Patient preferences in benefit-risk assessment: Experiences by the Institute for Quality and Efficiency in Health Care (IQWiG)

by Sarah Mostardt and Ralf Bender (IQWiG)

created at

This is an article written by Sarah Mostardt and Ralf Bender from the IQWiG. The IQWiG is the scientific advise body for the political decision body in terms of reimbursement of new drugs – the Federal Joint Committee (GBA) – in Germany. It was established in 2004 folloing the so-called AMNOG process. The ANMOG is the German law defining the framework of the reimbursement process. The IQWiG states, that its goal is “to examine objectively the advantages and disadvantages of medical interventions for patients.” 

The scope is beyond drugs as it also provides reports about

  • non-drug interventions (e.g. surgical procedures),
  • diagnostic tests and screening tests, and
  • clinical practice guidelines (CPGs) and disease management programmes (DMPs).

The IQWiG is lead by Prof. Dr. med Jürgen Windeler and the deputy director PD Dr. med. Stefan Lange. The authors of the following article lead the Medical Biometry Department (Prof. Dr. Ralf Bender) and the Health Economics Division (Dr. Sarah Mostardt). They write:

Obituary: Richard Nixon, 1972–2016

This is the obituary written by Frank Bretz and Janice Branson. As Richard Nixon contributed significantly to the field of benefit-risk, I’d like to share this more broadly:

It is with great sadness we want to share that on Friday, August 26, the pharmaceutical statistics community lost Richard Nixon, one of its most talented statisticians, after he endured a yearlong journey with cancer.

It is difficult to find the right words to describe Richard as he really was a true role model for many of us. He was someone filled with love, kindness, sincerity and humor. He had a tremendous impact on drug development throughout his short life as he was a thought leader for Novartis and indeed the Pharma industry in areas of decision analysis and benefit-risk evaluation to name just a couple.

Richard was highly respected and his achievements were widely recognized within the pharmaceutical statistics community and beyond. For example, Richard received, in May this year, the prestigious PSI/RSS Award for Statistical Excellence in the Pharmaceutical Industry in recognition of his scientific excellence and exceptional contributions as he has significantly advanced and influenced quantitative decision-making at Novartis.

Beyond his technical capabilities, Richard had a keen interest, focus, and impact working at the scientific interface between industry, academia and regulatory agencies to advance practices of drug development. This is evidenced by the many cross-industry collaborations that he supports as a core member, such as IMI PROTECT and the EFSPI SIG on Integrated Data Analysis. As such, Richard has published extensively, resulting in almost 2000 citations and an h-index of 26 according to his Google Scholar profile – an outstanding record for a methodological statistician in the pharmaceutical industry. In addition, Richard was an excellent communicator, who was able to explain complex concepts in laymen’s terms. Through their reputation and expertise, statisticians like Richard ensure that our profession has a place at the table and we are able to lead discussions on innovation in drug development and approval.

Outside of work, Richard enjoyed time with his wife Valda and their baby daughter Kyra. They loved outdoor activities, especially skiing and cycling.

We are very sad to lose Richard but we certainly are so proud to have been his work colleague and friend years and we can all treasure the moments and interactions we had with such an esteemed colleague. It is also a reminder to us all of the need for more drug research and development to combat such illnesses.

Frank Bretz and Janice Branson


Last Wednesday at the Joint Statistical Meeting in Chicago, George Quartey (Genentech) presented case studies in a session titled Emerging  Topics in Benefit-Risk Assessment in Clinical Development Decision Making #597.  Excellent assessment of subgroups. George also coauthored chapters in the newly released book Benefit-Risk Assessment Methods in Medical Product Development: Bridging Qualitative and Quantitative Assessments (editors Qi Jiang & Weili He).

FDA publishes draft guidance on BR assessment including patient perspective

The FDA published a draft guidance on “Factors to Consider Regarding Benefit- 11 Risk in Medical Device Product Availability, Compliance, and Enforcement Decisions”. Notably it contains a section called “How FDA Considers Benefit-Risk in Patient Focused Medical Device Product Availability, Compliance, and Enforcement Decisions”. It also discusses patient preferences on the benefit, patient tolerances of risk, patient impact, and patient preferences for availability.

ISPOR publishes two reports on the use of MCDA

The International Society for Pharmacoeconomics and Outcomes Research (ISPOR) has published two reports on the use of multiple criteria decision analyses (MCDA). ISPOR uses MCDA as a collective heading for a set of techniques, that use structured, explicit approaches to decisions involving multiple criteria which aim it is to improve the quality of decision making. These reports result from a task force being established in 2014. The goal of this task force was to provide a foundational report on the topic and focus also on initial recommendations on how best to use MCDA methods to support health care decision making.

Three presentations at the PSI conference 2016 in Berlin

Presentations give an overview of Benefit-Risk methodology in the HTA setting with special focus on the German environment – Presentations by the EFSPI SIG on benefit-risk in collaboration with the HTA SIG

The session in Berlin on Tuesday morning will be introduced by Friedhelm Leverkus. He will give an overview of the processes of determining a price under the new law (Arzneimittelmarktneuord- nungsgesetz, AMNOG) on the regulation of medicinal products that applies to all pharmaceutical products with active ingredients that are launched beginning January 1, 2011. There are two steps – the determination of the added benefit vs an appropriate comparator and the price negotiations itself. Friedhelm will discuss choice of the comparator, patient relevant endpoints, subgroup analyses, extent of benefit, determination of net benefit, primary and secondary endpoints, and uncertainty of the additional benefit in this environment and what impact this has on the final summary of additional benefits and risks.

Welcome to the benefit-risk BLOG of the EFSPI benefit-risk SIG

This is the first BLOG entry in hopefully a long journey of helpful information around benefit-risk.

The benefit-risk special-interest-group is a cross-pharma and academia group of statisticians working together on statistical topics related to benefit-risk. The group is chaired by Alexander Schacht (Lilly) and co-chaired by Ian Hirsch (AstraZeneca). Further members represent many other companies. The group is still growing and please contact Alexander Schacht ( if you’re interested.

The main aims of the Benefit-Risk Special Interest Group are split into 5 key areas