1. Patients access to investigational drugs Steinar Aamdal Dept. of Clinical Cancer Research Oslo University Hospital

2. Cancer treatment Most standard treatments for patients with advanced solid cancer fail sooner or later Failing standard therapy many patients opt for investigational therapies Investigational therapies? Drugs in development, being tested Phase I or Phase II trials Phase I: First trial in patients of a new therapy (different tumor types) Phase II: Trial in one particular tumor type Which patients ? Patients in good general condition Younger patients

3. 3 Benefits of Participating in Clinical Trials Early access to new treatments Active role in own health care Participation in advancing medical knowledge

4. 4 Barriers to Participation in Clinical Trials Physicians` and patients` unawareness of clinical trials Lack of access to trials

5. Awareness of Investigational therapy trials in the Nordics In Norway major hospitals may publish their trials on websites www.oslo-universitetssykehus.no www.Helsenorge.no In Sweden ? In Finland ? Denmark

6. Investigational therapy trials in Denmark A panel appointed by Danish Health Authorities comprising 3, senior, nationally and internationally well connected oncologists to advice patients on investigational therapies Panel offer advice and referral to sites with ongoing trials of investigational therapies which could be suitable for the individual patient, in Denmark or to sites outside Denmark. Costs for trial participation outside Denmark covered by the patients Amt/Region --- In 2014, advice given to 404 patients of which 51 were referred to investigational therapy trials outside Denmark

7. Changing landscape of cancer trials “Personalized medicine - Precision medicine- Individualized medicine” Patient selection based on mutational status in tumor rather than tumor type Low number of patients in each trial due to rarity of the mutations (1-5%)

9. Changing landscape of cancer trials “Personalized medicine - Precision medicine- Individualized medicine” Patient selection based on mutational status in tumor rather than tumor type Low number of patients in each trial due to rarity of the mutations (1-5%) Number of patients with the specific (rare) mutation in each Nordic country may be very low One trial site in the Nordics - patients referred, cross border, from other Nordic countries ?

10. Nordic, Phase I and early Phase II, trial sites Aarhus Bergen

13. Sweden: 9.6 Denmark: 5.6 Finland: 5.4 Norway: 5.2 Iceland: 0.3 Total: 26.1 Populations Similar treatment cultures Common Nordic platform ? Nordic Network for Early Cancer Trials, Nordic-NECT

14. Nordic Network for Early Cancer Trials Nordic-NECT Objectives Establish and develop a network to perform state-of-art phase I and early phase II trials in oncology to ensure all patients access to new investigational therapies. Work for a bilateral agreement between the Nordic countries allowing inclusion and treatment of patients in early clinical trial protocols cross borders. Harmonize the trial approval process in the Nordic countries Promote “One point of entry” for early clinical trials and common approvals for the Nordic countries. Establish a WEB-site with information about the trial sites and ongoing trials. Activity Standard phase I (-II) studies “First-in-man” through proof of concept programs Other complex studies requiring special scientific expertise

15. www.nordicnect.org

20. Nordic Cross Border Trial Collaboration Opportunities and challenges EU Cross Border Directive 2011/24/EU does not apply to clinical trial participation) Legal hurdles ?, different legislation in the Nordic countries Denmark Sweden Finland Norway High Costs ? – Drugs in clinical trial are provided by Pharma for free – Todays drugs commonly have low frequency toxicity – New treatments mostly administered ambulatory Risks ? – Who will carry incurring extra cost in case of complications - intensive care treatment ?

21. The number of drugs in clinical development is increasing Courtesy of Alain Herrera

23. To ensure patients access to investgational drugs we to see the Nordic region as one and reduce barriers for cross border trial participation

24. Number of clinical trials applications to Regulatory Authorities in the Nordic Region Mia Bengtström, Pharma Industry Finland