Will Biogen survive without aducanumab

Focus on diversity in a few indications

Doctors newspaper:Dr. Wagner, from doctor to company boss: Does your medical socialization help you to better empathize with the emotional world of doctors?

Dr. Steffen Wagner: In any case. I do think that I can empathize with the doctor and patient situation very well. The idea of ​​having a patient in front of you for whom you have no treatment option is very difficult for any doctor.

And that's where biotech and other research-based pharmaceutical companies come into play, and they may be able to offer a solution for such cases. We still have far too many diseases for which the treatment options are very limited. Think of the 8,000 rare diseases.

Have you developed a feeling for which innovations have great potential?

SW: The close exchange with researchers and doctors at universities and in the company is important to me. This gives you a sense of what counts, what works and what doesn't. A medical background is helpful for this.

Dr. Steffen Wagner

» Actual position: Managing Director of the German branch of Biogen in Ismaning

» Career: Studied medicine at the Ludwig Maximilians University in Munich; practical year in Sydney, Johannesburg and New Haven (USA).

» Career: Before joining Biogen, he was Area Vice President at Alcon, the ophthalmology division of the Novartis Group. After working as a doctor, he began his professional career as a management consultant at Bain and McKinsey with a focus on the pharmaceutical industry.

Biogen is one of the leading biotech companies worldwide - the focus is on researching new therapeutic options in the field of neuroimmunological, neurodegenerative and rare genetic diseases. How do you define success?

SW: For me, success means pioneering work: developing drugs for areas with a high medical need for which there are currently no or only few treatment options - such as Alzheimer's or spinal muscular atrophy. We are currently experiencing an extremely exciting phase. We understand the genetics and causes of many diseases better and develop new potential. And to be involved in this as a research-based biotech company is a great challenge and what drives us.

The terrain is highly complex - supposed successes in development can also fail later. How do you and your employees deal with failure?

SW: You need stamina and perseverance. It often takes ten years from development to the finished product. You need employees who stay on the ball and who do not allow themselves to be discouraged by setbacks. You need expertise, and we have that especially in the field of neurology.

In order to compensate for individual failures, you have to focus, but not only bet on one horse. You have to examine a whole range of therapeutic approaches - that's what we do in Alzheimer's research, for example. Translated, this means: We rely on our specialized know-how and our many years of experience to deal with new challenging neurological diseases such as multiple sclerosis, Alzheimer's, spinal muscular atrophy (SMA), ALS, Parkinson's and others. to fight.

In your opinion, is the risk of failure too little perceived by politicians and the public?

SW: I guess so. This is also due to the fact that the topic is highly complex - if only because of the lead time. Often only the end route and thus success is seen. Little or no consideration is given to the hard work and the billions in investments in advance.

However, I also have to say that we as an industry have not always managed to communicate the complicated interrelationships simply and adequately. Completely correct: The perception is not where it should be.

What role does the early benefit assessment according to AMNOG play here?

SW: A very important one. The additional benefit assessment is a core component of the system. In general, I support the idea of ​​a benefit assessment. However, the current implementation of this idea is not working optimally.

This can be seen from the fact that three out of four drugs that have not been awarded any additional benefit fail for formal reasons. In quite a few cases, IQWiG and the Federal Joint Committee also come to different assessments.

Here is a huge gap between the perception of doctors, patients and pharmaceutical entrepreneurs on the one hand and the system on the other. This gap needs to be closed.

Will the processes improve after the AMNOG reform?

SW: The most recent changes have not changed any of the core problems. There are examples in which the specialist societies have recommended a product for first-line therapy, but the GBA has not found any additional benefit for formal reasons.

Here medical opinion and guidelines are inconsistent with the benefit assessment. This is not a good thing because it can irritate doctors and patients. And the big challenges will still come when it comes to taking biomarkers into account in the benefit assessment, as is the case with Alzheimer's research.

This is an important but very complex subject.

