Tensions between broad access to new medical technologies and efforts to contain health care costs are perennial. In recent years, this has become evident in the uncomfortable discontinuities that can occur between health technology assessment (HTA) and regulatory approval of medicines. In autumn 2009, OHE and the Institute for Regulatory Science at CMR International collaborated on a two-day workshop on the issues raised. Representatives of HTA bodies, regulatory agencies, the pharmaceutical industry and academics participated.
The basic problem identified in the workshop is that the remits and requirements of regulatory authorities and HTA agencies both differ and diverge. Satisfying the data requirements of each is an increasingly complex and costly undertaking for new drug sponsors. Moreover, while regulatory agencies have worked for decades to harmonize requirements for approval, differences in HTA requirements seem to be increasing across countries.
Workshop participants discussed the challenges and possible responses in plenary sessions and in a series of syndicates. The primary challenge identified was the need to develop ways for all parties to coordinate and cooperate, simplifying and rationalizing the process as much as possible in order to ensure timely access to important new therapies. The four syndicate sessions brainstormed a total of twenty recommendations for action to move in that direction, ranging from modular approaches for common dossiers to increased transparency, conditional approval mechanisms and quality review of HTA analyses.
Importantly, the syndicate recommendations and the workshop discussions explicitly recognised that more must be known before solutions to successfully address the challenges can be developed. This includes:
- more information about the data needs of HTA groups, both as a precursor for efforts to align or harmonize requirements across countries and to enhance the pharmaceutical industry’s ability to develop and provide necessary data in a timely manner
- a better understanding about how and how much the data requirements of HTA bodies and regulatory agencies can be aligned while still maintaining separate processes
- more information about how best to work with the pharmaceutical industry to produce dossiers that can provide as much core data as possible for both regulators and HTA assessors
- improving understanding of payers’ perspectives by specifically engaging in dialogue with them about what information they actually need to make informed decisions on access and pricing/reimbursement
‘Mismatched outcomes’ were identified as a particular concern during the workshop. These occur when ‘a regulatory body grants an accelerated approval for a new medicine for unmet medical need, and that approval is not compatible with current HTA requirements’. The workshop recommended that a framework to address this crucial challenge be piloted and that a registry of such pilots be available. Again, then, additional information is crucial to effectively addressing this issue.
CRMI (CMR International). (2009) Review and reimbursement: A special case for better co-operation. Report of a workshop. 29-30 September 2009. Surrey: Institute for Regulatory Science, CMR International.
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