Role of clinical trials in professional development

Transcription

1 Role of clinical trials in professional development Peeter Padrik Tartu University Hospital University of Tartu Estonian Society for Medical Oncology

2 How we do treatment decisions? Personal experience? Recommendations from colleagues or third persons? Observations? Clinical trials Systematic information from clinical trials clinical practice guidelines

3 Are treatment results good enough? 5-year relative survival; diagnosed EUROCARE-4 (%) US SEER-13 (%) Testicular cancer Melanoma Hodgkin s lymphoma Breast cancer Prostate cancer Cervical cancer Colorectal cancer Kidney cancer Gastric cancer Lung cancer All cancers, MEN All cancers, WOMEN

4 Clinical trials are the only way to translate laboratory discoveries into treatments that extend and improve the lives of patients

5 Clinical trials in oncology In cancer research, clinical trials are designed to answer questions about new ways to: Treat cancer Find and diagnose cancer Prevent cancer Manage symptoms of cancer or its treatment Not only trials with medical therapies!

6 Phases of clinical trials in oncology Phase 0 Phase I Phase II Phase III Phase IV

7 To find a safe dose Phase I Trials To decide how the new treatment should be given To see how the new treatment affects the human body Conducted in cancer patients who have failed standard therapy patients in a study

8 Phase I Trials: our experience 2 trials New agent or a new combination Time and resource consuming At the beginning suspicious attitude for approval: But why in Estonia? Why in cancer patients?

9 Phase I Trials: our experience Strong interest from patients side to participate in the study We can do that; in international cooperation E.g.: Padrik, P.; Price, TJ.; Spear, M.A.; Townsend, A.; Longenecker, A.; Palladino, M. A.; Lloyd, K. G.; Cropp, G. F.; Millward, M. (2008). PHASE 1 STUDY OF THE NOVEL PROTEASOME INHIBITOR NPI-0052 IN PATIENTS WITH ADVANCED MALIGNANCIES INCLUDING LEUKEMIAS. In: Annals of Oncology : European Society of Medical Oncology Congress, Stockholm, Sept Oxford University Press, 2008, (Supplement: Suppl. 8 ),

10 Phase II Trials The primary goal of these trials is to screen the new agent or regimen for efficacy and to estimate the level of activity Less than 100 patients in a study

11 Phase II Trials: our experience Professionally interesting: possibilities to see treatment efficacy with new agents or approaches Possibility to conduct own investigator-initiated studies E.g.: Padrik, P.; Leppik, K.; Arak, A. (2004). Combination therapy with capecitabineand interferon alfa-2a in patients with advanced renal cell carcinoma: a phase II study. Urologic Oncology-Seminars and Original Investigations, 22(5),

12 Phase III Trials To compare the new treatment with the current standard treatment From 100 to thousands of patients Large international or global studies

13 Phase III trials: our experience Most common oncology trials in Estonia Studies with positive and negative results Our small contributions to the global progress E.g.: Slamon, D.; Eiermann, W.; Robert, N.; Pienkowski, T.; Martin, M.; Press, M.; Mackey, J.; Glaspy, J.; Chan, A.; Pawlicki, M.; Pinter, T.; Valero, V.; Liu, M.; Breast Cancer International Research Group incl. Padrik, P. (2011). Adjuvant trastuzumabin HER2-positive breast cancer. New England Journal of Medicine, Oct. 6,

14 Standardized approaches useful also for quality of practice outside of trials

15 Phase IV Trials To further assess the long-term safety and effectiveness of a new treatment Several hundred to several thousand of patients

16 Phase IV Trials: our experience Main benefit: making available for patients therapies, which are not reimbursed in Estonia User experience for doctors E.g.: Smith, I.; Pierga, J.; Biganzoli, L.; Cortés-Funes, H.; Thomssen, C.; Pivot, X.; Fabi, A.; Xu, B.; Stroyakovskiy, D.; Franke, F.; Kaufman, B.; Mainwaring, P.; ATHENA Study Group incl. Padrik, P. (2011). First-line bevacizumab plus taxane-based chemotherapy for locally recurrent or metastatic breast cancer: safety and efficacy in an open-label study in 2,251 patients. Annals of Oncology, 22(3),

17 Example of attitude: NCCN Guidelines Clinical Trials: NCCN believes that the best management of any cancer patient is in a clinical trial. Participation in clinical trials is especially encouraged.

18 In Estonia? Are we ready to declare and accept, that clinical trials are the treatment of choice for patients also in Estonia? Clear understanding is needed in medical community: Clinical trials are usual and preferable approach in everyday clinical practice

19 The costs of procedures and therapies in clinical trials The costs of care of patients participating in a clinical trial fall into two general categories: 1) routine care costs 2) research costs Routine patient care costs" are the usual costs of medical care, that patients would receive whether or not they are participating in a clinical trial These costs are usually covered by health insurance Research costs are costs associated with conducting a clinical trial Research costs are usually covered by the organization that sponsors the trial.

20 KLIINILISTE RAVIMI-JA TEADUSLIKE UURINGUTE LÄBIVIIMISE KORD KLIINIKUMIS, Ver Ravimuuringute protokollijärgsete tegevuste eest ei esitata arveid Eesti Haigekassale. See ei kehti haige seisundist tuleneva standardravi tegevuste kohta, kui konkreetne ravi on patsiendi valikukriteeriumiks uuringusse.

21 American Society of Clinical Oncology about cancer research Blueprint for Transforming Clinical and Translational Cancer Research

22 ASCO vision: Cancer science is in a period of revolutionary change (due to rapidly growing understanding of the biology of cancer) Clinical cancer research and patient care could be vastly more targeted, more efficient and more effective While progress has been substantial, it has generally been the result of incremental advances over time Research sponsors currently devote substantial resources to trials and therapies that promise only marginal improvements over current standards of care FASTER, SMARTER CLINICAL TRIALS are necessary

23 ASCO recommendations for further research Prioritize trials with the greatest potential benefits for patients Select study populations based on molecular characteristics Employ flexible, efficient trial designs Train health care providers in clinical research

24 Thank you!