An Ethical Pathway to register pharmaceutical & biological products

1. An Ethical Pathway to register pharmaceutical & biological products Judit Rius Sanjuan Knowledge Ecology International NLARx Winter meeting Washington, DC - January 21, 2011

2. Outline • Problem - Current regulatory regimes in the protection of clinical test data in the United States create exclusive rights and monopolies that not only have a negative effect on prices/cost and competition but also force the unethical duplication of clinical trials in humans and animals. • Proposal – Senator Sanders introduced S. 3921 to the 111th Congress, the Ethical Pathway Act of 2010 - A bill to ensure that rules for the approval of pharmaceutical and biological products do not require violations of medical ethics in the testing of products in humans and vertebrate animals.

3. Data Exclusivity • The originator of a new drug that submits regulatory test data to the FDA to prove the safety and efficacy of the product is granted a protection precluding for a prescribed period of time the approval or delaying the submission of second and subsequent applications (generics) that rely in the originator’s regulatory test data.

4. Barriers to generic competition • Pharmaceutical Products – 5/3 years of data exclusivity of Hatch-Waxman Act of 1984 • Biological Products – 12 years of data exclusivity of Biologics Price Competition and Innovation Act (BPCI Act) of 2009 • Co-exist with other “data/ marketing exclusivity” regimes: • Orphan drug exclusivity • Pediatric drug exclusivity • Generic drug exclusivity (180 days) • Medical devices exclusivity • Patent protection (and term extensions)

5. Pharmaceutical Products Two main regimes: • 5-years exclusivity: for drugs with New Chemical Entities • Effects: no generic applications can be submitted • 3-years exclusivity: for drugs without NCE but with “other significant changes”, such as a new use/indications, for which the sponsor conducted or sponsored new clinical investigations that were essential to approval of the application. • Effects: generic applications can be submitted but their approval is suspended

6. Biological Products • 2009 Biologics Price Competition and Innovation Act (BPCI Act) Exclusivity • Generic application can not be submitted until 4 years since originator product was approved • Approval of a generic application can not be made effective after 12 years since originator product was approved

7. Paths for generic entry • Wait until data exclusivity expire; or • Obtain authorization of originator to use/rely on test data; or • Present own data on already demonstrated safe and effective products and therefore some times unnecessary forcing  duplication of clinical data and testing on human beings and animals. • Proposal: + Sanders Ethical Pathway

8. Helsinki Declaration on Ethical Principles For Medical Research Involving Human Subjects • 20. Physicians may not participate in a research study involving human subjects unless they are confident that the risks involved have been adequately assessed and can be satisfactorily managed. Physicians must immediately stop a study when the risks are found to outweigh the potential benefits or when there is conclusive proof of positive and beneficial results. • 21. Medical research involving human subjects may only be conducted if the importance of the objective outweighs the inherent risks and burdens to the research subjects.

9. Proposal: Ethical Pathway • Senator Sanders Bill S. 3921 The Ethical Pathway Act of 2010 To ensure that rules for the approval of pharmaceutical and biological products do not require violations of medical ethics in the testing of products in humans and vertebrate animals.

10. Proposal: Ethical Pathway Create an ethical pathway in the registration of pharmaceutical and biological products when the repetition of a clinical would violate medical ethics Pathway: • Companies would be allowed to rely upon existing test data when applying for marketing approval. • If the test data was still under the effective term of a regime of protection, the reliance would be subject to a cost sharing agreement.

11. A cost-sharing agreement The applicant would be required to pay a “reasonable and fair fee” to the entity that bore the costs of producing the relied-upon data or the rights holder. (1) The holder of the rights over the data and the applicant voluntarily negotiate a reasonable and fair fee and authorize reliance upon the data. (2) If either party failed to voluntarily negotiate, or such negotiation failed to produce an agreement, the holder and applicant would be referred to binding arbitration by the FDA Commissioner to determine a reasonable and fair fee for reliance upon the data. (3) If either party refuses to participate in binding arbitration, the FDA Commissioner would determine a reasonable and fair fee.

12. Increasing R&D transparency • For transparency and to make the system more predictable for rights holders and applicants, rights holders would be required to disclose the costs of generating the test data, and the cost- sharing payments by the applicants would be made public.

13. Why is relevant for States Legislators? • The proposal would eliminate data exclusivity in most cases, in the United States, accelerating generic entrance into the U.S. market and therefore have a positive impact on the cost of health care products.

14. What’s next • November 2010 - FDA Hearing & Senator Sander letter to FDA • 2011 Ethical Pathway bill – Support!

15. More Information: Contact: Senator Sanders Office & Knowledge Ecology International http://www.keionline.org