Where are we today?

Where are we today according to BMS satellite symposium at EADO 2015:

PFS or OS?

One of the topics at EADO 2015 was "Which endpoint for which trial?" regarding whether progression free survival (PFS) or overall survival (OS) should be the primary endpoint for a clinical trial. OS is regarded as the golden standard primary endpoint in oncology clinical trials but comes at a cost with longer trials, larger patient population (harder to recruit) and that the OS may be affected by crossover design trials (which lets patients to continue with another treatment/study arm if the first one didn't work). Clinicians want their numbers right, but but who wants to stay on an inferior arm waiting to die just to prove that the other stuff is better? Not me.

The speaker Prof. Dr. Axel Hauschild showed in his talk a retrospect study that the correlation between PFS and OS is very high, with an R-value of 0.95 (1.0 = absolute correlation) so why don't we use PFS as the new "golden standard"?

The retrospect study was done on melanoma trials and according to http://www.cancerworld.org/pdf/8995_pagina_39_43_eGranround.pdf  (posted on the MPNE forum) PFS as primary endpoint cannot be used for every single cancer.

First day at EADO

EADO overview of last four years achievements in immunotherapy: ipilimumab, nivolumab and pembrolizumab. Good to see again the confirmation of their efficacy in melanoma.
(to be continued)

New melanoma advocates at Quickstart pre- EADO workshop in Marseille

Today has started Melanoma QuickStart pre-EADO workshop organized by MPNE and Melanome France around EADO conference.  


I was glad to meet there 
Gilliosa Spurrier (Melanome France), Fredrik Östman (Sweden), Gislaine Cramers (Stichting Melanoom, Netherlands), Antonella Romanini (ACM, Italy), Anca Andronic (Melanom Romania), Kacie King (Belgium), Henriette Sandven (Norway),Corinne Forest (France), Debora Podestà (Italy), Gudrun Uthaug (Norway) and Martina Kiehl (Germany). 

In total 15 patients, carers and patients advocates from different countries participated to the workshop and will attend EADO Congress in Marseille (28-31 october), learning about the new treatments landscape in melanoma and how to share the information with their own national groups. 

 Presentations and discussed subjects - to be completed 

Vitiligo and clinical response to pembrolizumab

Interestingly for patients on pembrolizumab, vitiligo (white spots on the skin) seems to indicate a positive response of the drug. Good news to me at least :)

Caroline Roberts put it very cautiously that "at least it is not very bad [for the patients]" at EADO 2015. 

Even though the dataset was small  (N = 67),  out of  the 17 people who got vitiligo only two of them had a progressive decease. But the rest were either stable (3), or had partial (9) or complete (3) regression. So if  you get vitiligo it might mean good news, but a larger dataset is needed to show if it is statistically significant.

Prof Luc Thomas of the University Hospital Lyon Sud.

Patients must be prepared to lobby HTA and Reimbursement authorities as they frequently do not listen to Expert opinions, which leads to decisions that are NOT in the interest of Patients - Melanoma Patients are often well-informed ..

PATIENTS AS INDEPENDANT STAKEHOLDERS

SO - WHO ARE THE STAKEHOLDERS IN MELANOMA TREATMENT/TRIALS ?

What is a stakeholder ? 

A group of people with shared interests that are different from other groups


Clearly the most important stakeholders are those who commit their lives : 

THE PATIENTS

Then we have : CARERS and  FAMILIES  

also GOVERNMENTS who bear the costs of healthcare

then we have CLINICIANS and RESEARCHERS who do the work

we have the PHARMACEUTICAL COMPANIES who fund development

we have HEALTH TECHNOLOGY ASSESSORS who decide on the risks/benefits

we have THE PUBLIC