EURORDIS and Health Technology Assessment

Health policy decisions are becoming increasingly important as the costs of making wrong decisions continue to grow. But how are health authorities taking these important and often life-changing decisions? One answer is Health Technology Assessment (HTA).

HTA is a multi-disciplinary field of policy analysis that examines the long term effects of any existing or new health care technology. This includes drugs, medical devices, procedures and organisational systems used in health care. HTA also evaluates the medical, social, ethical and economic implications of these interventions. The goal of HTA is to support health care decisions and to serve policy making through objective information.

Today HTA plays an important role in determining reimbursement decisions.

For example, HTA can be used to analyse the cost relative to the benefit of medicines in order to determine prices and reimbursement policy of orphan drugs, something that has long been crucial to many rare disease patient advocates. “HTA should be the booster to implement the “criteria” developed in the rare disease sector to decide on risk benefit, relative effectiveness and cost effectiveness of health interventions,” argues Gerard Nguyen, a EURORDIS volunteer from Rett Syndrome Europe who attended an HTA training session. “Concepts which have been long promoted by EURORDIS, such as the significant benefit, clinical added value and relative effectiveness of orphan drugs, should be used in HTA reports to decide good healthcare policy.”

HTA is an ‘evidence-based’ process. So what evidence can patients provide? Patients have something to say about all aspects of an HTA, but the most important thing they can contribute is a description of the benefits or unwanted effects of a healthcare technology. However, it’s not easy to understand how to put these important views, needs and preferences into a form of evidence that can be used by HTA institutions. That is why it is so important for patients to understand HTA and be involved in its definition.

“EURORDIS has already started to allocate significant resources to build the capacity of its staff and volunteers, and will continue to be involved in HTA-related activities to better serve patients needs in this strategic and rapidly evolving field of policy,” says EURORDIS’ CEO Yann Le Cam. “EURORDIS understands the importance of HTA because it is directly linked to patient access and to quality of care. We are convinced that HTA is starting to have and will increasingly have a direct impact on the reorganisation of health services for rare disease patients, including centres of expertise, registries, drugs, standards of diagnosis and care, training for professionals and information. If badly used, with a narrow economic approach, HTA raises major challenges of patient access to care. However, HTA also has the potential to help regulate the offer for care based on quality, relative effectiveness and cost effectiveness, as well as to redefine consistent patient-centred healthcare pathways for long term quality and sustainability of healthcare services”

Indeed, five EURORDIS staff and volunteers received an introductory HTA training at the University for Health Sciences, Medical Informatics and Technology in Austria in February. HTA was also the focus theme of the last EURORDIS summer school in September. More than 30 patient representatives were introduced to the main HTA assessment tools by academics, HTA agency and industry representatives and public health experts. In addition, patient representatives had an opportunity to present their HTA experiences in a panel discussion.

Patient and industry involvement in HTA decision-making will also be at the core of the discussions at the next Drug Information Association HTA Conference. The conference which is linked to the 3 Nov DIA Conference entitled “Future Direction of Orphan Drugs in Europe”, will take place on November 4-5 and EURORDIS patient fellows from several countries will be present.

EURORDIS is also heavily involved in the EPPOSI project “Patients’ Involvement in Health Technology Assessment”. A first closed meeting, at the end of June, aimed at determining the role patients could and are willing to play. EURORDIS participated as one of the 25 experts invited to give their opinion and share their experience. EURORDIS will also participate with several representatives in the next EPPOSI Workshop in November. This open meeting will address the definition of a broader HTA approach – one that goes beyond considering cost effectiveness to include ethical, social and other concerns.

“Social and ethical considerations are rather “soft”, not well-defined in the dominant definition of HTA, and this is an area where EURORDIS and other patient organisations may be able to contribute to making this aspect of HTA more rigorous,” argues Rob Camp, another EURORDIS representative who received in-depth HTA training. “Patient representatives should be vigilant regarding the misuse of HTA limited only to “pricing”, especially when dealing with health interventions that affect rare disease patients,” adds Gerard Nguyen. “Patient organisations can influence the process via advocacy in HTA agencies.”

This is why EURORDIS sought involvement in the European network for Health Technology Assessment (EUnetHTA) and has been appointed as one of four patient representatives at the EUnetHTA Stakeholders Forum, which held its first meeting in June. EUnetHTA is an EU initiative to improve coordination and harmonise the assessment tools used by the main HTA agencies in Europe.

Read more:
Health Equality Europe Guide to HTA for Patients
HTA International Consumer and Patient Glossary

Author: Paloma Tejada
Photo credits: © EURORDIS

This article was first published in the November 2010 issue of the EURORDIS newsletter