Download Barriers to Innovation & Comparative Trials

PRIORITY MEDICINES FOR
EUROPE AND THE WORLD
Barriers to Pharmaceutical
Innovation
Richard Laing
EDM/PAR
WHO
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Barriers to Innovation:
Background
• Cost and duration of medicines development have
increased
– Many projects in early development stages, but not enough of
this potential innovation is being translated into market
approvals.
• For some diseases, there remains an inadequate
understanding of basic science, and potential
targets for medicines development have not been
identified.
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Recent Publications
• Three papers published almost simultaneously,
plus commentary on Priority Medicines website
– EMEA: Discussion Paper: The European Medicines
Agency Road Map to 2010: Preparing the Ground for the
Future
– FDA: " Challenge and Opportunity on the Critical Path to
New Medical Products"
– Perspectives – "Cutting the Cost of Drug Development?"
M.D. Rawlins
– Sue Middleton: GSK Commentary on "Barriers to
Innovation"
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Barriers to Innovation:
Suggested Solutions
• All authors agreed that every aspect of the regulatory process
should be re-examined and that the evidence base for
regulatory practices should be critically analysed using modern
methodologies.
• Preclinical studies
– Many old tests and methods
– New analytic tools needed
– Validation of old methods required
• Clinical Studies
– Regulatory authorities are risk averse so the RCT has become the
gold standard
– Other approaches even using historical controls may be possible
and desirable
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Neglected Areas:
Post marketing Surveillance and
Patients
• All papers neglect Phase IV studies as a part of
the innovation process. Great opportunities for
Europe from use of electronic data bases for
Phase IV studies on both efficacy and safety
• Apart from the industry paper, none of the three
other papers mention any role for patients in this
review. They are referred to as beneficiaries of
the process but never as contributors to the
decision-making.
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Key Unanswered Question?
What should be the role of patients,
patient organizations and consumer
organizations in promoting innovation?
Patients have been very effective in AIDS
and orphan disease drug development.
Patients are involved in the development of
treatment guidelines for NICE
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A "barriers to innovation"
Research Agenda
• Bottlenecks and barriers in the current medicines development
process are to be identified and solutions elaborated to
overcome them.
– Range of stakeholders required: academia, clinicians, patient
organizations, large and small industry, regulatory and ethics
specialists
– Every requirement within the medicines development process
whether clinical or preclinical should be questioned for its
regulatory relevance, costing, and predictive value.
– The involvement of the EMEA and the various European national
regulatory agencies and their scientists is critical to this initiative.
– 6th Framework already has issued a call closing November 16th
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PRIORITY MEDICINES FOR
EUROPE AND THE WORLD
Comparative Clinical Trials
Warren Kaplan
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Comparative Trials: Background
•
For most medications at launch, we know little about whether or not a
given medication is better value for the money than alternatives.
– This knowledge gap exists because most drug regulatory authorities do not
examine "value for money"
•
For most medications at launch, clinical trials are not designed to look
at whether or not a given medication is better than another drug.
•
Typically, efficacy is measured versus a control which is often, but not
invariably, a placebo lacking any active ingredient.
– Many registration authorities do require comparative benefit studies
•
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To estimate comparative benefit between two medicines, large (time
consuming and expensive) trials are needed since there are often
small differences between treatment outcomes when comparing the
interventions.
Comparative Trials:
Who should pay for them and who
conducts them?
• Industry? Government? Or…
•
Private insurers and the government could set aside some fraction of
their annual medicines spending to endow an organization to provide
an independent source of reputable research into comparative
effectiveness and cost.
– Electronic prescription and medical databases to conduct Phase IV and/or
pharmacoepidemiologic studies in place of controlled, comparative clinical trials.
– EU has a great comparative advantage over the USA as this kind of electronic
linkage of prescribers and medical records is still fragmented in the USA
– Phase IV studies could add a randomization step ("randomized epidemiology")
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Comparative Trials/
Comparative Effectiveness
• Europe 2005: "An information society for all",
available at
http://europa.eu.int/information_society/eeurope/2002/news_lib
rary/documents/eeurope2005/eeurope2005_en.pdf.
• This Action plan on "electronic health" should be
used as a way of creating post-marketing
studies to better understand comparative
effectiveness and cost-effectiveness.
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