Overview

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The Association works diligently to maintain and improve access to plasma protein therapies through its global advocacy efforts. PPTA works to promote understanding among policymakers regarding the unique nature of the plasma protein therapeutics industry, and the rare disease populations who rely on these therapies. Increased diagnosis, treatment and reimbursement form the basis of these efforts.

Below you will find complete recor of access to care issues by region and PPTA activities in support of access to therapies.

The Association manages global policy activities that impact patient access to care. PPTA’s global access initiatives are designed to identify and break down barriers to access which result from global factors such as:

  • Economic issues
  • Clinical requirements
  • National Policies

Activities include:

  • Differentiation of plasma protein therapies.
  • Development of economic models.
  • Documentation of evidence that demonstrates the value and cost effectiveness of the therapies.

The global economic crisis has elevated the significance of these efforts. The Access to Care team has undertaken a strategic program to identify barriers and threats to access plasma protein therapies worldwide and develop tactics to overcome them and ensure access where appropriate through:

  • Engagement with authorities involved in approving and reimbursing treatments.
  • Engagement with patients receiving treatments.
  • Engagement with physicians , pharmacists, nurses and other health care professionals.


There are five core tactics used to accomplish these goals:

  • Product support, identity and differentiation 
    • via practice guidelines that determine how therapies are administered and used by qualitative comparisons to other therapies 
    • Monitoring developments in the clinical use of the therapies

  • Stakeholder support
    • Patient Groups
      • Work with patient groups to secure surveys for Quality of Life data
      • Conduct surveys with patient groups and others regarding usage
      • Share data and findings to support patient access to therapies and advocacy efforts

  • Clinical Investigators
    • Work globally to further scientific and medical knowledge
    • Sponsor scientific meetings and panels
    • Collaborate with experts on research projects

  • Industry advocacy
    • Liaise with international bodies to promote facts and findings.
      Currently, this includes the AABB's and the International Society of Blood Transfusion

  • Clinical barriers
    • Work to increase diagnosis and access to therapies
    • Eliminate and reduce clinical barriers through evidence-based medicine.
    • Develop meta-analyses for albumin and immunoglobulin.
    • Reimbursement barriers.
    • Work to eliminate or reduce reimbursement barriers that limit patient access to therapies.

FIND-ID

The key objectives of FIND-ID network are to create awareness for PID, including the warning signs and simple diagnostic tests, among general practitioners (GPs), specialist physicians, ENT-experts, pulmonologists etc., and physicians in regional hospitals, throughout Germany, and encourage them to refer their patients to established and specialized PID centers. The network includes patients, centers and clinicians.

PPTA, together with a powerful coalition of patients, clinicians, health economists and policymakers has developed health technology assessment models (HTAs). These economic models demonstrate the cost-effectiveness of plasma protein therapies latent therapeutic demand for therapies such as immunoglobulin. This contributes to the understanding of stakeholders including treaters and funders regarding the clinical demand for the therapies.

  • Latent Demand in PID: A Decision-Analysis Model for Estimating Latent Therapeutic Demand for Immunoglobulin Therapies in Primary Immune Deficiencies
  • Prophylaxis in Hemophilia A is More Cost-Effective Than On-Demand Therapy in a Cost-Utility Model
  • Colloid Therapies in Sepsis Models

 

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