Development of drugs in  public-private partnership (PPP) environments Marija Mavar Haramija / Nathaniel Stanley / Pascal ...
Agenda 15-min slides + 10-min debate <ul><ul><li>Introduction </li></ul></ul><ul><ul><li>The Old Method </li></ul></ul><ul...
Introduction <ul><ul><li>Process of introducing new drugs in the market is expensive, long, and risky </li></ul></ul><ul><...
The Old Method A long and costly process <ul><ul><li>Traditional drug discovery usually involves exclusively private secto...
The Old Method A long and costly process
The Old Method A long and costly process <ul><ul><li>&quot;Slowing pace of new drugs coming out of the [US] pharmaceutical...
Orphan Diseases Definition and some facts <ul><ul><li>&quot;orphan&quot; or &quot;neglected&quot; or &quot;tropical diseas...
Orphan Diseases The Challenges <ul><ul><li>80% of rare diseases have identified genetic origins </li></ul></ul><ul><ul><ul...
Orphan Diseases Some examples <ul><li>Malaria ( ORPHA673, Prev.: 1-9 / 100 000 ) </li></ul><ul><ul><li>247 million cases /...
Public-Private Partnerships a complementary collaboration <ul><li>Public sector </li></ul><ul><ul><li>has a great interest...
Public-Private Partnerships Structure <ul><ul><li>Funding </li></ul></ul><ul><ul><ul><li>private sources, government, inte...
Public-Private Partnerships Advantages <ul><ul><li>reduces cost on technology, staff, access to compound libraries, etc. <...
Public-Private Partnerships Disadvantages <ul><ul><li>Overhead of having distributed workforce </li></ul></ul><ul><ul><li>...
Public-Private Partnerships Issues <ul><ul><li>duplication of efforts among teams </li></ul></ul><ul><ul><li>&quot;many PP...
Example of PPP  Bayer's initiative: Grants4Targets <ul><li>Grants4Targets allows public researchers to apply for research ...
Example of PPP  Bayer's initiative: Grants4Targets <ul><li>Advantages: </li></ul><ul><ul><li>Works well for very early sta...
Debate <ul><ul><li>How do you feel about this model? Are you convinced that this kind of partnership will be successful?  ...
References some additional reading  <ul><li>Nwaka S, Ridley R. Virtual drug discovery and development for neglected diseas...
<ul><li>  </li></ul><ul><li>  </li></ul><ul><li>Backup slides </li></ul>
New Drugs market entry by disorder and primary indication Source:  Christopher P. Adams and Van V. Brantner. Estimating Th...
The example of Malaria WHO Malaria Report 2008 Estimated incidence of malaria per 1000 population, 2006
Grants 4 Targets Distribution of applications received National distribution of applications received, (RoW, Rest of the W...
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Development of drugs in public-private partnership (PPP) environments

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Development of drugs in public-private partnership (PPP) environments

  1. 1. Development of drugs in  public-private partnership (PPP) environments Marija Mavar Haramija / Nathaniel Stanley / Pascal Maugeri April 2011   MSc Bioinfo - Computer Assisted Drug Discovery
  2. 2. Agenda 15-min slides + 10-min debate <ul><ul><li>Introduction </li></ul></ul><ul><ul><li>The Old Method </li></ul></ul><ul><ul><li>The Challenges of Orphan Diseases </li></ul></ul><ul><ul><li>The Public-Private Partnership model </li></ul></ul><ul><ul><li>Debate </li></ul></ul><ul><ul><li>References </li></ul></ul>
  3. 3. Introduction <ul><ul><li>Process of introducing new drugs in the market is expensive, long, and risky </li></ul></ul><ul><ul><li>Drug makers only have incentive to introduce drugs to markets where customers can pay development costs </li></ul></ul><ul><ul><li>Most of the resources and experience in drug discovery are held by the private sector </li></ul></ul><ul><ul><li>90% of the world's diseases receive only 10% of the resources, leading many of those diseases neglected, or &quot;orphaned&quot; </li></ul></ul><ul><ul><li>Public-Private partnerships (PPPs) could allow for some of these diseases to receive the attention they need while. </li></ul></ul>
