Transcript Fra informasjon til viten

Patients access to investigational drugs
Steinar Aamdal
Dept. of Clinical Cancer Research
Oslo University Hospital
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
Benefits of Participating in Clinical Trials
• Early access to new treatments
• Active role in own health care
• Participation in advancing medical knowledge
3
Barriers to Participation in Clinical Trials
• Physicians` and patients` unawareness of
clinical trials
• Lack of access to trials
4
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
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
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%)
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 ?
Nordic , Phase I and early Phase II,
trial sites
Bergen
Aarhus
Common Nordic platform ?
Populations
Sweden: 9.6
Denmark: 5.6
Finland: 5.4
Norway: 5.2
Iceland: 0.3
Total:
26.1
• Similar treatment
cultures
Nordic Network for Early Cancer Trials, Nordic-NECT
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
www.nordicnect.org
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 ?
The number of drugs in clinical
development is increasing
Courtesy of Alain Herrera
To ensure patients access to investgational
drugs we to see the Nordic region as one
and reduce barriers for cross border trial
participation
Number of clinical trials applications to
Regulatory Authorities in the Nordic Region
Mia Bengtström, Pharma Industry Finland