Blog

Conversations and healthy debate about issues facing our industry and the health care system are critical to addressing some of today’s challenges and opportunities. The Catalyst welcomes guest contributors including patients, stakeholders, innovators and others to share their perspectives and point of view. Like in our Conversations series, views represented here may not be those of PhRMA, though they are no less key to a healthy dialogue on issues in health care today.

To continue the dialogue about clinical pathways and what they mean for treatment options, we’re pleased to host a guest blog from Alan Balch, chief executive officer, National Patient Advocate Foundation.

Patient care is complex and there are competing schools of thought to consider. Should personalized medicine reign supreme? Is a standard of care better to oversee quality and control costs?

Clinical pathways sit at the center of this debate, although I don’t see the two ideas as mutually exclusive. Critics coin clinical pathways as a “cookie-cutter” approach that can hinder the personalization of patient care, and supporters consider them an important tool to reduce errors and cost, while increasing quality and efficiency. Whichever side you’re on, it’s important to keep the patient at the center of the discussion.

Clinical pathways can provide a detailed roadmap with essential steps for patient care based on clinical
guidelines — spanning from diagnosis to treatment to monitoring. The extent to which pathways promote or impede patient-centered care depends greatly upon the design of the pathway and the extent to which it is deployed in a manner that supports clinical judgment. Thus, pathways should be judged based on how they are put to use in a clinical setting and whether or not they are inherently comprehensive enough to support personalized medicine.

From the patient advocate perspective, the use of properly designed clinical pathways provide opportunities to improve care by enabling treating physicians to navigate their patients through various health care decisions, including clinical trials, based on different safety and effectiveness profiles as well as cost considerations. This will lead to clinical decisions that meet the unique needs of an individual patient. But, pathways can also pose challenges. A clinical pathway may be based too heavily on parameters associated with the “average patient” and cost containment and fewer options. This approach will leave little room for tailoring to individual needs thereby, contradicting a personalized medicine approach.

As conversations around the use of clinical pathways continue, it’s important to ensure the process is designed to optimize and personalize treatment protocols for each patient based on individual needs and their physician’s clinical judgment — not the financial interests of health care companies

Some pathways, for example, try to pigeonhole physicians into prescribing a limited set of therapies that are preselected by insurance companies, which may hinder patient access to more effective treatments. This limited approach more closely resembles common techniques payers use, like step therapy or “fail first” requirements, which regulate how a patient gets a medication — particularly more expensive ones — to control costs.

An important consideration is who is actually developing clinical pathways. Ideally, it’s the experts in the provision of health care (i.e., medical practitioners) who should be charged with developing and implementing pathways, with clear mechanisms for involving the recipients of that care (i.e., patients and patient advocates) throughout the pathway development process. Well-designed pathways will support shared decision making and allow patients to express their preferences for how to individualize their care, leading to a personalized treatment plan. Patients need to have confidence and faith that pathways will not simply be a hidden tool to steer them to a limited range of treatment options preselected by their insurance provider. Ultimately, the answer to patient involvement is simple: give them information. They deserve no less.

For more information about The National Patient Advocate Foundation’s (NPAF) position on clinical pathways, check out their new white paper.

High cholesterol itself is not a disease, but it can lead to disease. Every 40 seconds, an American adult dies from a heart attack, stroke, or related vascular disease, which equates to nearly 800,000 deaths per year. For those who do survive a heart attack or stroke, many are faced with serious illness, disability, and decreased quality of life. Despite being largely preventable, high cholesterol is a main risk factor for heart disease.

According to the Centers for Disease Control and Prevention, two in three Americans with high cholesterol do not have it under control, putting them at twice the risk for heart disease, and 44% of the population is expected to have some form of cardiovascular disease by 2030. If this becomes a reality, a projected $818 billion will be spent on direct care, and an estimated $276 billion will be lost in productivity.

Fortunately for patients, the death rate from cardiovascular disease has dropped 31% in the last decade, thanks in large part to innovative medicines and treatment options. In fact, in 2007, the average cholesterol level for U.S. adults was within the ideal range for the first time in 50 years. This drop is attributed to the increased use of cholesterol-lowering medicine by those over 60. Adherence to treatment regimens not only helps improve the well-being of individuals living with high cholesterol, it also reduces spending on other health care costs – including prescriptions and other non-pharmacy medical costs.

