March 2016 | Volume 31, Issue 1

Many exciting activities have occurred over the course of this month. TIF has completed many delegation visits, and has finalised the 8th of May toolkit and website! We will leave you to continue reading our newsletter and we hope you enjoy what you read!

TIF's Activities around the world!
Promoting TIF's activities in Romania!
A TIF delegation visit to the capital, Bucharest, took place between 24-25 March 2016 which aimed to promote TIF’s activities and goals in Romania, and to find out the latest developments and policies regarding health care services for rare and chronic disorders.

Dr Michael Angastiniotis, TIF Medical Advisor and Dr Christina Stephanidou, TIF Board Member represented TIF in this delegation visit. In Bucharest, the TIF delegates attended a workshop, where patients, parents and doctors were invited to participate. After the workshop, they had the opportunity to discuss with the doctors, the patients and their families who were present at this event. In addition, the two delegates also had the opportunity to visit the local Hospitals as well as the National Institute of Hematology. Finally, TIF gave Mr Costin Ganescu from the Romanian Thalassaemia Association a donation, in recognition of the valuable work the association is carrying out.

TIF reiterates its aim to further support national efforts to address the control of thalassaemia, and to identify solutions to improve thalassaemia patients’ quality of life and access to quality healthcare.

TIF is also currently in the process of identifying experts or doctors in order to translate the 3rd edition of the Guidelines for the Clinical Management of Transfusion Dependant Thalassaemia in Romanian.

Rare Disease Day honoured by TIF in Cyprus
TIF in collaboration with the Cyprus Alliance for Rare Diseases ( CARD ) have organized a Press Conference to commemorate the Rare Disease Day, celebrated every year on 29 February.
The conference was held on the 29th of February in Nicosia. The theme of this year's Rare Disease Day in accordance with the European Organisation for Rare Diseases ( EURORDIS ) was: "Patient Voice". The day’s slogan was: "Join us in making the voice of rare diseases heard".

The theme of the press conference was: "Reform in health and rare diseases in Cyprus”. On this day individuals who were affected by rare diseases, together with patient organizations, politicians, carers, health professionals, researchers and the pharmaceutical industry, united their voice to inform and sensitize the society on rare diseases and the challenges that patients face.

During the Press Conference, a presentation of TIF’s and CARD’s activities took place, with a special emphasis on TIF’s smartphone application, ThaliMe . Additionally, a film created by the members of the Cyprus Alliance for Rare Disorders was screened as well as the official video of EURORDIS.

EUPATI Webinar

TIF virtually attended the EUPATI Webinar: Informed Consent for Vulnerable Populations on 21 March 2016. In the current Clinical Trials Directive, the definition of conditions for inclusion of vulnerable populations is limited to clinical trials with minors and incapacitated adults who are not able to give informed legal consent. Released in April 2014, the new “EU Clinical Trial Regulation” (EU Regulation 536/2014) expands on information given for the above populations.

Additionally, it also goes further and defines the conditions, especially the informed consent process for clinical trials in pregnant and breast feeding women as well as in emergency situations. Although, the objective is to harmonise the performance of clinical trials in Europe, certain opt-out options for Member States are foreseen and thus might lead to exclusion of certain countries from some trials.

In this EUPATI Network Webinar the speakers have presented the new conditions for informing and including these vulnerable populations in clinical trials. Participants had the opportunity to ask questions and discuss the impact of these new regulations on increasing the number of clinical trials in these populations and thus on enabling new treatments based on reliable data.

MARK YOUR CALENDAR! 2nd Middle East, Gulf, Maghreb and African Regions (MEGMA) Conference
2nd Middle East, Gulf, Maghreb and African Regions (MEGMA) Conference on Thalassaemia and Other Haemoglobinopathies

Autumn 2016
TIF is delighted to announce the organisation of the long-awaited 2nd MEGMA Conference on Thalassaemia and Other Haemoglobinopathies, covering the Middle East (ME), Gulf (G), Maghreb (M) and African (A) regions.

Who should attend?

Patients and Patient Organisations
Healthcare Professionals/ medical specialists
Academics/ researchers
Policy-makers at national, regional, and international level
Industry

A truly inspiring event, the two day conference aims to bring together stakeholders from 22 countries to discuss avenues of action with a particular focus on PATIENT EMPOWERMENT and patient-association CAPACITY BUILDING. Furthermore, the scientific programme will cover a broad range of topics on the clinical management and MULTIDISCIPLINARY CARE and most importantly novel and INNOVATIVE TREATMENT methods thalassaemia and other haemoglobinopathies.

More details will be available soon

Many activities in store for the International Thalassaemia Day 2016!
The 2016 theme for the 8th of May activities which is:
‘Access to Safe & Effective Drugs in Thalassaemia’.

This year’s theme highlights the need to support policy decisions that safeguard health and reduce inequalities in the health field. TIF’s mission is to ensure equal access to quality health care for all patients, so that they receive appropriate treatment and drugs, free of charge or reimbursed by the government, and in concurrence with the guidelines and standards of international experts.

