ADDITIONAL CLINICAL BENEFIT FOR PATIENT RELEVANT ENDPOINTS- INVESTMENT DISINCENTIVES BY AMNOG IN THE EXAMPLE OF PARKINSON'S DISEASE

Author(s)

Walzer S*1, Holbein B2 1MArS Market Access & Pricing Strategy UG (h.b.), Weil am Rhein, Germany, 2University of Bremen, Bremen, Germany

OBJECTIVES: After the introduction of the new German law AMNOG a statistically significant and clinically meaningful difference in patient relevant endpoints are required in order to achieve a (significant) clinical benefit for a new (drug) therapy. The question remains if such an incentive is sufficient in all disease areas for positive investment decisions for the pharmaceutical industry. METHODS: In Parkinson’s disease motor fluctuations and dyskinesias are two major patient relevant outcomes. Different target profiles for a clinical development programme of a theoretical new treatment were developed including scenarios following the regulatory guidelines only, an efficacy-driven programme and one including the above mentioned key patient relevant endpoints. An investment decision model was developed based on these profiles with respect to additional clinical benefit by the joint federal committee and premium pricing in Germany were assessed. RESULTS: Positive investment decisions were possible for the regulatory and efficacy-driven scenarios even though that the additional clinical benefit profile was uncertain and pricing not optimal. When developing potential budget estimates based on sample size and complexity of a clinical trial for motor fluctuations or dyskinesias as primary endpoint a significant clinical benefit was highly likely and premium pricing possible. However, a positive investment decision was uncertain due to the high development cost. CONCLUSIONS: Even though there are incentives to add patient relevant endpoints in the development programmes the return on investment could potentially not be high enough for a positive investment decision. Target profiling including market access and pricing outcomes could optimize investment decisions.

Conference/Value in Health Info

2013-11, ISPOR Europe 2013, The Convention Centre Dublin

Value in Health, Vol. 16, No. 7 (November 2013)

Code

PND54

Topic

Health Policy & Regulatory

Topic Subcategory

Coverage with Evidence Development & Adaptive Pathways

Disease

Multiple Diseases, Neurological Disorders

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