HTA in Question

HTA in Question is a series of events led by Consilium Scientific to explore the current role, pros and cons of the Health Technology Assessment (HTA) processes and methodologies.

Four events will be moderated by Dr. Leeza Osipenko (Consilium Scientific, CEO) and will take place between October 21, 2021 and April 21, 2022 featuring a permanent panel of experts

Prof Carole Longson, CBE - former Head of Centre for Health Technology Evaluation at NICE
Eric Low, OBE - former CEO of Myeloma UK, now CEO of Eric Low Consulting
Dr. Dan Ollendorf - former CSO of ICER, now at Tufts Center for the Evaluation of Value and Risk in Health.
Dr. Wija Oortwijn - Senior Scientific Researcher - Radboud University Medical Centre, President of HTAi

More details about the events:

Each event will explore a different theme relevant to HTA and featuring guest discussants. Each panellist will discuss their perspective on pros and cons of the current HTA purpose and process and propose solutions / potential policy changes. This is an interactive event where attendees will be invited to ask questions, contribute to discussions, and help shape
policy solutions.

The objective is to produce a position paper summarising the current issues with HTA and propose an agenda for the future to make the processes best serve the public, patients, policy makers, payers, product developers, academia and regulators.

All events will be recorded and made available on the Consilium Channel.

Event 3: February 10, 2022 5pm BST

Evaluation Process and Methods

HTA agencies in different jurisdictions are using varying methodologies and processes for the evaluation of health technologies to inform adoption, pricing and reimbursement. Clinical practice characteristics, health system design, availability of resources and local data, and other contextual aspects contribute to such differences.

Many countries now follow several basic principles when applying their value frameworks, but none are viewed as a ‘gold standard’ in the field. Economic evaluation is an integral part of HTA-informed decision making in some jurisdictions while in others it is not used at all. Further differences in HTA processes and value judgements may lead to different outcomes in reimbursement decisions.

The timing of the HTA assessments has improved in many regions to bring reimbursement decisions closer to the marketing authorisation. However, certain processes and HTA capacity continue to be issues. For example, some HTA agencies pride themselves in transparency and inclusiveness of their processes while redacting vast amounts of clinical and economic data, while other HTA agencies are being criticised for obscurity of decision-making and approaches towards evaluation altogether. Capacity challenges may preclude assessment updates as new evidence emerges or even the number of technologies that can be evaluated at all during a given cycle.

Industry, academics, patient groups and some HTA agencies question whether current HTA methods are suitable for evaluation of medicines in rare diseases, cell and gene therapies, medical devices, or digital health applications. Such challenges push the field for further developments, methodological updates, and alignments.

How much the difference in methodological approaches and processes contributes to a divergence in HTA recommendations across countries? Are we being inefficient, redundant, and bureaucratic by having different methodologies and processes in every country?

We have centralised processes and methodologies for the regulatory evaluation of medicinal products in Europe. Should we have common methods and processes in HTA? The Regulation on HTA was adopted by the European Parliament on 13 December 2021. This regulation is designed to strengthen cooperation and quality of services and will help EU countries determine the effectiveness and value of new technologies and decide on pricing and reimbursement by health insurers or health systems. This is a major move towards the HTA methodological and process alignment in Europe. Will it work?

The third HTA in Question event will discuss the above problems and explore how these issues can be tackled.

We look forward to your participation, reflections, and questions during the session!

Event 1: October 21, 2021 5pm BST  Video | Slides

Evidence Generation: Quality of data for decision-making

Guest Experts: Dr. Frank Hulstaert (KCE, Belgium)

                          Prof Ian Tannock


Problem Statement:

Health technology assessment (HTA) has always faced an issue with comparators: global randomised controlled trials (RCTs) are often run against comparators which do not represent standard of care in the jurisdiction conducting an evaluation. For the same reason, populations in the trial might not be generalisable. Indirect treatment comparisons are routinely used by many HTA agencies, which increases uncertainty for decision-makers. In addition, while the methods for conducting indirect comparisons have evolved significantly in the past 10 years, important differences in trial design, measurement, and timing between contemporaneously-approved medicines often make such comparisons problematic or even impossible. Many HTA methodological frameworks rely on modelling of long-term outcomes not available from the trials and the less and poorer evidence base we have at the start, the more uncertainty we are facing with extrapolation.

Over the past 10 years the innovation agenda, pressures from investors, patient groups, policy incentives to accelerate access to new medicines have influenced clinical trial design. Speed of drug development has become a key metric of success. The regulators benchmark their performance on the number of medicines approved as it is not within their remit to consider the value that these medicines bring to patients and society.

In oncology and rare disease settings in particular, we are seeing an increased number of approvals based on single arm Phase II trials and unvalidated surrogate endpoints. A requirement from the FDA of 2 identical Phase III trials is no longer the norm. In general, trial duration and trial sizes are shrinking resulting in fewer data points and less robust evidence for reimbursement decisions. Despite an increase in the collection of quality-of-life data in clinical trials over the past decades, we are still far from this being a routine practice.

The issues highlighted above are increasing in volume and impact. There seem to be no signs of improvement on the quality of evidence requirements, pushing health systems to resolve uncertainty through price discounts, managed access agreements and the use of real-world evidence.

The first HTA in Question event discusses the above problems and explore how these issues can be tackled.

Event 2: December 2, 2021 5pm BST Video | Slides | Discussion

Regulation and HTA

Guest Experts: Rob Hemmings,  Rick Vreman, PhD

Problem Description:

Decisions on reimbursement of medicinal products can happen only after marketing authorization has been granted by a regulatory body in a given jurisdiction (FDA, EMA, MHRA, AIFA, etc – ‘the Regulators’). Regulators are responsible for assessing the medicines’ safety, quality and efficacy. The domain of HTA agencies is related to but distinct from this remit, in that it is to inform fair reimbursement of these medicines according to the value these medicines promise to deliver.

Regulators set the rules for the pharmaceutical industry regarding the required evidence base for marketing authorization long before HTA agencies entered the decision-making landscape. Today regulators continue to play a dominating role in access to medicines, and this has a significant impact on subsequent HTA activities and decision-making.

However, things are beginning to change and there is now evidence of closer working between regulatory and HTA agencies to optimize drug development and facilitate faster access to medicines. Does this mean they are becoming collaborative partners or uncomfortable inevitabilities for each other? How functional, balanced and appropriate is the current system of interactions when fundamentally regulators and HTAs have different processes and goals? If regulatory and HTA agencies are fulfilling such different remits, are they enabling an environment that effectively serves the needs of society? Whose interests does this interaction serve?

Regulators have been under fire recently for lowering the bar by changing evidence requirements for the approval of medicinal products. At the same time, drug prices have continued to grow across the globe, along with questions about whether these prices are aligned with the clinical benefits brought by new treatments. Decision volumes are increasing and so are the pressures on the healthcare system. What can be done to improve the regulator-HTA interaction to make it better serve patients and the public?

The second HTA in Question event discusses the above problems and explore how these issues can be tackled. 

Event 4: April 21, 2022 5pm BST
Translation into Practice

Event 5: June 2, 2022 5pm BST
HTA in Low-to-Middle-Income Countries
in partnership with Center for Global Development