How do you classify the current discussion about the decision of the LSG Berlin / Brandenburg, according to which the reimbursement amount for pharmaceuticals must not be a mixed price and in the case of subpopulations without additional benefits, economic viability cannot be guaranteed?

SW: Professor Hecken described the decision as extremely dangerous. I explicitly agree with him here. If this is implemented in this way, then I fear that there will be extensive exclusions from ordinances. That is not what the patient wants.

So you are calling for the mixed price system to be adhered to?

SW: Yes, I think the mixed price system makes sense. It offers the doctor security in the prescription practice because he can decide individually. This gives patients who might be excluded access to innovations.

If you will: mixed pricing dampens the inadequacies of the AMNOG system. What we need is clear legal regulation.

Back to the topic of innovations: a few days ago the European approval for a drug for the therapy of spinal muscular atrophy was granted. We are talking about a therapy breakthrough. . .

SW: I can confirm that. The genetic causes of spinal muscular atrophy have been known for over 20 years. Now we can offer therapy for the first time. From July 3, Nusinrsen (Spinraza®) will be available in Germany for spinal muscular atrophy, incidentally the most common genetic cause of death in infants and young children.

Spinraza® has not only been approved for babies and toddlers, but also for teenagers and adult patients. This is important news for all affected patients and their families.

How many patients are we talking about?

SW: The incidence is 1 in 10,000 newborns. In the most severe form, infantile SMA, we are talking about around 100 patients. Otherwise there are different assessments of the number of young people and adults affected.

Is it correct that the study was terminated prematurely?

SW: We have set up an extensive study program with a large number of patients. In terms of survival and motor function improvements, the data were highly significantly positive.

After all, the difference was so clear that the study was terminated early in order to offer placebo patients the chance of therapy. In addition, we have set up a hardship program in which over 90 patients with the most severe form of the disease in Germany could be treated free of charge before admission.

How important is such an innovation compared to developments in MS?

SW: Multiple sclerosis is and will remain a core focus for us. In the past twenty years we have built up a broad portfolio with six different therapy options alone and have significantly expanded the options for treating MS individually.

And now, with Spinraza®, we are moving into the next area that we want to fundamentally change. Not only with the current therapy option, but we are also researching gene therapy.

My greatest wish would be that what we say today about treatment options and advances in MS therapy will later also be said about spinal muscular atrophy, Parkinson's, ALS and Alzheimer's.

Keyword Alzheimer's: Here the reports on the development of an active ingredient have so far been less optimistic - what about Biogen?

SW: As I said earlier, we don't just rely on one horse. We have built up a broad Alzheimer's portfolio at full speed. It comprises six candidates who use different mechanisms.

The six active ingredients are in different stages of development - two in phase 3, two in phase 2, one in phase 1 and one still in a pre-clinical phase. We have already included more than half of the patients worldwide for our phase 3 study with aducanumab.

Half a year ago you took over the helm at Biogen in Ismaning - Biogen has been in Germany for 20 years. What conclusions can you draw today?

SW: Let me make it clear with the treatment options for MS. I am proud that Biogen has helped make MS a treatable disease. As a result, the quality of life of the patients has also changed positively. We certainly have to stay on the ball here.

At the same time, we are concentrating on new areas in neurology, such as spinal muscular atrophy and Alzheimer's research - with billions in investments in research and production of innovative therapies.

The Biogen company

Branch: World's leading biotechnology company based in Cambridge, USA. Concentration on the areas of multiple sclerosis, neurodegenerative diseases and rare genetic diseases. Biogen has the most extensive portfolio for the fight against multiple sclerosis. Further products against MS are in development. In the rare disease area, Biogen has developed a first drug for spinal muscular atrophy. In the area of ​​Alzheimer's disease, Biogen has one of the largest pipelines with six active ingredients. Biogen also brings biosimilars to the market for advanced biopharmaceuticals.

Sales 2016: $ 11.4 billion worldwide

R&D expenses 2016: $ 2 billion

Employee: 350 in Germany, 7000 worldwide