284.26 - Designing Strategies for Neglected Disease Research

Meeting Time:
TuTh 8:35-9:50

Instructors:

Professor Stephen Maurer
302 Goldman School of Public Policy
510-642-6511
smaurer@berkeley.edu
Office hours: Thursdays 10 am - noon

Professor Amy Kapczynski
885 Simon Hall
510-642-1819
akapczynski@law.berkeley.edu
Office hours: Tuesdays 10am - noon

Course Description:
Of the 1393 new medicines developed between 1975 and 1999, only 16 were for tuberculosis and tropical diseases. Evidence shows that global R&D is overwhelmingly allocated to the medical needs of those living in wealthy countries.  Recently, significant public attention and financial resources have begun to be directed at the rest, the so-called “neglected diseases” (e.g. malaria, dengue fever) that disproportionately afflict the developing world. Even so, funds remain painfully limited. Success will require strategies that are careful, clear-eyed, and evidence-based. This course will introduce students to the neglected disease problem, review the various schemes that observers have proposed to promote R&D, and explore what the fields of law, economics, public policy, business, and public health have to say about each strategy’s strengths and weaknesses.

Syllabus:
To see the current Syllabus, click here.

Course Requirements:
Grades will be based on a midterm project (25%), final paper (50%) and class participation (25%).

Midterm Project:
Students will be assigned to teams for the midterm project, and asked to critically evaluate existing initiatives in the neglected diseases domain.  The assignment will be posted on the course website in the first week of class.  Midterm projects will be due Friday, March 6 (5pm).

Final Paper:
By 5:00 p.m. on April 16, teams of 4-6 students each must email to us a White Paper (balanced policy brief) analyzing some problem related to neglected disease R&D incentives (for example, the way forward for a specific disease or class of technologies, how to choose between alternative frameworks, etc.). This should be a well-written, thoughtful, well-referenced and researched, coherent analysis, 20-40 pages in length.  (Stop writing when you have nothing left to say on your topic!)  You should analyze your topic as if you were trying to help a policy maker understand and devise solutions for the problem.  The policy maker has his/her own values and politics, but s/he needs to know what the relevant facts and arguments are.  Try to provide this advice in a neutral way.  The goal is to unearth, analyze, and organize facts in a balanced way, providing perspectives and policy options.

Although each team can structure itself as it desires, our recommendation is that each team divide the topic into sub-topics (“chapters” of the report) with each team member (or a small group of team members) acting as the “lead author” for that portion.  (However, all team members should review and suggest changes for the entire report – the overall report must be smooth and integrated – not a “staple job” of 4-6 independent sections.)

Choosing topics. We will suggest a number of topics.  It’s fine for a team to modify a topic to suit its interests, or to devise a topic of its own.  It’s also fine for multiple teams to tackle the same topic.  We will review all topic choices, and provide guidance.

Milestones along the way:
Wednesday, March 18 (5pm): Topics must be selected by this date.  If you want to form a team on your own, send us a list of names and the topic by this date.  (This is by *far* the most desirable way to form a team – participate actively in the wiki process, gravitating naturally towards a topic and a team.)  If you have not organized a team on your own, you still must let us know by this date the topic you want to work on, via email to the instructors.  We will create teams from students who have selected similar topics by Friday March 20th.

Thursday April 16 (5pm): Final White Papers are due via email to the instructors.  Each team will jointly present its results during the last two class sessions.  Your grade will be based on the overall quality of the White Paper, your individual chapter / topic, and your presentation in class. Remember to indicate in your White Paper which team members primarily responsible for which topics and/or chapters.

Class Participation:
This course will have relatively little in-class discussion.  Instead, we have established an on-line wiki page for each lecture.  You should use this site to ask questions and discuss both the readings and lectures for the week (raising questions, offering critiques or extensions) with other students, the instructors, and guest lecturers.  Remember that 20% of your grade will depend on class participation.  The wiki, which provides a permanent record of comments, will be particularly salient in this regard. As always, thoughtful analysis will count for much more than quantity.

You can log on to each wiki page at any time. In general, however, we expect each page to become active on the day of the lecture and to complete discussion by the following weekend. Try to submit you initial postings for the week by Friday. This will allow the lecturer to log-on, answer questions, and otherwise interact with students over the next few days.

