Health Technology Assessments (HTAs) and Rare Diseases Therapies

IPOPI’s recently released a co-edited issue of Pharmaceuticals Policy and Law in collaboration with Professor Jose-Luis Valverde (University of Granada, Spain) looking at Health Technology Assessments (HTAs) and Rare Diseases Therapies.

Health Technology Assessments (HTAs) in Europe and similar assessment procedures in other regions such as comparative effectiveness in the United States, are increasingly being used to measure the clinical efficacy and cost effectiveness of health technologies. Ultimately these mechanisms are also in place to control healthcare budget expenditure.

The topic of HTAs has become in recent years increasingly debated in various international fora and it has become obvious that in order for such processes to be transparent, unbiased and carried out in a robust manner, the involvement of key stakeholders such as patient organization representatives, physicians, health economists and industry representatives amongst others is absolutely necessary.

This is particularly relevant in the case of rare diseases therapies. There are indeed many hurdles that come into play when assessing rare diseases therapies such as the unavailability of Randomised Clinical Trials (RCTs) due to the small patient populations and the seemingly high cost of such therapies.

This edition of Pharmaceuticals Policy and Law seeks to provide an international overview of these challenges by outlining the viewpoints of key expert stakeholders in the field.

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Focus on Immunodeficiencies

This issue of the Pharmaceuticals Policy and Law series (Volume 10) published by IOS press and edited by Professor Jose-Luis Valverde, was co-edited by David Watters, IPOPI and released in February 2008. The publication brings together articles from various expert authors representing key Primary Immunodeficiencies (PID) stakeholders such as patient representatives, physicians, regulatory agencies, policy makers, industry experts, and others. The articles discuss why primary immunodeficiencies are a worldwide public health priority and intend to develop greater awareness of the main issues confronting the PID community such as the need for early diagnosis and access to optimal treatment. Published in hard copy only (currently out of stock).

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IPOPI First NMO Survey

This is the first international survey carried out by IPOPI. The survey was conducted between February 2010 and August 2010 to establish a mapping of several criteria among IPOPI’s NMOs including estimated number of patients in each country, known diagnosed PID cases, patient demographics, presence of registries, collaboration with international registries, provision and funding of care, availability of treatments, presence of national medical advisory panels.

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The Story of Primary Immunodeficiencies “Despite the challenges, the story is not one of despair”

Maj-Lis Hellström, founder in 1978 of PIO, the Swedish organisation for patients with primary immunodeficiency, and an officer of the IPOPI board for eight years, has put together a collection of stories from 14 countries. 49 authors mainly patients and parents, but also doctors and nurses have contributed to this book.

In reading the book you may recognise your own problems and situation if you are a patient or parent, but also, hopefully, find support, comfort and hope. As a student or a doctor you should find much to learn; how it feels being a PID patient or parent; their side of the story. You will also find good case studies for your practice.

This book should be on the reading list for medical students and student nurses. For people in general, this collection of experiences will be very useful and important in gaining some idea of primary immunodeficiency and its impact on affected families. It should lead to a better understanding and ability to help.

The book will be distributed through t.he IPOPI member organisations. Others who are interested can require copies from info@ipopi.org. For multiple copies a contribution to postage costs may be asked.

Sections from the book:
Table of Contents
Article: “It’s alright for you to be ill – You are used to it.”
Article: “You should stop worrying, take your son home, and feed him.”

The book has been published in Sweden by Andersson & Strand Advertising Agency and Certus Printing firm Ltd, 1999. The printing of this book was made possible by the support of Baxter and Bayer.

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Living With Primary Immunodeficiencies

A helpful guide for patients and caregivers

By Sara J. LeBien

CONTENTS:

Section One WHAT HAPPENS AFTER THE DIAGNOSIS

Section Two WHAT TO EXPECT AS YOUR CHILD GROWS OLDER

Section Three ADULT ONSET

Section Four IMPORTANT ELEMENTS FOR SUCCESSFUL COPING

Section Five SUMMARY OF DO NOT’S WE SHOULD
BE AWARE OF

Section Six SUMMARY OF DO’S TO KEEP IN MIND

Section Seven WHAT IS IPOPI and HOW CAN IT HELP?

Section Eight HELPFUL PUBLICATIONS

PREFACE

This booklet is written by an American mother of an adult son with a primary immune deficiency disorder; common variable immune deficiency and chronic malabsorption. It represents her family’s experiences of living with chronic illness over a period of thirty seven years, and her interaction with other parents of children with primary immune deficiency as well as adult patients. She addresses the emotional side effects of this disorder, and includes experiences of other parents of pediatric primary immune deficiency patients and adult patients who kindly offered suggestions from their personal experiences. In addition, there are quotes from other publications that focus on living with chronic illness.

It is our hope that this booklet will help you be better prepared for what you may encounter, and that it offers practical suggestions for coping with typical daily challenges. In addition, it may help you maintain a more stable management of your loved one’s health, as well as maintain your family’s stability while living with chronic illness. It is encouraging to know that cures are being found for some of these disorders.

We do not have a cure for the disorder, but we have solutions for many of the problems. Some of us have had a difficult journey, and now we want to pass along information and suggestions to make your journey less stressful.

INTRODUCTION

PRIMARY IMMUNE DEFICIENCY or PRIMARY IMMUNODEFICIENCY disorders often go undetected because many doctors are unfamiliar with them and only treat the symptoms rather than diagnose the basic disorders. These disorders manifest themselves in a variety of ways, and the seriousness of primary immune deficiency varies considerably . Many people with primary immune deficiency have recurring infections, but nothing serious enough to require hospitalization, while others do have more serious complications requiring hospitalization. Some have few restrictions in their daily living, while others have many restrictions.

To deal with these recurring illnesses the parents or spouses of an immune deficient person face many challenges – physically, intellectually, and emotionally. It can be physically tiring to take care of a sick child or spouse along with all the other family responsibilities, and it requires questioning and research to understand the disorder. Many articles and publications are available to us through our primary immune deficiency organizations and various web sites. But living with a primary immune deficiency also has emotional effects on both the children and adult persons with this disorder and the other family members.Most of us experience several emotions while coping with PID. If we are prepared for some of the possible negative aspects of chronic illness and how they affect our family we can begin to deal with those problems effectively before they become serious.

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