You are kindly requested to send us your plans of action for the 8th of May 2016 through completing the following form.
or alternatively by sending them to us at thalassaemia-lp@thalassaemia.org.cy.

We have also devoted a weebly blog for our members to share their plans for the 8th of May. We invite you to share your ideas for the day.

International Photography Contest 2016
Thalassaemia International Federation (TIF) is organising a Photography competition for the year 2016, with the aim of creating awareness about thalassaemia, in the context of the International Thalassemia Day, celebrated each year on May 8.

Participation in this contest is open for all ages, and all countries across the world

Deadline
The deadline for the competition is Tuesday, 30 April 2016.

Subject
The subject of this photography contest is based on this year’s theme for the International Thalassaemia Day
The prizes granted by Thalassaemia International Federation for this competition are the following:
The first winner - $ 500,
The second winner - $ 300
The third winner - $ 100

Submissions
Submissions should be sent to thalassaemia-lp@thalassaemia.org.cy with the name and surname of the participant as well as the title of the photography contest title in the subject of the email.
For further clarifications, please contact Thalassaemia International Federation (TIF) at thalassaemia-lp@thalassaemia.org.cy or 00357 22 319 129

Click here for the Terms and Conditions of this contest.
Click here for the FAQ section.

Anthem translated and sung in English for this year's International Thalassaemia Day!
As some of you may already know, an anthem for thalassaemia has been created in Greek by Mr. George Theofanous, a world renowned composer, musician, and lyricist. The music for this anthem was composed by him and the lyrics were written by Mr. Stavros Stavrou. This composition was sung by the children of his Musical Workshop and it was presented for the first time during TIF’s formal Musical Evening and Gala Dinner, entitled Precious Rubies, held at the Presidential Palace on 6 May 2015 to honour last year’s International Thalassaemia Day.

We are delighted to share with you that this anthem has now been translated and sung in English! You can listen to it by clicking the following link:

We invite you to translate them into your language, so that we can spread the joyous and hopeful message conveyed by the anthem to all those affected by thalassaemia for the International Thalassaemia Day 2016.

Closing, we would like to express a special thank you to Mr George Theophanous, our Musical Ambassador for his support and contribution to advocating the rights of thalassaemia patients.

Toolkit available!
Click here for the poster.
Click here for the banner.

Publications

OUT NOW!
Publications

Guidelines for the Management of Transfusion Dependent Thalassaemia (TDT), 3rd Edition (2014), English

Translations of Publications

A Guide for the Haemoglobinopathy Nurse (2013), Greek

COMING SOON
Publications

Shorter Version of the Guidelines for the Management of TDT (2014), English, Turkish
Shorter Version of the Guidelines for the Management of Non Transfusion Dependent Thalassaemia (NTDT) (2013), English, Turkish
Shorter Version of the Prevention of Thalassaemias and other Haemoglobin Disorders, Vol 1, and 2, 2nd Edition (2013), English
Update of the Guidelines for the Management of NTDT (2013), English

Translations of Publications

A Guide for the Haemoglobinopathy Nurse (2013),Farsi, Myanmar
Guidelines for the Management of TDT 3rd Edition (2014),French, Turkish, Greek, Arabic, Chinese
Sickle Cell Disease (2008), Arabic
Guidelines for the Clinical Management of Non-Transfusion Dependent Thalassaemias(2013) in French
Translations of Videos
All about Thalassaemia Cartoon Animation (2013) is being translated into Bahasa Indonesian, Malaysian, Chinese, Hindi, Urdu

POSITION PAPERS
It is TIF’s policy to present its position on key issues of concern to patient groups, health authorities, professionals and society as a whole. Position papers are curently being prepared on the following topics:

▀▀ Liver disease in thalassaemia - liver disease is emerging as a major cause of morbidity and mortality in thalassaemia patients. TIF therefore maintains that its prevention, monitoring and case management must be a priority for thalassaemia services.

▀▀ Effective monitoring of patients - TIF is concerned that few centres follow full monitoring protocols, with the result that only a minority of patients are benefiting from early identification of and protection from potential threats to their health.

▀▀ Adherence to treatment – the need for strict adherence to chelation and other aspects of the thalassaemia treatment protocol, as per international guidelines, is a subject that must be revisited, especially in view of new therapies that may lead to a final cure, but from which patients may only benefit if they maintain good overall health The first of these position papers will be released before the upcoming months..

International News
Tee Off with the Staten Island Chapter on May 4
Staten Island’s South Shore Golf Club is the place to be on 4 May 2016 as the Staten Island chapter’s 2016 Cooley’s Anemia Golf Outing takes to the greens.

Registration and lunch are at 11:00, in advance of the 12:30 shotgun start. Cocktails and dinner follow at 6:00. Want more? There’s also a hole-in-one contest, a putting contest, performance prizes, door prizes and raffles – pretty much something for everyone.

