Regulatory policies vs HTA practices and their impact on access to innovative treatments

Mills, Mackenzie John (2023) Regulatory policies vs HTA practices and their impact on access to innovative treatments. PhD thesis, London School of Economics and Political Science.

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Abstract

Introduction: Innovative therapies must overcome two key hurdles to be made available for routine use in a patient population. The first hurdle is regulatory approval, where the safety and efficacy of a therapy is evaluated to ensure it has a positive benefit-to-risk ratio. The second hurdle is health technology assessment (HTA), where a therapy is compared to the existing standard of care in terms of relative therapeutic benefit and, in many cases, cost-effectiveness to inform funding decisions. Objectives: This paper-based thesis explores the relationship between regulatory approval and HTA and its impact on patient access to innovative treatments. Specifically, this thesis examines two types of regulatory pathways which reduce clinical development time and expedite regulatory approval: 1) conditional marketing authorisation and 2) approval of medicines with multiple therapeutic indications. Methods: Study 1 presents a rich descriptive analysis on the evidence gap between regulatory approval agencies and HTA agencies on conditionally approved medicines, along with an examination of the key clinical and economic issues raised by HTA agencies during assessment. Study 2 explores whether current pricing practices generate perverse incentives for the launch of medicines with multiple therapeutic indications through a mapping and analysis of regulatory approval and HTA approval sequence of multi-indication medicines. Study 3 presents the results of semi-structured interviews with current and former healthcare payers on policies relating to the assessment and pricing of multi-indication medicines. Finally, study 4 explores whether conditionally approved medicines face increased barriers at HTA level relative to products with standard regulatory approval, through an econometric analysis on the determinants of HTA decision-making. Key Findings: Results from confirmatory trials for conditionally approved medicines are frequently not available at the time of HTA, requiring HTA agencies to make recommendations on the basis of single arm or early phase studies. Rejection rates for conditionally approved medicines vary significantly across settings. Rejected medicines have a higher frequency of unresolved issues in magnitude of 13 clinical benefit, study design and economic modelling. Relative to medicines with standard approval, conditionally approved medicines likely face increased barriers at HTA level, both in terms of probability of HTA approval and time to HTA approval. Current pricing practices likely generate perverse incentives to sequence the launch of multi-indication medicines. Pharmaceutical firms show a tendency to prioritise niche indications with high unmet need for their first indication and frequently withhold the launch of subsequent indications. Despite evidence of launch sequencing, most healthcare payers express concern that a formal indication-based pricing model would involve high administrative burden and expressed confidence that current systems would facilitate access to innovative therapies when an unmet need is genuinely addressed. Policy Implications: HTA, pricing, and reimbursement systems likely limit the extent to which expedited regulatory pathways can accelerate patient access to conditionally approved and multi-indication medicines. Greater alignment on evidence requirements could be achieved through enhanced use of joint early dialogue or through implementation of conditional reimbursement policies such as England’s Cancer Drugs Fund. If a formal indication-based pricing system is not feasible, then existing methods should be refined to better capture the incremental value of individual indications.

Item Type:	Thesis (PhD)
Additional Information:	© 2023 Mackenzie John Mills
Library of Congress subject classification:	R Medicine > RA Public aspects of medicine
Sets:	Departments > Health Policy
Supervisor:	Kanavos, Panos and Costa-Font, Joan
URI:	http://etheses.lse.ac.uk/id/eprint/4696

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