Beginning with the second lecture, we will ask each of you to prepare reporter’s notes for one session of the class.  We will circulate a sign-up sheet so that you can pick the lectures that most interest you (first come, first served).  Depending on class size, pairs of students can work together.  Your reporter’s notes should contain (a) a concise set of class notes, followed by (b) 3-5 one sentence statements summarizing the main points that a policymaker would want to know before thinking about R&D incentives.  Bear in mind that your notes will be the main source of information for the economists participating in our April 9 roundtable.  Please think carefully about what they would want to know.

You should post your reporter’s notes by midnight on the day of the lecture.  Timeliness counts, and will allow the other students to suggest comments and revisions.

Readings:
Readings are all hyperlinked to the course syllabus.  The only exception is the Kremer book (lecture 4B), which can be inexpensively purchased through Amazon, and which we will make available on reserve in the library. You may have to configure your browser to log in through UC Berkley’s proxy server to access some journal articles from off campus – instructions are here.  Let us know if you have trouble accessing anything.

Class Notes:
The students and instructors in this course have prepared notes for most of the lectures. Click here to download a copy. It is important to note that these notes have been prepared for convenience only and have not been reviewed by the various speakers whose presentations are summarized. The instructors are solely responsible for any errors or omissions.

Wiki:
If you are a UCB student enrolled in the Course, click here to reach our class discussion wiki. Other users are welcome to join by permission of the instructor. Please send your requests to smaurer@law.berkeley.edu.

Resources:
All of the assigned readings can be found on the course Syllabus. If you would like to see some additional resources, click here.

Class Schedule/Video Lecture Archive:

 Lecture Topic  Speaker  Readings  Resources 
1A
Jan. 13
Introduction; What Are "Neglected Diseases"? Why the World Needs a Rational, Evidence Based Strategy to Deal With Them. AK - Troullier et al., “Drug development for neglected diseases.” The Lancet 359:2188 (2002)
- Global Forum for Health Research, 10/90 Report on Global Health Research 2003-2004 Chapter 1.
- W. Fisher & T. Syed, Global Justice in Healthcare, 40 U.C. Davis L. Rev. 581 (2006)

Recommended:
- WHO Commission on Intellectual Property Rights, Innovation, and Public Health, Public Health, Innovation, and Intellectual Property Rights [especially pages 11-28]
- UK Commission on Intellectual Property Rights, Integrating Intellectual Property Rights and Development Policy, Chapter 2 (2001) [for more background on access issues and their relation to IP]
  PPT
 Video
1B
Jan. 15
Course Overview: Thinking Through the Problem. SM Same.  PPT
 Video
2A
Jan. 20
What Do We Know About Neglected Diseases? (what are the disease burdens) Kik Smith

- J. Breman et al., Conquering Malaria (2004)
- J. Remme et al., Tropical Diseases Targeted for Elimination: Chagas Disease, Lymphatic Filariasis, Onchocerciasis, and Leprosy (2004)
- Catand et al., Tropical Diseases Lacking Adequate Control Measures: Dengue, Leishmaniasis, and African Trypanosomiasis (2004)
- Abegunde et al,  The burden and costs of chronic diseases in low-income and middle-income countries, 370 Lancet 1929 (2007). 

Recommended:
- S. Bertozzi et al., HIV/AIDS Prevention and Treatment (2004)
- C. Dye and K. Floyd, Tuberculosis (2004)

 PPT
 Video
2B
Jan. 22
What Innovations Do We Need? (what drugs, diagnostics, vaccines, etc. would be most effective?) Julia Walsh Same.  PPT
 Video
3A
Jan. 27
What Do We Really Know About The Drug Discovery Business? David Ridley

- J. DiMasi, R. Hansen & H. Grabowski, “The Price of Innovation,” Journal of Health Economics 22:151 (2003).
- Global Alliance for TB Drug Development, The Economics of TB Drug Development, Chapter 3  (2001)
- Public Citizen, Rx R&D Myths: The Case Against The Drug Industry’s R&D "Scare Card" (2001)
- Light and Warburton, Extraordinary Claims Require Extraordinary Evidence, 24 Journal of Health Economics 1030 (2005)

Recommended:
- DiMasi et al reply to TB Alliance and Public Citizen
- DiMasi et al reply to Light and Warburton
- Resources collected by CPTech
- Christopher Adams and Van Brantner. 2008. “Spending on New Drug Development.” http://papers.ssrn.com/sol3/papers.cfm?
abstract_id=869765

