1. What is a Health Technology Assessment (HTA)
How patients can contribute to the Process
Avril Daly
June – Retina International Congress

2. What is HTA?
A Health Technology Assessment HTA is a form of policy research that examines the short and long-term consequences of using a healthcare technology. It is a multidisciplinary process that summarises information about the medical, social, economic and ethical issues related to the use of a health technology in a systematic, transparent, unbiased, robust manner. Where there is a lack of data, HTA can be used to generate information. HTA is used by healthcare providers across the world, and its use is increasing.
It Asks:
Does the technology work?
For whom?
At what cost?
How does it compare with alternatives already on the market?

3. Origins of Technology Assessment
The term "technology assessment" was introduced in 1960s in the US House of Representatives emerging out of a growing appreciation of the critical role of technology in modern society and its potential for unintended, and sometimes harmful, consequences.
Experience with the side effects of a multitude of chemical, industrial and agricultural processes, and such services as transportation, health, and resource management contributed to this understanding.
Early assessments concerned such topics as pesticides, automobile pollution, nuclear power plants, supersonic airplanes, and the artificial heart.
The concerns at the time that TA would be a means by which government would impede the development and use of technology remain with HTAs to this day.
Practitioners and policymakers recognise that HTA is evolving, flexible, and should be tailored to each specific task.

4. What is a Health Technology?
Health Technology is a term used to cover any aspect of healthcare. Examples include:
Prevention programmes (example: childhood vaccination programmes)
Diagnostic tests (example: the rapid diagnosis of Group-B streptococcus during labour)
A device or piece of equipment (example: Retinal Microchip)
A drug (example: the use of rapid-acting insulin analogues in patients with diabetes mellitus type 1)
A procedure (example: laparoscopy

5. What is the purpose of a HTA?
The goal of HTA is to inform the development of safe, effective, health policies that are patient focused and seek to achieve best value as defined by decision makers. HTA underpins decisions such as:
Should this treatment be reimbursed in a national healthcare system?
For which patients should it be provided?
For how long should patients receive the treatment?
HTA is a broad concept: for example, in reviewing a drug it may include efficacy (how it works in the ideal setting of a clinical trial), safety, real world effectiveness, and the likely social, legal, ethical and political impact of using this drug.

6. Approaches
HTA may look at the impact of a technology on an individual patient, on a
group of similar patients, on the healthcare system as a whole, or on all these.
HTA may assess evidence from a range of sources, for example:
Systematic reviews of clinical trials
Economic evaluations
Assessments of implications for healthcare services
Evidence from users of the technology
HTA may also use modelling, where specific assumptions are used to
make an estimate or ‘best guess’ to predict, for
example, the cost of using a technology in a certain
setting

7. How is health technology assessment used?
HTA is used differently depending on the country.
In the UK, it is used to guide decisions about whether treatments and other technologies should be available on the National Health Service, based on a judgement of whether they provide value for money. In other countries the focus may be less on value for money and more on evidence of effectiveness and cost effectiveness.
HTA can provide information to support decisions about priorities in healthcare or specific decisions about whether new treatments should be introduced, for example:
Health authorities thinking of putting in place screening programmes;
Health care payers deciding which technologies (e.g., operations, drugs) should be paid for;
Health care organisations deciding whether to exclude or implement new technologies (e.g. modern types of radiotherapy);
Health care companies producing new products (e.g. to demonstrate
a level of benefit for the product that justifies the cost).

8. Difficult Decisions
People facing the challenge of illness need to receive effective treatment and care to give them the best possible chance of health. At the same time, there is a public health need to prevent disease wherever possible. But resources may be limited and must be allocated appropriately, based on affordability and effectiveness
Decisions such as ‘Should every available treatment for rheumatoid arthritis be provided or should the resources be used to give home care to people with dementia?’ and ‘Should everyone over 50 have their cholesterol tested or should the funds be used to provide vaccines against HPV (human papilloma virus) for teenage girls?’ are the sorts of challenges faced by policy makers.
Decisions about what treatments should be made available for example,
the available treatments may be relevant to a small group of
affected patients only or may have side effects which some patients find
intolerable.