  4. 4. The Old Method A long and costly process <ul><ul><li>Traditional drug discovery usually involves exclusively private sector companies. Occasionally they work together, but this is a &quot;simple&quot; process for them. </li></ul></ul><ul><li>  </li></ul><ul><ul><li>The process of introducing a new drug in the market is usually 10-15 years long </li></ul></ul><ul><li>  </li></ul><ul><ul><li>Huge investment required: average is $868 million but the range is $500-$2.000 million depending on company strategy and therapy targeted   </li></ul></ul>
  5. 5. The Old Method A long and costly process
  6. 6. The Old Method A long and costly process <ul><ul><li>&quot;Slowing pace of new drugs coming out of the [US] pharmaceutical industry&quot; (New York Times, Jan. 22, 2011) </li></ul></ul><ul><ul><li>Large drug makers are reducing their R&D spending </li></ul></ul><ul><ul><li>Waves of acquisitions of small biotech companies to temporarily fill in the gap of patents expiry </li></ul></ul><ul><ul><li>Dubious tactics focused on taking advantage of the legal/patent system and drug approval process to extend monopolies on drugs (CBS news article, 12 Jul. 2001) </li></ul></ul>
  7. 7. Orphan Diseases Definition and some facts <ul><ul><li>&quot;orphan&quot; or &quot;neglected&quot; or &quot;tropical diseases&quot; or &quot;diseases of poor&quot; </li></ul></ul><ul><li>  </li></ul><ul><ul><li>A disease which has not been &quot;adopted&quot; by the pharmaceutical industry because it provides little financial incentive for the private sector to make and market new medications to treat or prevent it. </li></ul></ul><ul><li>  </li></ul><ul><ul><li>Diseases that: </li></ul></ul><ul><ul><ul><li>affect small number of patients in developed countries </li></ul></ul></ul><ul><ul><ul><li>affect many patients in developing countries, but because they are unable to pay, drug developers don't invest in that market </li></ul></ul></ul><ul><ul><li>Orphan Diseases = Rare Diseases + Neglected Diseases (EURORDIS) </li></ul></ul><ul><ul><li>EURORDIS estimates the existence of 5000-7000 Orphan Diseases and Orphanet references 5954 diseases (April 2011) </li></ul></ul>
  8. 8. Orphan Diseases The Challenges <ul><ul><li>80% of rare diseases have identified genetic origins </li></ul></ul><ul><ul><ul><li>Cystic fibrosis, Turner sindrome, Huntington disease, muscular distrophy  </li></ul></ul></ul><ul><li>  </li></ul><ul><ul><li>most of neglected diseases are tropical infections  </li></ul></ul><ul><ul><ul><li>  trypanosomal parasites (African sleeping sickness..), bacterial and viral infections (cholera, yellow fever...) </li></ul></ul></ul><ul><ul><li>only 10% of global R&D resources are directed at diseases accounting for 90% of the global disease burden </li></ul></ul><ul><ul><li>existing regulatory incentives (tax incentives, market exclusivity...) may no longer apply </li></ul></ul><ul><li>  </li></ul>
  9. 9. Orphan Diseases Some examples <ul><li>Malaria ( ORPHA673, Prev.: 1-9 / 100 000 ) </li></ul><ul><ul><li>247 million cases / year according to WHO </li></ul></ul><ul><ul><li>881 000 deaths  in 2006  and could double in the next 20 years, 91% were in Africa and 85% were of children under 5 years of age </li></ul></ul><ul><ul><li>&quot;In the case of malaria, the immediate problem is drug resistance&quot; </li></ul></ul><ul><li>Tuberculosis ( ORPHA3389, Prev.: 1-5 / 10 000 ) </li></ul><ul><ul><li>world wide distribution is not uniform: 80% of the population in many Asian and African countries, 5–10% of the US population </li></ul></ul><ul><ul><li>2007: 13.7 million chronic active cases, 9.3 million new cases, and 1.8 million deaths , mostly in developing countries </li></ul></ul><ul><li>Cholera ( ORPHA173,  Prev.:  1-9 / 1 000 000) </li></ul><ul><ul><li>2010: affects 3-5 million people and causes 100,000-130,000 deaths a year </li></ul></ul><ul><ul><li>one of the earliest infections to be studied by epidemiological methods. </li></ul></ul>