- Joseph A. DiMasi, Ronald W. Hansen, and Henry G. Grabowski. 2007. “The Cost of Pharmaceutical R&D: Is Biotech Different?” Managerial and Decision Economics 28:469-479.
- Seth W. Glickman et al. 2006. “A Portfolio Model of Drug Development for Tuberculosis,” Science 311:246-247.
- Fabio A. Thiers, Anthony J. Sinskey and Ernst R. Berndt. 2008. "Trends in the Globalization of Clinical Trials." Nature Reviews: Drug Discovery. 7:13-14.
- Johan P.E. Karlberg. 2008. "Globalization of Sponsored clinical Trials." Nature Reviews: Drug Discovery. 7:458.
- Fabio A. Thiers, Anthony J. Sinskey and Ernst R. Berndt. 2008. "Globalization of Sponsored Clinical Trials: Reply ." Nature Reviews: Drug Discovery. 7:458.

 PPT
 Video
3B
Jan. 29
Introduction to Innovation Economics: Prizes, Competitive Bidding, Grants etc. SM - M. Kremer and R. Glennerster, Strong Medicine: Creating Incentives for Pharmaceutical Research on Neglected Diseases (Princeton: 2004), Chapters 5 & 6  PPT
 Video
4A
Feb. 3
Introduction to Innovation Economics: Price Discrimination and Access Pricing. SM

- J. Lanjouw, “A Patent Policy Proposal for Global Diseases,” (2001)
- J. Lanjouw, “Outline of the Foreign Filing License Approach” (2005)
- A. Taubman, Public-Private Management of Intellectual Property for Public Health Outcomes in the Developing World, Global Forum for Health Research (2004)

Recommended:
- Eiss et al, Ensuring Global Access Through Effective IP Management, MIHR Handbook of Best Practices 2008

 PPT
 Video
4B
Feb. 5
Drug Portfolio Management : How Well Can We Predict Success and Drugability? James McKerrow (UCSF) - Michelle Arkin & James McKerrow, "Low Hanging Fruit: An Anti-Parasitic Drug Database," Public Library of Science (2008).
- Adam R. Renslow & James H. McKerrow, "Drug Discovery and Development for Neglected Parasitic Diseases," Nature: Chemical Biology 2: 701-710 (2006).
- Solomon Nwaka & Robert G. Ridley, "Virtual Drug Discovery and Development for Neglected Diseases Through Private-Public Partnerships," Nature Reviews: Drug Discovery 919-928 (2003).
 PPT
5A
Feb. 10
Access Pricing AK Same.  PPT
5B
Feb. 12
Schemes and Proposals: Prizes and regulatory standards William Fisher TBA  Video
6A
Feb. 17
Schemes and Proposals: DEFEND (and Patent Pools) Keith Maskus

- K. Maskus, “Parallel Imports in Pharmaceuticals” (2001)
- M. Ganslandt, K. Maskus, and Eina V. Wong. 2001. “Developing and Distributing Essential Medicines to Poor Countries: The DEFEND Proposal.” World Economiy 24:779-795.
- John Barton. 2001. "Differentiated Pricing of Patented Products" Commission on Marcoeconomics and Health Working Paper.
- F.M. Scherer and Jayashree Watal. 2002. "Post-TRIPS Options for Access to Patented Medicines in Developing Nations." Journal of International Economic Law 913-939.

Recommended:
- MSF on UNITAID patent pool proposal
- KEI, Cost-Benefit Analysis of UNITAID Patent Pool.

 PPT
 Video
6B
Feb. 19
Schemes and Proposals: Financing Saul Walker - Mary Moran et al. Neglected Disease Research: How Much are We Really Spending? George Institute 2008  PPT
 Video
7A
Feb. 24
Schemes and Proposals: The Health Impact Fund Aidan Hollis

- Aidan Hollis, The Health Impact Fund: A Useful Supplement to the Patent System? Public Health Ethics, May 2008.
- Summary of the Health Impact Fund

Recommended:
- Hollis & Pogge, The Health Impact Fund: Making Medicines Available to All (book-length explanation)

 

