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)

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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



MES: Benefit-Risk Assessment Frameworks

As part of the European Medicines Agency (EMA) Benefit-Risk project, the PrOACT-URL framework was adapted for use in benefit-risk assessment of medicinal products, and the Work Package 3 conducted field tests, and conclude with a list of proposals for endorsing the framework, as an adjunct to current CHMP processes, including “adopting the PrOACT-URL framework to guide the process of evaluating the benefit-risk balance” …