  10. 10. Public-Private Partnerships a complementary collaboration <ul><li>Public sector </li></ul><ul><ul><li>has a great interest in stimulating discoveries </li></ul></ul><ul><ul><li>knowledge of multiple diseases </li></ul></ul><ul><ul><li>links to governments </li></ul></ul><ul><ul><li>strong networks in disease-endemic countries </li></ul></ul><ul><ul><li>has not developed its own drug development infrastructure </li></ul></ul><ul><li>  </li></ul><ul><li>Private sector </li></ul><ul><ul><li>strong R&D expertise, marketing, PM, and regulatory affairs </li></ul></ul><ul><ul><li>seeks new therapies by converting basic scientific discoveries </li></ul></ul><ul><ul><li>development of biotech companies brought high degree of specialization </li></ul></ul><ul><ul><li>has disengaged since '70s from tropical diseases R&D. </li></ul></ul><ul><ul><li>&quot;a culture of controlling R&D collaboration&quot; </li></ul></ul>
  11. 11. Public-Private Partnerships Structure <ul><ul><li>Funding </li></ul></ul><ul><ul><ul><li>private sources, government, international, philantropic and charitable foundations </li></ul></ul></ul><ul><ul><li>Goals </li></ul></ul><ul><ul><ul><li>discovery and development of new drugs for orphan diseases </li></ul></ul></ul><ul><ul><ul><li>improvement of characteristics of existing drugs (to achieve low cost, easy administration, quick effect, to overcome resistance...) </li></ul></ul></ul><ul><ul><li>Large organizations </li></ul></ul><ul><ul><ul><li>UNDP/World Bank/UNICEF/WHO Special Programme for Research and Training in Tropical Diseases (TDR) </li></ul></ul></ul><ul><ul><li>Small organizations </li></ul></ul><ul><ul><ul><li>  Medicines for Malaria Venture (MMV) </li></ul></ul></ul><ul><ul><ul><li>  Global Alliance for Tuberculosis Drug Development (GATB) </li></ul></ul></ul><ul><ul><ul><li>  Drugs for Neglected Diseases Initiative (DNDi) </li></ul></ul></ul><ul><ul><ul><li>  Institute for One World Health (IOWH)  </li></ul></ul></ul>
  12. 12. Public-Private Partnerships Advantages <ul><ul><li>reduces cost on technology, staff, access to compound libraries, etc. </li></ul></ul><ul><ul><li>expertise can be shared </li></ul></ul><ul><ul><li>&quot;The administration of a portfolio of projects offers advantages both scientifically and administratively&quot; </li></ul></ul><ul><ul><li>flexibility: projects may be started at facilities (university and/or company) that have the knowledge or materials </li></ul></ul><ul><ul><li>develop long-term relationships between entities </li></ul></ul>
  13. 13. Public-Private Partnerships Disadvantages <ul><ul><li>Overhead of having distributed workforce </li></ul></ul><ul><ul><li>Strong management is required: not easy to manage projects in different locations across different cultures and timezone </li></ul></ul><ul><ul><li>Potential for lawsuits over intellectual property the further along partnerships go, with which private companies have much more experience </li></ul></ul>
  14. 14. Public-Private Partnerships Issues <ul><ul><li>duplication of efforts among teams </li></ul></ul><ul><ul><li>&quot;many PPPs are still in their infancy and are themselves social experiments&quot; </li></ul></ul><ul><ul><li>&quot;scientists in developing countries do not want to be just ‘testers and developers’ of other people’s products.&quot; </li></ul></ul><ul><ul><li>&quot;To ensure the long-term sustainability of these programmes, greater involvement of disease-endemic countries has to be built into the PPP model&quot; </li></ul></ul><ul><ul><li>unclear who owns Intellectual Property Rights </li></ul></ul>
