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WORKING WITH NATIONAL REGULATORS AND HEALTH

AUTHORITIESWhat 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

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Australian Treasury projectionsEffect of age group on health spending

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Australian Treasury projectionsSpending on health

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Australian Treasury projectionsSpending on health – sectorial breakdown

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What are the priorities of government officials?

1 Get government re-elected

2 Deliver micoreconomic reform (while husbanding votes)

3 Deliver better population health outcomes (without

affecting 1 & 2)

Through awareness of mood in marginal electorates understanding of swinging voters’ core values knowledge of market research mechanisms

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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

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•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.

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FDA

PEI

TGA

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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

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• 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

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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(?)

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“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.”

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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

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“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: Strategic meetings Pre Clinical Trial meetings Pre Marketing Application Submission meetings.

Alice in WonderlandAlice in Wonderland

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Conclusions

• 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