Health technology deals with all aspects of healthcare, from preventive programs (vaccination drives), and diagnostic tests, to the development of devices or parts of equipment, drugs, or any medical procedure. Health technology assessment (HTA) entails a systematic appraisal of the short- and long-term outcomes of healthcare technologies. HTA provides information on various aspects of the use of health technology, and in the process highlights the value—clinical, economic, and humanistic—of such technology, in a systematic, transparent, unbiased, and robust manner in the real world. HTA aids in the evidence-based development of safe, effective, and patient-focused health policies.
Regulation on HTA
The European Union (EU) Regulation on HTA came into force in January 2022 and will be applicable from January 2025; it is believed that this regulation will bring the following important and comprehensive changes:
- Improvement in the availability of innovative technologies in the area of healthcare such as medicines and certain medical devices to patients and efficient use of resources, thereby strengthening the quality of HTA across the EU.
- Establishment of a coordination group of HTA national or regional authorities, a stakeholder network and to enact rules on the involvement of patients, and clinical experts in joint clinical assessments (JCAs) and joint scientific consultations (JSCs).
- Reduction in the overlapping of efforts for national HTA authorities and industry, easy business predictability, and assured long-term relationship of EU-HTA cooperation.
Implications of new EU-HTA Regulations
The most important implication of the regulation is the introduction of EU-level JCAs that will be applicable from January 12, 2025, for medicinal products with new active substances for oncological indications and advanced therapy medicinal products (ATMPs). JCAs involves the assessment of the effectiveness conducted by designated national HTA experts at the EU level.
The new regulation will impact all centrally approved medicines as well as new indications for HTA-assessed medicines and some medical devices and serve as the basis for national value assessments and price negotiations, making it essential for the industry, payers, and patients.
Pharmaceutical companies will have to submit complete reports of evidence for their clinical programs designed keeping the HTA requirements in mind for the JCAs, 45 days before the Committee for Medicinal Products for Human Use (CHMP) [the European Medicines Agency’s (EMA) committee responsible for human medicines] issues its opinion on granting marketing authorizations to the study products. When the medicine is approved by the commission, the JCA report will be published within 30 days. The JCA report will therefore have a significant effect as the first assessment, globally.
Role of JCAs
JCAs cover scientific analyses of the relative effects of health technologies on health outcomes against chosen parameters based on the assessment scope. They will include only a clinical assessment and will not comment on any recommendations about the product’s value or reimbursement. The member states will deal with any cost-effectiveness assessment, value assessments, and price negotiations or decisions as required. The decisions about comparators and endpoints will be made at the EU level in the future. The JCA report will serve as a basis for national decisions with respect to the clinical part of HTA in all EU countries, replacing national assessments.
The evidence submitted by pharmaceutical companies for JCAs should follow international standards of evidence-based medicine, and randomized, blinded, controlled, direct comparative studies are recommended as appropriate. The companies thus need to generate sufficient data to match the requirements of the regulators and the clinical and economic assessment by the HTA bodies during the early clinical developmental phase of their products while simultaneously managing budget and time constraints for early approval and timely introduction of therapies to patients. Besides regulatory approval, the pharmaceutical industry should strive to make new therapies available to patients by demonstrating their clinical and economic benefits compared to medicines already available.
Conventionally, before the introduction of EU regulations, reimbursement reports for HTA authorities have been prepared and submitted at the last stage of the drug development process. However, at this stage, most findings of the clinical trials, including efficacy and safety demonstration for the product intended indication, have been either finalized or are close to completion. Hence, the opportunity to add specific clinical effectiveness results may be potentially lost, resulting in an unfavorable impact on the HTA committee evaluation and authorization approval. Therefore, pharmaceutical companies can benefit from engaging with HTA authorities early in the drug development process and receiving inputs towards the data required as per HTA authorities’ expectations, thus avoiding many of the current issues.
At Bioviser, our team of experts in clinical regulatory processes and product market access supports clients in an early product development phase, aids in engagement with HTA authorities, and provides all the relevant information to meet HTA committee requirements and streamline the process of developing and launching new products.
