen and increases the likelihood that a patient will respond.

Although the Food and Drug Administration approved only 13 personalized medicines in 2006, today there are more than 140. Additionally, personalized medicines accounted for more than 25% of FDA approvals last year, marking a record year for these innovative therapies and a year on which PhRMA member companies hope to build greater success.

Beyond the science, there remains a great deal of work to do in order to best serve America’s patients in accessing their medicines. Many, for example, are increasingly facing challenges when it comes to out-of-pocket health care costs. No patient should have to worry about whether they can afford the care they need.

PhRMA is committed to finding ways to build on and improve the competitive marketplace for medicines as we move toward a value-driven health care system. Pragmatic solutions, such as updating regulations to help increase certainty and predictability for payers and addressing outdated regulations that pose challenges to innovative payment arrangements, could enhance the market and address cost drivers.

The fact is that better access and adherence to treatments can improve overall health outcomes and provide savings to the health care system by reducing spending on other medical expenses, like hospitalizations and skilled nursing care. PCSK9 inhibitors are a good example, yet 70% of prescriptions for these cholesterol busters are being denied today by insurers. America can do better for patients — and must, as medicines also help with prevention and wellness, as adherence to prescribed therapies can help avoid the worsening of an underlying disease, reducing the burden on caregivers and improving productivity.

Biopharmaceutical innovation is tougher than ever as researchers pursue complex diseases, like Alzheimer’s and other neurological conditions that have vexed scientists for decades or even centuries. The research and development process is long and complicated: In Alzheimer’s there were 123 attempts to bring a new medicine to market between 1998 and 2014. Only four were approved. Still, the promise of hope continues, and biopharmaceutical researchers persevere in their search for the next generation of treatments and cures.

Like Chain Drug Review’s readership, we work for America’s patients. In 2017, we believe that this work will continue to pay dividends in longer, healthier American lives. And we believe that those dividends will be on the shelves of America’s chain drug stores in the near future.

Stephen Ubl is president and chief executive officer of Pharmaceutical Research and Manufacturers of America (PhRMA).


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