Understanding the benefit-risk balance of medicines

Walking in Many Shoes: A metaphor for understanding the benefit-risk balance of medicines

07.30.13 | By Sascha Haverfield

The assessment of benefits and risks is critical in the development of new medicines. Last year’s reauthorization of the Prescription Drug User Fee Act (PDUFA) included a new Patient-Focused Drug Development initiative at the Food and Drug Administration (FDA) to gain patient insight, including perspectives on the benefit-risk balance of medicines, in 20 disease states over five years.

PhRMA welcomes the FDA’s efforts to advance Patient-Focused Drug Development in order to incorporate patient perspectives on benefits and risk in the regulatory review of new therapies

As the following guest blogpost by Frank Rockhold, Senior Vice President of Global Clinical Safety and Pharmacovigilance, and  Marilyn Metcalf, Senior Director of Benefit Risk Evaluation, at GlaxoSmithKline explains, the expectation of benefits and tolerance for risks often varies not only from disease to disease, influenced by factors such as the severity of the condition and other available treatment options, but also from patient to patient based on personal preference and lifestyle.

We are committed to working with the FDA and all stakeholders on a benefit-risk framework to facilitate discussions with biopharmaceutical companies throughout the drug development process to facilitate a more efficient, high quality review of new medicines to meet currently unmet patient needs.

Benefit-risk is one of those ideas that seem straightforward.  Does the good outweigh the bad?  As with most simple questions, the answer becomes more complicated when you start talking with someone whose life journey takes a different path from your own. 

The idea is grounded in patient well-being.  In medicine development, we look for a treatment’s effects on how a patient feels or what s/he is able to do.  We want to know how much the medicine helps to avoid or improve a condition, how long these effects last, and whether we can predict if the treatment will work for some patients differently than for others.  Similarly for risks, we look for unfavorable effects-- their severity, duration, predictability, and whether we can monitor patients appropriately if these effects occur.  We also check to see what happens if dosing is reduced, temporarily interrupted, or stopped altogether.  We are one part of a healthcare system that tries to address patients’ needs in a number of ways, so as drug developers we must walk in a variety of shoes.    

In most cases, a pharmaceutical company or researcher is looking for “the patient’s” point of view.   But of course, there isn’t only one patient.  There are many patients—with different opinions, cultures, responsibilities, etc.—whose needs we are trying to meet.  While they all may seem to have the same or similar goals for their treatment – to maintain their health or get better – their circumstances can vary significantly.  For example, one patient may want to preserve his current health state for as long as possible, but may also have family or career considerations.  Another may want to maintain her more active lifestyle, or continue traveling, etc.  Each patient’s risk tolerance for the amount of benefit they hope to receive can be affected by a number of these circumstances. 

As developers of new medicines, we take into account these kinds of patient considerations as we decide whether to continue or cease developing a medicine.  We work to understand whether this medicine will make enough of a difference to be worthwhile to patients.  If we believe that it will, we present our evidence to drug regulators, who must decide whether the medicine should be available to a population of patients.  Different regulators may consider this question differently for the patients in their own countries.  Those who are responsible for paying for the medicine then assess the benefit-risk to determine whether the benefit provided – and the overall value of the medicine – is worth the cost.  Ultimately, once a medicine is approved by regulatory authorities, all of this work points to the patient, and often his or her healthcare provider, who must (together) determine the benefit-risk based on the patient’s individual circumstances.  The evidence and considerations of drug developers, regulators, and other patients can help to inform individual decision making, and that is where communication of benefit-risk in a way that has relevance for “a patient” has paramount importance.

There is opportunity for everyone involved in this process to learn how we can continue to improve our understanding.  As we improve the study of benefit-risk, we ask ourselves:  How can we better incorporate the perspectives of patients into drug development decisions?  How can we more effectively communicate the evidence for benefit versus risk?  In what ways can information from clinical trials be translated to inform diverse individual patient decisions in everyday practice?  What are the best ways of collecting ongoing patient experience in the broader “real world” while respecting privacy and not inconveniencing patients?  How do we maintain the scientific rigor needed for this kind of observational data to be informative?

We’ve always had to focus on weighing the benefits vs. the risks of potential new medicines and vaccines, yet in many ways it is still an emerging field.  It is challenging, sometimes frustrating, and always invigorating to see how far we have come in determining the benefit-risk balance of a medicine, but there is much left to do.  In the coming years, we will look increasingly to patients for their insights.  And we believe drug development will be a more fulfilling field as a result. 


Guest post written by Marilyn Metcalf, Senior Director, Benefit Risk Evaluation, and Frank Rockhold, Senior Vice President, Global Clinical Safety and Pharmacovigilance, at GlaxoSmithKline.



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