9. HTA can provide information to support decisions about priorities in healthcare or specific decisions about whether new treatments should be introduced, for example:
Health authorities thinking of putting in place screening programmes;
Health care payers deciding which technologies (e.g., operations, drugs) should be paid for;
Health care organisations deciding whether to exclude or implement new technologies (e.g. modern types of radiotherapy);
Health care companies producing new products
(e.g. to demonstrate a level of benefit for the product
that justifies the cost).

10. How is health technology assessment carried out?
Although the process varies, it is generally acknowledged that four core components are considered in a health technology assessment:
The existing medical need which is not already being met
The clinical process which addresses that need
The evidence on the technology being considered and the interpretation of the evidence
Value for money of the technology (although this is not always considered)
The evidence may be used to assess the answers to questions such as:
Does the technology work?
What benefit does it provide and for whom?
What does it cost (to the healthcare service, to the patient, etc.), including opportunity costs (what could be gained if the funds were spent on alternative healthcare strategies)?
How does it compare in terms of efficiency with the available alternatives?
Does it work in this healthcare setting? Should we do it here?
If so, how should we do it?

11. Why patient experience is important
Although the outcomes of HTAs may be relevant to a range of stakeholders, the public, patients and their caregivers are often the group most directly affected by HTA decisions.
HTA tends to review evidence that is collected in scientific studies As a result there may only be limited evidence about the real impact of the technology on the daily lives of patients, their caregivers and the public
No one knows better what it is like to live with an illness day in, day out, than those who are doing this - the patients and their family and friends who care for them.
This experiential’ evidence gives this unique insight that patients and patient groups can most usefully contribute to the HTA process. Patients and their caregivers understand and can describe the true benefits delivered by a treatment and the real extent of the
unwanted effects.

12. Continued
Saying you were sick five times each day is less meaningful than explaining that this means you cannot manage to go to work, or need a carer.
Recording that the fatigue caused by existing treatments is so severe that it means you have to lie down all day and so cannot look after your children.
Explaining that a pill is more acceptable than an intravenous treatment not just because it means less trips to hospital but because it allows you to continue living a more normal life.
Describing the effect a treatment has on your daily life – such as, ‘it makes it impossible to stand on my feet all day, which means I cannot work’.

13. What is patient evidence?
Patient evidence might include:
The burden of the illness & the nature of the illness:
Is it hort or long term, some limited symptoms or many, symptoms that are difficult to live with, fatal or not?
The limitations it imposes on:
Daily life, ability to work, social life
enjoyment of family and friends
The impact on a person’s mental wellbeing
Activities which people living with the illness find difficult
Whether the illness prevents people from fulfilling
their chosen role in life

14. Continued The technology being assessed:
What patients and carers expect from a new technology
What benefits the technology brings
How the benefits compare with those of existing treatments
Whether it helps affected people fulfil their chosen role in life
How important the benefits are to the patient themselves
The outcome from a treatment they would value the most
How the benefits impact on patients’ daily life
What unwanted effects the technology causes
How the unwanted effects compare with other treatments
How the unwanted effects are balanced against benefit
What would happen to patients if there was limited access to the technology
How patients and carers value the technology
How easily the technology fits into patients’ daily life
What is the impact on a person’s mental wellbeing?
The financial impact of the technology

15. When to become involved
Patients and the public may have an opportunity to be involved at all stages of the HTA process, but this will vary between and within countries
Careful consideration should be given to whether or not a HTA would be useful or whether it could actually reduce access to a technology. This may depend on how HTAs are used: if they are used to determine whether a technology is value for money, a negative assessment could damage access
Where a treatment is very new, or where it is relevant for a small number of people only, there may not be enough evidence to demonstrate value for money which may lead to a negative recommendation.
This is a major concern for those representing people with rare diseases – raising the issue of concern to us
Orphan Medicinal Product and Orphan Drugs

