TLV presents proposal to the Swedish government to facilitate pricing negotiations for new gene therapies
The TLV, the agency in charge of health economic evaluations in Sweden, recently presented to the Swedish government its proposal detailing how a reform could positively influence patient access to new gene therapies. The proposal focuses on reducing the risk of paying more for a gene therapy than its additional benefit is worth. The TLV proposes to manage risk through economic modelling and risk-adjusted ICERs, as well as volume-based pricing, and the inclusion of relatives’ QoL in the assessment. The TLV hopes to increase its involvement in suggesting outcomes-based agreements, that will facilitate negotiations between the regions and manufacturers to increase patient access to these high-cost, one-time therapies. Details have not yet been made available on how this approach to access for gene therapies will differ for other ATMPs.
Gene therapies and the need for cost-effectiveness
- Gene therapies differ from conventional therapies, often being a one-time treatment with significantly long-term benefits over a number of years. This means that the duration of effect for gene therapies is vital in showing cost-effectiveness in the long term
- ICERs that are weighted against the probability that the additional benefit will be delivered to the patient over a sustained period of time, and can help payers to manage their budgets and mitigate potential risk in reimbursing therapies with high degrees of uncertainty
- The TLV has proposed that it should be granted authority to support county councils in defining the best OBA which would aid negotiations between the regions and manufacturers, therefore facilitating patient access. This would be tested specifically for diseases where the size of the payment to the manufacturer depends on how large the health benefit is likely to be
Economic models for risk reduction
- Due to the uncertainty associated with gene therapies, it is currently challenging for both payers and companies to evaluate the financial implications of choosing a certain payment model
- Economic modelling can estimate the level of risk reduction between payment models
- The TLV is developing a simulation tool to be used by regions to better understand the effects of various types of OBAs
Should the cost of a therapy be influenced by volume of use?
- Currently, gene therapies are high-cost treatments that target rare diseases. Limited certainty in their efficacy at launch, as well as high costs as a one-time treatment, limit the ability of some gene therapies to demonstrate cost-effectiveness.
- In order to not limit access to these therapies the TLV has often accepted ICERs with higher-than-usual prices paid per unit of health gain
- The TLV has proposed to look in-depth at how volume of use should be allowed to affect price, for drugs aimed at any size of patient population
Economic models for risk reduction
- Due to the uncertainty associated with gene therapies, it is currently challenging for both payers and companies to evaluate the financial implications of choosing a certain payment model
- Economic modelling can estimate the level of risk reduction between payment models
- The TLV is developing a simulation tool to be used by regions to better understand the effects of various types of OBAs
Should the cost of a therapy be influenced by volume of use?
- Currently, gene therapies are high-cost treatments that target rare diseases. Limited certainty in their efficacy at launch, as well as high costs as a one-time treatment, limit the ability of some gene therapies to demonstrate cost-effectiveness.
- In order to not limit access to these therapies the TLV has often accepted ICERs with higher-than-usual prices paid per unit of health gain
- The TLV has proposed to look in-depth at how volume of use should be allowed to affect price, for drugs aimed at any size of patient population
Relatives QoL to be considered in pricing?
- Many conditions have a significant impact on the relatives’ QoL. To date, this has not been taken into account by the TLV when considering pricing negotiations for therapies, due to the lack of sufficient high-quality data and a standard method of data collection
- The TLV believe that in certain situations it is reasonable to consider these effects on QoL, as the availability of treatments could have off-target effects by increasing the QoL of the patient’s relatives. This will likely to be taken into consideration by the TLV when manufacturers are able to demonstrate a clear improvement between the therapy and the relatives’ QoL, by improving the patient’s health condition
ICER: Incremental Cost-Effectiveness Ratio; OBA: Outcomes-Based Agreement; QALY: Quality-Adjusted Life Year; QoL: Quality of Life; TLV: Dental and Pharmaceutical Benefits Agency
Article published 12 May 2022.