 PPT
 Video
7B
Feb. 26
Schemes and Proposals: Funding Clinical Trials Dean Baker - D. Baker, The Benefits and  Savings from Publicly Funded Clinical Trials of Prescription Drugs, CEPR (Mar. 2008)
- T. Lewis, J. Reichman & A. So,  The Case for Public Funding and Public Oversight of Clinical Trials, Economists’ Voice (Jan 2007)
- Arjun Jayadev and Joseph Stiglitz, Two Ideas to Increase Innovation and Reduce Pharmaceutical Costs and Prices, Health Affairs (Dec. 2008)
 PPT
 Video
8A
Mar. 3
Schemes and Proposals: Advanced Market Commitments Michael Kremer - Advanced Markets Working Group, “Making Markets for Vaccines” (2004)
Recommended:
- A complete set of Prof. Kremer’s work on vaccine prizes can be found at http://www.economics.harvard.edu/faculty/
kremer/vacine_research_kremer
.
 Video
8B
Mar. 5
  Zakir Thomas Class Cancelled Due to Technical Difficulties. Will Be Rescheduled as a Special Evening Session of the Class.  
9A
Mar. 10
Schemes and Proposals: PDPs Suerie Moon - K. Buse and A. Harmer, “Seven Habits of Highly Effective Private-Public Partnerships,” Social Science and Medicine 64:259 (2006)
- M. Moran et al., The New Landscape of Neglected Disease Drug Development (2005)
- T. J. Tucker and M. Makgoba, “Public Private Partnerships and Scientific Imperialism,” Science 320:1016 (2008).
 PPT
 Video
9B
Mar. 12
Regulatory Approval David Kessler

- Glickman et al., Globalization of Clinical Research, New England Journal of Medicine 360:816 (2009).
- Peter Folb, Tackling the critical question of drug regulation, DNDi Newsletter
- Workshop on Clinical Trials Capacity in Low- and Middle-Income Countries: Experiences, Lessons Learned and Priorities for Strengthening, Initiative for Public-Private Partnerships on Health
- KEI comments on the priority review voucher
- IAVI comments on PRV

Recommended:
- Trouiller et al., Is Orphan Drug Status Beneficial to Tropical Disease Control? Comparison of the American and Future European Orphan Drug Acts, 4 Trop. Med. & Int’l Health 12 (1999)
- Ridley et. al, Developing Drugs for Developing Countries, 25 Health Affairs 313 (2006) (proposing priority review vouchers)

 Video
10A
Mar. 17
Innovation Economics 3: Beyond the Ivory Tower: Making Practical Choices) SM - S. Maurer, "Choosing the Right Incentive Strategy for R&D in Neglected Diseases," (2006).
- A. Mahmoud, P Danzon, J. H. Barton & R.D. Mugerwa, “Product Development Priorities” (2004)

Recommended:
- S. Maurer, "The Right Tool(s): Designing Cost-Effective Strategies for Neglected Disease Research," (2005).
 PPT
 Video
10B
Mar. 19
Political Economy: Foundations, NGOs, WHO, USG. James Love

- Bart Wijnberg & Marleen Monster, Innovation and access: medicines for the poor – the IGWG strategy and plan of action,  Global Forum Update on Research for Health Volume 5 (2008)

Recommended:
- Families USA, The World Can’t Wait: More Funding Needed for Neglected Diseases (2008) (providing data on US gov. R&D for neglected diseases in 2007)

 
11A
Mar. 31
Class Discussion: Designing R&D Strategies SM, AK --  
11B
Apr. 2
Class Discussion: Designing R&D Strategies (ctd.) SM, AK --  
12A
Apr. 7
Solomon Nwaka: The Neglected Disease Pipeline SM See readings under 4B.  
12B
Apr. 9
Innovation Economics 5: Economist Roundtable Brian Wright (UCB), Suzanne Scotchmer (UCB), and Nisvan Erkal (Melbourne) --  
13A
Apr. 14
Delivering Medicines to Patients: The Leaky Pipeline. NGOs, Developing World Health Systems, Hospitals, National Health Accounts, etc. Julia Walsh - S. Tollman, J. Doherty and J. Mulligan, “General Primary Care” (2004)  PPT
13B
Apr. 16
Private Sector Health Care Delivery in the Developing World Nicole Prata    
14A
Apr. 21
Student Papers SM, AK    
14B
Apr. 23
Student Papers; Concluding Remarks SM, AK    

 

Coming Soon: Complete Video of All Class Lectures and Slides