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Drugs in the Real World
The most publicized way of determining the efficacy and effectiveness of a drug is through randomized clinical trials. However, how would one determine these after the drug goes into the market? This is when real world studies into the effectiveness and success of drugs come into play. Generally called outcomes research, this is research into the final outcomes of the treatments. Very commonplace in the United States and Europe, organizations in Asia have also begun looking into and placing priority on outcomes research.

Rachel Lim speaks to Dr. Nancy Dreyer, Global Chief of Scientific Affairs from Quintiles Real World & Late Phase Research to find out more about this research and its importance.

How would you describe outcomes research?

Outcomes research is the study of patterns, treatments and other factors about people’s lives to try to understand what factors influence how effective or how risky a treatment may be, and whether the benefits outweigh the risks for various types of patients. It is a study of the interrelationships of factors in people’s lives, including the medications they take, what other illnesses they have, whether they are physically active, etc., and how all those things influence the development of disease.

What we study with outcomes research includes the natural progression of disease, also sometimes called the natural history of disease. Taking leukemia as one example, some areas this would cover include what people who develop leukemia can expect when they are first treated; what happens to people who develop it at a certain age; how are they treated; how long the treatments generally work; how many people survive for one year, five years, ten years, etc.; and what characteristics differentiate those who respond well to treatment and those who don’t. For some diseases, we also look at those who develop complications or what makes their disease more active than others. We try to understand factors that affect one’s prognosis.

The way it’s used in the pharmaceutical industry is that we try to understand whether treatments work once they’re introduced into what we commonly call the real world. When drugs are being developed to be brought onto the market, they are studied in optimal patients, in the best situations, the cleanest patients (meaning as few co-morbidities as possible) and the best doctors. We are looking at a situation where if a person only has one disease in a certain stage, would this drug work?

But once the drug comes onto the market, it is used much more widely. So then we can begin the serious study of what we call treatment heterogeneity, or which subgroups treatments work best for, and what the balance between risks and benefits is for those subgroups. This type of information is used by doctors and patients and health systems to understand whether a treatment is effective, whether it is cost- effective, when it is most effective, whether it needs to be the first treatment, does it work well in combination with other medications, and most important, for what type of patient does it work well.

For example there are a lot of interesting treatments for rheumatoid arthritis, we have some registries — studies of people who have the disease — where we study how they get diagnosed, how they get treated, every treatment including dose, mode of administration, etc.. Sometimes the dose needs to be modified for certain types of patients, like the elderly. Sometimes we study one product, all treatments, or all treatments in the class. Then we follow up with the patients to look to see who gets better or who gets serious side effects.

Outcomes research is usually funded by pharmaceutical/ biotechnology companies because they are the ones who need the information, for themselves or to supply to others. They are now increasingly being used by medical societies and academic researchers who try to understand the effect and risks and benefits of treatments in conditions of real world use.

It is very widely done in America and Europe but more are now being done in the Asia-Pacific countries.

Why is outcomes research important?

The success of the treatment depends not only on just the drug, but also when it is administered, how it is administered, the ethnicity of the patient and the culture around medicine taking and what interactions you might experienced. (Outcomes research) guides the practice of medicine and guides the determination of treatment choices. The new questions people ask when they go to doctors include does this treatment work for people like me?’ and ‘how do people like me tolerate this treatment?’ or how do they feel?’ Patients are becoming more engaged in their treatment choices. Physicians are asking more questions about patients that were never studied during clinical trials. For example, with anti depressants, a patient might become pregnant and say ‘can I keep taking my anti depressant medicine?’ and ‘will this harm my baby?’ Well, where would you get information like that? So this information that comes from outcomes research has a very practical impact on a large percentage of the population who represent conditions and situations that were never studied in clinical trials.

The data that comes out from these observational studies are of great interest and are becoming published in major medical journals more often and are being used by physicians and medical congresses.

Outcomes research is especially important for those involved in innovative medicine. Because as we bring these exciting new products to the market we’re hearing more questions about the long term benefits and the long term safety. Outcomes research can be very important here, especially for medicines that are being used in populations that were never studied prior to market authorization.

Why is there an interest in outcomes research in Asia?

Asia, like the rest of the world, has focused most of its energies on drug safety, ensuring that when drugs reach the market, they are safe and effective. But now, regulators and other authorities in Asia are questioning about how drugs really work once on the market. So the natural questions are arising about whether one treatment is better than another treatment. We would never have that information without conducting outcomes research.

As more treatments come out on the market, they’re looking for the answers to questions like ‘how do I know which is the best for my patient?’ I was in Shanghai for the China Drug Information Association meeting and we talked about these non-interventional studies to a very large room, very crowded with people asking information about how to conduct these studies because there is such interest, so now they want to know how to do these studies correctly.

