Event 1: Evidence Generation: Quality of data for decision-making
October 21, 2021 5pm BST Video | Slides | Transcript
Evidence Generation: Quality of data for decision-making
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: Regulation and HTA
December 2, 2021 5pm BST Video | Slides | Discussion | Transcript
Regulation and HTA
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 3: Evaluation Process and Methods
February 10, 2022 5pm BST Video | Slides | Transcript
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 discusses the above problems and explores how these issues can be tackled.
Event 4: Translation into Practice
April 21, 2022 5pm BST Video | Slides | Transcript
In an ideal world, patients should have access to interventions which have been proven to improve outcomes, the healthcare system should be able to afford such interventions and discontinue using the less effective ones. Society and patients should benefit from healthcare innovations.
Gradually, practice corrects itself, old technologies are replaced with the new ones, harmful practices/interventions are removed from the market, innovations increase efficiency and quality of care. However, this process is often slow and faulty in many ways. We withdraw harmful products decades too late; we pay premium prices for medicines that have not demonstrated patient-relevant outcomes, we continue to use processes and technologies which should long be in history, we perform many unnecessary procedures in the fee-for-service systems, and we face waiting lists and late diagnoses in resource limited settings.
Health technology assessment has been developed and introduced into practice to help us improve by increasing efficiency of the healthcare system, informing policy, saving money, demonstrating which interventions are worth paying for and enabling patient access. How successful has it been in different settings? What have been the implications for patients, clinicians, payers, and policymakers?
How has HTA improved effectiveness and efficiency of healthcare in the setting with universal healthcare coverage? What can we do better?
Is HTA helping with priority setting in different healthcare systems and should it or is it the other way around - do system’s priorities define the direction and scope of the HTA activities?
Our fourth HTA in Question event focuses on looking at the impact that HTA has or should have in the healthcare system.
Event 5: HTA in Low-to-Middle-Income Countries
June 2, 2022 5pm BST Video | Slides | Transcript
Many low- and middle-income countries (LMIC) have historically relied on external support and funding to contribute to their efforts to achieve universal health coverage (UHC). Increases in healthcare costs as well as reductions in healthcare budgets (including reduction in aid funding), currently challenge this panorama. It is ever more crucial that countries obtain more health per resource spent, creating efficiencies in spending while considering equality and equity concerns.
In response to the World Health Assembly Resolution, WHO is increasingly focused on supporting and leveraging countries' efforts to improve health care decision making by the application of evidence-based priority setting mechanisms such as HTA.
Several factors challenge the implementation of HTA processes in low-income countries (LIC) as well as in middle-income countries (MIC), such as the lack of capacity and capability at both human and monetary levels, lack of available relevant local data, lack of interest in transparency and accountability and overreliance on HTA products and recommendations applied or adopted in higher-income countries with very different health systems, epidemiological profiles and decision problems.
Furthermore, HTA is conducted by interdisciplinary groups that use explicit analytical frameworks drawing on various methods. For these multidisciplinary groups to feel the ownership of the process, they must have clear roles and responsibilities. Involvement of the relevant stakeholders in the HTA process is necessary and this must follow an inclusive and transparent approach since the level of buy-in from each stakeholder into the process is crucial for its successful implementation. In addition, stakeholder engagement and overall HTA success is closely tied to the strength of political and public support for the role and remit of such efforts.
HTA is used in some form in many LMIC countries but the level of engagement from different stakeholders proves challenging in many of them. Barriers to stakeholder involvement include lack of transparency, confusion about the roles and responsibilities and power dynamics tensions such as litigation procedures and budget bias. There is also a perceived lack of trust between the stakeholders due to inherent vested interests.
This session discusses some of these issues, including:
- How can LMIC reduce their reliance on external funding for their UHC efforts?
- What key steps should LIC and MIC consider when embarking in HTA institutionalisation?
- Do higher-income countries have a duty to share their experiences and learnings in HTA with LMIC? How can this support be contextualized to local settings, particularly in very fragmented healthcare systems?
- How different are the challenges encountered by HTA agencies in LIC, MIC and those in higher income countries?
- What are key examples of LIC and MIC that have successfully institutionalised HTA processes, and what can others learn from these?
- How have power dynamics and vested interest of different stakeholders handicapped HTA efforts in LIC and MIC? How can these be mitigated?