  15. 15. Example of PPP  Bayer's initiative: Grants4Targets <ul><li>Grants4Targets allows public researchers to apply for research grants from Bayer Healthcare </li></ul><ul><ul><li>Applicants apply for grants via the Grants4Targets website using a standard format application. </li></ul></ul><ul><ul><li>A committee of researchers at Bayer then decide on the proposal </li></ul></ul><ul><ul><li>Grants are decide in as little as 8 weeks </li></ul></ul><ul><ul><li>Grants come in three sizes, ranging from €5K-250K. Up to €125K, no IP discussions are necessary, applicant keeps IP. </li></ul></ul>
  16. 16. Example of PPP  Bayer's initiative: Grants4Targets <ul><li>Advantages: </li></ul><ul><ul><li>Works well for very early stage development </li></ul></ul><ul><ul><li>Low bureaucratic hurdles </li></ul></ul><ul><ul><li>Not very complex and &quot;no-strings-attached&quot; IP rights </li></ul></ul><ul><ul><li>Potential for future collaboration </li></ul></ul><ul><li>Disadvantages: </li></ul><ul><ul><li>No strong collaboration </li></ul></ul><ul><ul><li>Over 70% of requested grants are for Cancer or Heart disease, NOT orphan diseases </li></ul></ul><ul><ul><li>Only applicable to the very first stages of development </li></ul></ul>Snapshot of Grants4Targets project  after 3 rounds By percentage, in what fields grants have been requested
  17. 17. Debate <ul><ul><li>How do you feel about this model? Are you convinced that this kind of partnership will be successful? </li></ul></ul><ul><ul><li>Is private industry benefitting more than they are contributing, or vice versa? </li></ul></ul><ul><ul><li>Do you see any potential problems that could come from disputes over intellectual property rights? How are we certain drugs will be made available for a reasonable price to those in need? </li></ul></ul>
  18. 18. References some additional reading  <ul><li>Nwaka S, Ridley R. Virtual drug discovery and development for neglected diseases through public-private partnerships. Nat Rev Drug Discov. 2003:2. </li></ul><ul><li>Christopher P. Adams and Van V. Brantner. Estimating The Cost Of New Drug Development: Is It Really $802 Million?  Health Affairs, 25, no.2 (2006):420-428 </li></ul><ul><li>Monika Lessl et al. Grants4Targets – an innovative approach to translate ideas from basic research into novel drugs. Drug Discovery Today . Volume 16, Numbers 7/8, April 2011 </li></ul><ul><li>Harrris, Gardiner. &quot;Federal Research Center Will Help Develop Medicines&quot;  The New York Times . 22 Jan. 2011. </li></ul><ul><li>http://www.nytimes.com/2011/01/23/health/policy/23drug.html </li></ul><ul><li>&quot; Why Do Generic Prescription Drugs Take So Long to Hit the Market?&quot; CBS News . 12 Jul 2001. </li></ul><ul><li>http://www.cbsnews.com/stories/2002/01/31/health/main327667.shtml </li></ul><ul><li>World Health Organization. World Malaria Report. 2008 http://whqlibdoc.who.int/publications/2008/9789241563697_eng.pdf </li></ul>
  19. 19. <ul><li>  </li></ul><ul><li>  </li></ul><ul><li>Backup slides </li></ul>
  20. 20. New Drugs market entry by disorder and primary indication Source:  Christopher P. Adams and Van V. Brantner. Estimating The Cost Of New Drug Development: Is It Really $802 Million? Health Affairs, 25, no.2 (2006):420-428
  21. 21. The example of Malaria WHO Malaria Report 2008 Estimated incidence of malaria per 1000 population, 2006
  22. 22. Grants 4 Targets Distribution of applications received National distribution of applications received, (RoW, Rest of the World)

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