It’s a relatively new science. Historically, everyone paid attention to clinical trials, we know that the CFDA has a very thorough program of accrediting hospitals for good clinical trial practice and that is important but now for studies that happen after approval, we need different methods, because these studies are usually much larger and often go for much longer time periods.

Outcomes research is a very common practice in Europe as well as the US, but we are now seeing more studies that include the Asia Pacific.

In the US and Europe, there are payers, whether insurance companies or single payers and those companies or organizations that pay for the medicine want to pick out what they think are the medicines that give the greatest value.

So the growing interest we see is not only because of the demands of physicians but also the growth in Asia of comprehensive single payer programs in China or other health insurance programs. The people who pay for medicines want evidence in the types of patients they provide coverage for, and if you have good evidence for them, you are more likely to get reimbursed.

But I think also, in countries like China, the population is aging so there is greater interest in understanding how to manage this population, whether it is cancer, cardiometabolic disease or something else. A lot of the patients with these kinds of chronic illnesses are coming to medical attention so the potential for giving people the wrong treatments or spending money on treatments that don’t make a difference is becoming a greater concern.

That’s why there’s so much attention on the Chinese edition of a book we have written on “Registries for evaluating patient’s outcomes: a user’s guide.” This book was originally created for a payer in the United States that provides healthcare coverage for the elderly, the poor and children. They need evidence so they know what to pay for. But they rarely had reliable evidence on the type of patients they cover, because you don’t do clinical trials on the elderly or children or the very poor. So we need to use this non-interventional research approach, and to know how to do it and what is the best thinking. The US government funded the creation of this User’s Guide for registries and observational studies in 2007. It was so popular and so widely used that they asked us to update it in 2010 and again in 2014. We entered into a relationship with the University of Peking, the Chinese CDC and Hoffman-La Roche, all of whom worked to translated the book into Chinese and made this information available to China. And it has been very well received

What are some current ongoing case studies of outcomes research?

Quintiles Real-World & Late Phase Research is in early discussions to participate in a type two diabetes registry in several Asian countries to form the ‘Asian Diabetes Outcomes Consortium’ (ADOC) to leverage the registry data to improve diabetes treatment. While not final, the countries targeted for the registry are China, India, South Korea, Taiwan, Indonesia, Malaysia, Thailand, and Singapore.

The consortium (the registry is part of ADOC) would include academic institutions, biopharma and medical device companies, and other stakeholders. ADOC members would get semi-annual reports using data generated from the registry on the comparative safety and effectiveness classes of diabetes treatments, along with other information. For example, academic partners will get access to information on how their practices compare to other physicians with similar training and experience, and who work in similar settings.

The Asian diabetes consortium and registry are very important for a variety of reasons.

  • Type 2 diabetes is a rapidly growing world-wide health dilemma. According to International Diabetes Federation estimates, 382 million people had diabetes in 2013; by 2035, 592 million people will.
  • The Western Pacific and Southeast Asia regions had the
    highest levels of diabetes, at 138 million and 72 million
    respectively in 2013; these numbers are expected to increase
    by 46% and 71% by 2035.
  • China and India have the largest current and projected
    populations of people living with diabetes, at 98 million
    and 65 million respectively in 2013, growing to 143
    million and 109 million by 2035. Ageing populations, growing
    urbanization, physical inactivity, dietary factors, and other
    lifestyle changes are believed to play a major role. Genetic
    factors may also be important.

About the Interviewee

Nancy Dreyer is Global Chief of Scientific Affairs for Quintiles Real-World & Late Phase Research. She has more than 25 years’ experience in the design, conduct and interpretation of epidemiologic research. She heads a team of researchers who design, conduct, and interpret observational and interventional research on comparative effectiveness and safety, outcomes research, and quality improvement programs.

Prior to joining Quintiles, she was CEO of Epidemiology Resources, Inc. for 20 years, where she founded the peer reviewed journal, Epidemiology.

She serves as a senior editor of two AHRQ handbooks, “Registries for Evaluating Patient Outcomes: A User’s Guide,” now in its third edition; and “Developing a Protocol for Observational Comparative Effectiveness Research”.

She also leads the GRACE Initiative which is developing guidance on Good Research Practices for Observational Studies of Comparative Effectiveness (www.graceprinciples.org).

Additionally, Dr. Dreyer is an Adjunct Professor of Epidemiology at the University of North Carolina at Chapel Hill, a Fellow of the International Society of Pharmacoepidemiology and a director of the Drug Information Association (DIA). She is also a member of the Interim Steering Committee for the FDA’s new medical device epidemiology network (MDEpiNet), and is co-lead investigator with the European Medicines Agency for a study on developing new methodologies for pharmacovigilance.

In 2013, Dr. Dreyer was recentlyappointed as a member of the Academic Consulting Committee at the Center of Postmarketing Safety Evaluation at Peking University Health Science Center in China.

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