Interacting with GovernmentPresentation Transcript
WORKING WITH NATIONAL REGULATORS AND HEALTH AUTHORITIES What to expect and what to hope for ALBERT FARRUGIA SENIOR DIRECTOR GLOBAL ACCESS PLASMA PROTEIN THERAPEUTICS ASSOCIATION International Patient Organization for Primary Immunedeficiencies Congress, Hertogenbosch, the Netherlands October 2008
Australian Treasury projections Effect of age group on health spending
Australian Treasury projections Spending on health
Australian Treasury projections Spending on health – sectorial breakdown
What are the priorities of government officials?
3 Deliver better population health outcomes (without
affecting 1 & 2)
awareness of mood in marginal electorates
understanding of swinging voters’ core values
knowledge of market research mechanisms
Precautionism is becoming a dominant policy feature
“… In the event of a future threat to the blood supply, the FDA should encourage small, low-risk solutions to large, difficult problems...” IOM Report 1995
“… If harm can occur, it should be assumed that it will occur. If there are no measures that will entirely prevent the harm, measures that may only partially prevent transmission should be taken…“ Krever Commission 1996
Cross section of Australians questioned about the importance of costs in setting priorities in health care.
Generally, respondents felt unfair to discriminate against patients with high cost illness and that costs should not be a major factor in prioritising .
Majority maintained this view when confronted with its implications in terms total number of people who could be treated and their own chance of receiving treatment if they fall ill.
Results suggest that
Concern with allocative efficiency, as usually envisaged by the economists, is not shared by the general public
Cost-effectiveness approach may be an excessively simple value system upon resource allocation decision making.
FDA PEI TGA
Regulation of plasma derivatives
Produced in large batches from a homogeneous pool of starting material, through well-defined processes subject to standard pharmaceutical quality control.
Biologic drugs such as plasma derivatives cannot be considered as generic agents, and each manufacturing process requires individual assessment with full product specification.
In addition, each product has to be studied in the clinical setting prior to approval, as different brands of the same product have been shown to have different clinical properties and side effects
General properties leading to quality and safety may be reflected in standards of the pharmacopoeia.
But range of approaches to the manufacture results in significant differences between products.
This necessitates thorough evaluation for the potential effect of the process on the factors of interest and the impurities in the products.
Regulation of plasma derivatives
Regulation by the NRA … assuring quality, safety & efficacy
Establishing/maintaining a system of licensing & control
Dossier review and PAI
facility and product registration
inspection and enforcement
Providing standards and guidelines
Requiring that licence holders adopt and maintain appropriate quality systems
Providing competent, consistent, arrangements for post-marketing surveillance of products
NRA involvement in batch testing & release(?)
“ The core of the voting members may include one technically qualified member, selected by the Commissioner or designee, who is identified with consumer interest and is recommended by either a consortium of consumer-oriented organizations or other interested persons.”
Industry and regulators
DO NOT JUST ASK FOR ADVICE. SUBMIT A SOUND, TECHNICAL PROPOSAL DESCRIBING THE APPROACH TO A PROJECT.
DO NOT EXPECT THE REGULATOR TO DESIGN A PROJECT/PROTOCOL/DEVELOPMENT PROGRAM.
AVOID UNSUBSTANTIATED CLAIMS OR STATEMENTS. REGULATORY REVIEWERS ARE UNLIKELY TO ENDORSE SUCH INFORMATION.
DO UNDERSTAND REGULATORY AUTHORITIES POLICY AND ATTITUDES
DO TAKE ADVANTAGE OF THE OPPORTUNITY FOR PRE-APPLICATION MEETINGS
“ Cheshire puss” she begun “would you please tell me which way I ought to go from here?” “That depends on where you want to get to” said the cat.
Forward planning with Regulatory bodies:
Pre Clinical Trial meetings
Pre Marketing Application Submission meetings.
Alice in Wonderland
Demographic changes are increasing pressures on health care budgets
Cost containment, particularly in the expensive pharmaceutical sector, is therefore a government priority
However, the biggest priority for governments remains getting re-elected
This allows consumers a continuing influence in health care policy