Patient-Centered Clinical Trials USA 2015

Oct 19, 2015 - Oct 20, 2015, Philadelphia

Boost Clinical Trial Productivity – Collaborate with Patients at the Heart of your Research.

A Master Framework for Patient Centricity

Jeff Sherman on why industry engagement with patients should be thought of as a long-term relationship.

Instead of companies looking at patients as individuals on the outside of the process or as simply a contractual relationship, it is about changing that to a long-term relationship and asking how can we best collaborate.



Dr Jeff Sherman, Chief Medical Officer and Executive Vice President at Horizon Pharma explains to Danielle Barron why industry engagement with patients should be thought of as a long-term relationship

What is your bottom line definition of patient centricity in clinical trials?

Without patients there are no clinical trials; so you really have to think about it from that perspective. At the end of the day, the regulatory authorities are going to make a benefit/risk assessment on a drug vis-à-vis a patient population and health technology assessors are going to be looking at the value of the drug based on the treatment of the patient. So a lot of this revolves around how best to work together whether it is patient groups, study sites, industry or regulators, to facilitate this whole process. It’s not looking to co-opt anyone, it is about how best we can all work together to foster treatment advances in areas of unmet medical need. Patient centricity is the key to making that happen.

At the end of the day, all of us are patients. It’s in our best interests to ensure that clinical trials and drug development works well because we are all going to be dependent on those medications if not now, then in the future.

Certainly we are seeing more and more mention of patient centricity as it relates to drug development and clinical trials. I am involved with the Drug Information Association (DIA) as a former board member, past President, and inaugural fellow.  I am also involved with the Center for Information and Study on Clinical Research Participation (CISCRP) as an advisory board member.

One of the initiatives that these organizations have been involved in is the FDA Clinical Trial Transformation Initiative (CTTI) looking at how to streamline and speed up clinical trials as part of drug development. These groups have really embraced the concept of patient centricity and have started to look at best practices as it relates to patients, academia, and industry interactions. What you are going to see is more and more of a coalescence and roadmap of how best to interact and engage with patients, because at the end of the day, all of us are patients. It’s in our best interests to ensure that clinical trials and drug development works well because we are all going to be dependent on those medications if not now, then in the future.

How do you make sure that your concept is implemented internally?

When I joined Horizon Pharma in 2009 as the Chief Medical Officer, we were privately held and did not have a product approved. I was the eleventh employee. Now we have seven products on the market, are publically traded, have a five billion dollar market cap and are growing and evolving.

We focus in three main areas – primary care, specialty, and orphan or rare diseases. Certainly as you look at the spectrum we are operating in, rare diseases is an area where there is a marked need for close interaction with patient groups because the number of individuals with these disorders are very small. So we work with patient groups to find out how best to move a product forward as a potential treatment. But we are also involved in working in other areas such as osteoarthritis, which is a widely prevalent disease, which requires a different type of engagement.

Instead of companies looking at patients as individuals on the outside of the process or as simply a contractual relationship, it is about changing that to a long-term relationship and asking how can we best collaborate together to make this potential treatment happen.

We know we need to engage with patient groups early and often – so it is really about starting off a relationship, and you have to look at this relationship being long term.  Many companies may engage patient groups late in Phase III or maybe when they are running into difficulties in enrolment in a study for example, but the best way to engage is at the start of a program to get a better understanding of the disease, the natural history of the disease, and what are the challenges that patients face. You want to focus on what can get done to make this product as patient friendly and healthcare professional friendly as possible. We want to know what information we can provide to the healthcare community and patients so that they can make the most informed decision on whether this treatment may be right for them or not.

As a company, we are looking at what we can do to make the patient’s life easier – we treat individuals how we would want to be treated. We think like the individuals with the condition – if I were the patient involved in this study, what would I do to make my involvement as easy as possible.

Instead of companies looking at patients as individuals on the outside of the process or as simply a contractual relationship, it is about changing that to a long-term relationship and asking how can we best collaborate together to make this potential treatment happen. As a company, we are looking at what we can do to make the patient’s life easier – we treat individuals how we would want to be treated. We think like the individuals with the condition – if I were the patient involved in this study, what would I do to make my involvement as easy as possible. We always try to look at things by putting ourselves in the shoes of the person – say if I was the health technology assessor or the regulator or the healthcare provider at the trial site.

Tell us more about collaboration mechanisms that can be used to cross organizational barriers?

We need to make the product or treatment as accessible as possible for patients because otherwise it could be a great product, but if there are accessibility issues then it is difficult for a company to make it sustainable.

The way we try to approach things is with the end in mind, and since that end is having a product that is commercially available then you really have to look at what is needed to make that happen – it goes beyond just getting it approved. Particularly in Europe where health technology assessment is key, we have to anticipate the questions they will ask.

It is about showing a true value proposition for the product and trying to remove as many barriers as possible in terms of access to the product or treatment. Then you can work backwards from that goal, and it makes it a much more streamlined product development approach. It is about gathering the information and the networking to try and make it as easy as possible for the regulators to make their benefit/risk assessment and for the health technology assessors to make their value assessment.


Jeff Sherman, Chief Medical Officer and Executive Vice President at Horizon Pharma will be speaking at eyeforpharma’s Patient Centered Clinical Trials, Philadelphia, October, 19th-20th 2015. For more information on his presentation, click here.


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Patient-Centered Clinical Trials USA 2015

Oct 19, 2015 - Oct 20, 2015, Philadelphia

Boost Clinical Trial Productivity – Collaborate with Patients at the Heart of your Research.

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