Despite the progress made, there remains a significant unmet medical need as some patients are unable to sufficiently control their cholesterol levels. Continued innovation and lifestyle changes are critical to reducing the societal impact of heart disease, and to helping patients live longer, healthier lives.

Effective and responsible sharing of clinical trial data continues to expand scientific discourse and discovery, as we have chronicled here on the Catalyst.

Through the PhRMA-EFPIA Principles for Responsible Clinical Trial Data Sharing, the industry has made a firm commitment to publicize its research findings and created numerous avenues for qualified researchers to access this data.

Member companies such as Pfizer, Johnson & Johnson andGSK, among others, have robust data sharing mechanisms and most recently Astellas announced its participation in the independent online data hub ClinicalStudyDataRequest.com.

Recently, a New England Journal of Medicine study examined the reporting of clinical trial results for trials registered on Clinicaltrials.gov. The research covered the period between January 1, 2008, and August 31, 2012 and found that industry supported trials had the highest rate of results reporting, outpacing the reporting of National Institutes of Health (NIH) trials and other government or academic institution-funded trials.

It is important to note that the timeframe of this study predates the PhRMA-EFPIA Principles, so it is safe to conclude that even greater reporting of results is occurring today. Under the Principles, PhRMA member companies made the following commitments:

-Patient-level clinical trial data, study-level clinical trial data, full clinical study reports, and protocols from clinical trials in patients for medicines approved in the United States and European Union will be shared with qualified scientific and medical researchers upon request and subject to terms necessary to protect patient privacy and confidential commercial information. Researchers who obtain such clinical trial data are encouraged to publish their findings.

-Companies will work with regulators to provide a factual summary of clinical trial results to patients who participate in clinical trials.

-The synopses of clinical study reports for clinical trials in patients submitted to the Food and Drug Administration, European Medicines Agency or national authorities of EU member states will be made publicly available upon the approval of a new medicine or new indication.

-Biopharmaceutical companies have also reaffirmed their commitment to publish clinical trial results regardless of the outcome. At a minimum, results from all phase 3 clinical trials and clinical trial results of significant medical importance should be submitted for publication.

The ongoing sharing of information among government regulators, the academic and medical communities, physicians and the public improves patient health and informs our understanding of disease and potential treatments. The biopharmaceutical sector is proud of its track record of cooperation and looks forward to continuing our collaborative work of researching new medicines to meet patient needs around the world.

The biopharmaceutical industry works hard every day to achieve this progress and the results are strong:

The U.S. cancer death rate is down 20 percent since the all-time high in 1991

Since the mid-1970s, survival rates are up 58 percent for childhood cancers, 54 percent for lung cancer, 50 percent for prostate cancer, 36 percent for colon cancer and 21 percent for breast cancer.

Since the start of the war on cancer, investment in new treatments resulted in 23 million additional life-years and $1.9 trillion in value to society overall.

There are over 3,000 drugs in development for cancer around the world. Of those, about 80 percent have the potential to be first-in-class treatments

Does anyone really want to slow this progress? We’ve made tremendous strides and we must keep forging ahead. But sometimes we as a society forget what we have achieved and focus on the wrong things. We are often asked, what is the cost of this progress? In fact, spending on cancer medicines in the U.S. remains low, representing less than one percent of U.S. health care spending. So for that small fraction of overall health expenditures, we are achieving great results for patients. There are breakthrough treatments and even cures on the horizon for so many cancer patients. There has never been more promise for those suffering from cancer.

For patients to continue to benefit, however, they must have access to these needed medicines. Unfortunately, there are a number of obstacles that patients currently face. One of the most challenging is the fact that insurers are increasingly forcing patients to pay a disproportionately high share of prescription drug costs, particularly cancer medicines. This can make medicines artificially appear to be the most expensive part of the health care system, when in fact they are delivering some of the best value to patients.

A recent study found that while the Affordable Care Act (ACA) was created to help patients get care, the insurance plans in the health exchanges are actually limiting options, especially for individuals with chronic conditions like cancer. According to a recent analysis, 75 percent of exchange insurance plans subject cancer medicines to coinsurance, which could result in the first month. The purpose of insurance is to help patients get the care they need, not impose artificial barriers to access or unnecessary cost burdens.

In CJ's words, “I think it is very important that every patient be given the chance to survive longer.” We couldn't agree more. Learn more about the value of cancer medicines and the benefits to patients at www.phrma.org/cancer.