16. ORPHAN DRUGS
HTA – Orphan Drugs

17. The European Example
Research into Rare Diseases and the subsequent development of Orphan Drugs has been stimulated in Europe through Orphan Regulation 141/2000
BUT
In the EU equitable and timely access to approved orphan drugs for rare diseases remains a major issue
Not all Member States are able or willing to invest in those treatments

18. Current Challenges
Member States, when they have access policy for Orphan Medicinal Products, encounter difficulties in defining what a fair price could be for those products and how to manage budgets for treatment of rare diseases in a sustainable way.
The number of orphan medicinal products with marketing authorisation will increase in the coming years. It doesn’t mean that number of European patients having access to OMP will increase at the same rate if prices remain at their current level
The EU Commission Communication on Rare Diseases have highlighted the: “specific bottlenecks in access to orphan drugs through the decision-making process for pricing and reimbursement linked to rarity; the way forward is to increase collaboration at the European level”

19. To address these issues The Commission has agreed to set up a Working Party to exchange knowledge between Member States and European authorities on the scientific assessment of the clinical added value of orphan medicines.
It is hoped that these collaborations could lead to non-binding common clinical added value assessment reports with improved information that facilitate the national pricing and reimbursement decisions, without pre-empting respective roles of the authorities.

20. Assessment of the Therapeutic Added Value of Orphan Drugs (TAVOD)
Should be performed where the expertise is gathered, and this is not at national level, but within the EMA.
RD Patients agree a Working Party of the Committee on Orphan Medicinal Products (COMP) within the European Agency is in the best position to deliver an expert opinion on the scientific assessment of the TAV, which would support and speed-up decisions on pricing and reimbursement at national level.
The proposed Assessment structure for the Therapeutic Added Value of Orphan Drugs - composed of COMP members, European Commission experts, national Competent Authorities, payers and patient representatives – could perform a common scientific assessment of the TAV for each Orphan Drug and deliver an “opinion document”.
In this way Member States would pool their scarce scientific expertise to assess the TAV and would also recognise the value of this common assessment and opinion document. This system would avoid duplication of procedures at national level.

21. Increasing influence of HTA – European Example
An increasing number of European countries have HTA agencies: 35 HTA agencies or organisations in 17 countries in the European Union (2008).
Decisions on Health care are not based on explicit criteria. HTA is a way to have scientific basis for decision, comprehensive and systematic criteria.
Therefore a positive recommendation may not lead to positive decision – political decision
However HTA recommendations are not mandatory, therefore
it is important to try to influence decision making
afterwards as well

22. So what can we do?
Patients are most directly concerned by healthcare decision. They should make sure their point of view is taken into account in HTA process. Opportunity to advocate for patients needs.
HTAs take into account mostly scientific and sometimes economic evidence, evidences about impact of the disease on the daily life are limited (QALY – Quality Adjusted life year)
As mentioned already patients are best placed to contribute as they are expert in living with the disease. Only they know impact of disease and treatment on daily life.

23. Why we need to act now!
HTA is one of the single biggest issues facing the members of Retinal International in the short term as therapies come on the market therefore our patients need to be aware of the issues involved in their countries.
Therapies for rare retinal dystrophies will be extremely expensive and with economic pressures being as they are reimbursement may be challenged.
Having spent over 3 decades funding research to bring about therapies to be denied access because of a HTA decision would be disastrous.

24. Things to consider
Do you know what HTA is in your language?
Do you know the agency in your country who do HTA?
Are you involved in HTA?
If yes, at which stage?
How can you make your voice heard?
What sort of support you would need to be involved?
Who should provide it?
What should Retina International do?

25. Conclusion
HTA will become an increasingly important influence in decisions about what is provided in healthcare, whether screening for diseases or treatments for serious illnesses. Already in Europe there is considerable discussion about greater collaboration between HTA agencies
It is important that HTA takes account of the needs of the people who will be most affected by its recommendations: patients, their caregivers and the public. While participating in a HTA can be time consuming and challenging, it is a real opportunity to influence the delivery of healthcare. Patient and public input can help determine whether a new treatment is made available. This guide will, we hope, help patients and the public make the most of any opportunity to contribute to a HTA.

26. THANK YOU