HTA challenges

Rebecca Aris interviews Károly Kulich


In this interview with Károly Kulich of Novartis, he discusses the current challenges in HTA in pharma and why he feels so optimistic about the future of HTA.

The pharma industry faces significant challenges with regards to health technology assessment, as expectations from regulators and HTA bodies aren’t always aligned.

To find out more, we spoke with patient reported outcomes expert, Károly Kulich of Novartis, who highlights the role of patient reported outcomes (PROs) within HTA and explains why he so optimistic about the future.

Interview summary

RA: Could you please start by explaining your role within Novartis?

KK: I have been with Novartis for two years, and I have been in the patient reported outcomes field for 12 years. I support PRO related activities within cardiovascular, metabolic disease, and respiratory therapeutic areas.

RA: What changes have you noted in HTA throughout your career?

KK: PROs in HTAs were not really discussed 10 years ago, but their presence has increased dramatically in the past five years.

PROs are yesterday’s nice-to-have endpoints and today’s primary or co-primary endpoints. Not only in pain but in other disease areas the patient voice is critical. If you have a primary, co-primary or a key secondary endpoint as patient reported outcomes obviously the HTA bodies have to review that.

This is one explanation for why PROs are increasing in the HTA world. The other is that the HTA guidance system reviews spend more and more time on analytical methods relating to PROs.


“…it seems that the gap between regulators and HTA bodies is being bridged.”


RA: What would you say are the key challenges associated with HTA in relation to PROs?

KK: The key challenges are that this is relatively new. It’s not always clear which particular guidance the HTA is going to apply. Currently, it’s a case by case scenario, so it’s up to the sponsor to reach out to the HTAs in good time, and make sure that the study and the endpoints are aligned with the HTA body directions and advice.

The other challenge, which is an encouraging new development is the collaboration between regulatory bodies and HTA, and what that bridging of these agencies will look like in the future. We have seen some very encouraging guidance coming out this year, and it seems that the gap between regulators and HTA bodies is being bridged by these guidances. Hopefully, in the future the expectations from regulators and HTA bodies will be very similar, which will make the development programmes and choice of endpoints easier for the pharma industry.

RA: What is the role of patient reported outcomes within HTA?

KK: In many chronic symptomatic diseases, PRO is a primary or secondary endpoint. It’s a natural part of any dossier submitted to the HTA agency. There are some publications that came out in 2000 and 2011 that reviewed the frequency of PROs by HTA bodies, and the number is really rapidly increasing.

The gap between regulatory bodies and HTAs is being closed with some of the emerging guidelines. The challenges will be less given that we get clear guidance, and there will be some good examples of where PROs have been evaluated by the HTA bodies, and the regulators at the same time, especially if there is some consistency between the two evaluations. I’m very optimistic about the near future on this.


“The patient voice will increase even more.”


RA: What is key to getting patient and the public involvement in HTA decision making?

KK: Patient organisations are very important in making the patient voice heard in the drug development process. At the same time we, ourselves within the pharma industry, need to do our best to be the patient ambassador and translate the patient voice as clinical trials endpoints.

To translate the patient voice into scientific and other messages that are interpretable by not only the regulators but also by the HTA bodies.

RA: How do you think HTA will evolve in the next 10 years?

KK: The patient voice in drug development will increase even more. I also think we can expect much more clear guidance, in the next 2–3 years on how our value dossiers and our programmes will be appraised, and how to present this to the HTA bodies.

The process, the guidance, and some good examples will help us to get better aligned in a more efficient way than in the past. And the way the HTAs look at PROs will be far beyond EQ5D and utilities, which has been in the past. It will be more about improved symptoms, functioning and satisfaction with treatment.

RA: Károly, thank you very much for your time and for your insights.

KK: You are very welcome thank you.




About the interviewee:

Károly Kulich is the Director and Patient-Reported Outcomes (PRO) expert at Novartis Pharma (Global Health Economics and Outcomes Research) in Switzerland, leading PRO activities in two franchises.

He has worked in the pharmaceutical industry for more than twelve years and regularly involved in regulatory and HTA agency interactions. He is a member in the IMI PROActive consortium steering committee, in the C-PATH Asthma working group and in the COPD, CBQC consortium. His therapeutic experience is in respiratory, neurology, gastrointestinal, cardiovascular and in several other diseases with over twenty publications. He has substantial hands on experience in designing and conducting clinical trials and methodological studies, interpreting PRO data and implementing e-PRO data collection. He is a distinguished reviewer and member in the International Review Board of Quality of Life Research and is a guest lecturer at several universities across Europe.

My background is in psychology and in PRO instrument development and validation. I earned my Bachelor’s and Master’s degrees in psychology from the University of Lund, in Sweden and have a Doctorate degree in psychology at the University of Gothenburg, in Sweden.

How do you think HTA will evolve over the next 10 years?