Advanced Therapy Medicinal Products (ATMP) is a new class of drugs in the EU that includes medical products using gene therapy, cell therapy or tissue engineering. Several of these therapies can be expected to lead to elimination of symptoms or disease activity in severely ill patients and thus create long-lasting positive effects on both health and healthcare costs. In the near future, a breakthrough is expected for ATMP. The purpose of this study is to prepare proposals for new methods for analyzing the value of curative drug treatments, and to give suggestions for financing and payment for curative therapies in a health care system characterized by public budgets.
Current technology for calculating the gain in terms of increased number of life-years is based on an estimate of a survival function based on survival data from the clinical trial. This technique is not suitable for curative therapies because the proportion of patients who have been cured can be expected to return to the same risk of death as the general population. Graphically, this means that the survival curve reaches a plateau instead of speeding towards zero.
Current techniques for evaluating the effects of a new treatment are based on an estimate of the number of discounted quality-adjusted life-years (QALYs). The ATMP is associated with attributes that are not always captured by the QALY measure, such as the value of cure, possible spread effects of the technology and the value of providing hope for the patients. ATMP is also associated with significant uncertainty as to which alternative treatments will be available in the future. This indicates that the discount rate from ATMP should be higher than the interest rates normally used in healthcare.
The current payment model for new drugs is based on the payment being made during the time that the treatment is given. For curative therapies, this means that payment for large values realized over a long period of time must be paid during a short period of time. This can lead to affordability issues, where a drug can be cost-effective but not possible to pay for with the current payment model. ATMP also causes considerable uncertainty regarding what one pays for, as there may be limited possibilities for making full-fledged RCTs and the approval often takes place at an early stage while full effect can only be measured after several years. In addition, the incentives for the introduction of ATMP can be affected by the fact that the region paying for the treatment cannot ensure that they receive healthcare savings because the patient can move to another region.
Our proposal to create a more appropriate valuation of ATMP consists of (i) introducing a risk elimination premium for curative therapies, i.e. a higher cost per QALY gained can be accepted when the treatment leads to cure, (ii) estimating the long-term health effects with a so-called plateau model or mixed cure model, (iii) apply higher discount rates for curative ATMP treatments than for continuous cure treatments.
Our proposal to create a more efficient payment model for ATMP consists of developing (i) annuity payment with outcome-based agreement for the treatment of small populations or treatment of the incidence, (ii) flexible state or regional budgets for financing treatment of large populations or treatment of prevalence, and (iii) health and medical loans as an alternative to annuity payments or flexible budgets.
IHE Rapport 2019:1, IHE: Lund, Sverige