Assessing the Public and Philanthropic Financial Contribution to the Development of New Drugs: A Bibliographic Analysis

Claudia Wild,Louise Schmidt

doi:10.11648/j.stpp.20200401.12

Abstract

Background: There is wide debate on the cost of some pharmaceutical products and the impact this has on access to medicine. Little publicized knowledge on the public and philanthropic contribution to research and development costs exists so far. The objective of work reported here was to collect information on public contributions to research funding and thus contribute to the discussion on return on public investment. Methods: A multi-level search process was developed to search for public and philanthropic research funding based on 3 main steps: (1) identification of all generic and molecular names and terms, (2) systematic search for pre-marketing pathway information and related research funding, (3) systematic search for corresponding research funding amounts. Three Paediatric Orphan Drugs (Spinraza®, Brineura®, Crysvita®), which were approved by the European Medicines Agency (EMA) in 2017, were chosen to pilot the methods. Results: We estimated that public/philanthropic contributions to funding of product-related research ranged between approximately € 20 million (Spinraza®) and € 31 million (Brineura®). However, this is a very conservative estimate since pharmaceutical development calls upon basic research, which does not mention product-specific terms. For instance, for research into SMA as a whole, public and philanthropic research funding contributions totalling € 165 million were identified. Conclusions: Researching public and philanthropic R & D funding proved to be difficult and time consuming. Further piloting including the refinement and standardisation of the search strategy is underway.

Highlights

Up until recently the term “access to medicines” was mostly associated with discussions concerning the ability of developing countries to access cheaper, life-saving generic medicines to treat infectious diseases such as HIV and tuberculosis
An analysis by Global Justice reported it is estimated that the public pays for two-thirds of all upfront R&D drug costs and that around one-third of all medicines originate in research institutions in the public sector [3]
Paediatric-relevant products in the area of orphan medicine were chosen. 35 new active substances were recommended for approval by European Medicines Agency (EMA) in 2017, 13 of which were orphan products and four of these were declared by the EMA to be medicines for children representing an outstanding contribution to public health, three of which - Spinraza®, Brineura®, and Crysvita® - were selected for the analysis

Summary

Introduction

Up until recently the term “access to medicines” was mostly associated with discussions concerning the ability of developing countries to access cheaper, life-saving generic medicines to treat infectious diseases such as HIV and tuberculosis. In a recent seminal piece of work, Cleary and co-authors sought to establish the contribution of NIH funding to published research associated with 210 new molecular entities [11]. Their results suggest an enormous spend of over $100 billion between 2000 and 2016 representing around 20% of the total NIH budget over this period [11]. Results: We estimated that public/philanthropic contributions to funding of product-related research ranged between approximately € 20 million (Spinraza®) and € 31 million (Brineura®) This is a very conservative estimate since pharmaceutical development calls upon basic research, which does not mention product-specific terms. Further piloting including the refinement and standardisation of the search strategy is underway

Objectives

Methods

Results

Conclusion