The price is $185 per person; $100 if just coming for cocktails and dinner. Reservations are required by April 25. There also are a number of great sponsorship opportunities available.

Call Robert Aiello at (718) 369-3434 or the CAF Staten Island chapter at (718) 761-5380 for more information.

You can download an event brochure, which also includes sponsorship information, by clicking here.

Join the 2016 CAF Care Walk and Get Chance to Win Gift Card

Many activities held in Egypt!
Many activities were held in Egypt to create awareness about thalassaemia. A few of these are the following:

Many competitions were held which have ended on 19 February 2015. The winners, who have already been announced, won a free journey to visit Mecca and Madina Monoara in Egypt.
Additionally, the national association ofEgypt also held competitions in schools for the best composition titled a" thalassaemia patient's life".
The association also participated in a film about" the life of thalassaemia patient"
The association has also undertaken a Program for human development for children which aimed to promote the self-development of children with thalassaemia

Towards better prevention and management of chronic diseases

by Martin Seychell, Deputy Director General of the Directorate for Health and Food Safety, European Commission

The burden of chronic disease is staggering: 86% of all deaths, or 4 million per year, are related to chronic diseases in Europe. Chronic diseases develop slowly, are long-lasting and often incurable. They have caused great human suffering and placed an enormous burden on health systems as well. 70% to 80% of all healthcare costs in the EU - an estimated €700 billion - are currently spent on chronic diseases. In addition, chronic diseases completely stop many people from being able to work and nearly a quarter of those who do work - 23.5% - suffer from a chronic condition.

Read the whole article here (European Commission Newsletter, 15/02/2016)

IRDiRC publishes State of Play of Research in the Field of Rare Diseases: 2014-2015
The International Rare Diseases Research Consortium (IRDiRC) has uploaded an enlightening report which includes extensive information on the major developments across the globe in the field of rare diseases. The content of this report will be useful for any stakeholder whether it is to support decisions of policy makers and research funders, or educate the rare diseases community at large of the achievements and of observed trends which shape the future of research and development for rare diseases.

The report has been compiled by means of a systematic survey of published articles, between July 2014 and June 2015, in scientific journals and press releases. It identifies several major policy initiatives that were taken during this period notably, the funding provided by the Canadian Institutes of Health Research and Genome Canada to study models and molecular mechanisms of rare diseases, the joint proposal of the EMA and the FDA on research for new Gaucher disease medication, among others. The report highlights the guidelines and recommendations that are likely to benefit rare disease research. The report also published the outcomes of previous major initiatives such as FORGE, Deciphering Developmental Disorders, FDA’s Orphan Products Grants Program and EMA’s adaptive licensing pilot project. The report also describes useful databases such as Linked2Safety, ClinRegs and ClinGen. It highlights trends that will significantly impact rare disease patients such as involving patients at the EMA for discussions on benefit/risk assessment, importance of patient reported outcomes, among others.

More information: IRDiRC State of Play report, (Orphanews 29/02/2016)

RD-ACTION: the new European Rare Disease Joint Action
RD-ACTION is a new Joint Action consisting of the member states of the European Union for rare diseases, following the two previous Joint Actions - Orphanet Joint Action and EUCERD -, and represents renewed support of the European Commission (EC) to rare diseases, through its Directorate General for Health (DG SANTE).

RD-ACTION has three main objectives:

to contribute to the implementation, by member states, the recommendations of the EC Panel in relation to policies on these diseases,
to support the development of Orphanet and make it sustainable,
to help Member States to introduce the ORPHA code in their health systems to make rare diseases visible.

With a global budget of €8,344,079, this work will last three years (until June 2018), following the logic of coherence and continuity vis-à-vis the previous actions, but aims to go further in terms of concrete implementation and consolidation policies.

More information: European Commission press release, (Orphanews 29/02/2016)

Medical Corner (from TIF’s medical sources)
Bluebird bio reports new beta-thalassemia major data from Northstar study of LentiGlobin at ASH Annual Meeting: Transfusion reduction between 33 percent and 100 percent observed in patients with β0/β0genotype

The Northstar Study is an ongoing, open-label, single-dose, international, multicenter Phase 1/2 study designed to evaluate the safety and efficacy of LentiGlobin BB305 drug product for the treatment of subjects with beta-thalassemia major. As of October 28, 2015, 13 subjects with beta-thalassemia major have undergone infusion with LentiGlobin BB305 product candidate. As of October 28, 2015, nine of these subjects had at least six months follow up. Results in these patients as of October 28, 2015 include: Median HbAT87Q production at six months follow up was 4.9 g/dL among patients of all genotypes (n=9), 4.9 g/dL among patients with non-β0/β0 genotypes (n=5) and 5.0 g/dL among patients with the β0/β0 genotype (n=4).

Bluebird bio reports new beta-thalassemia major and severe sickle cell disease data from HGB-205 and HGB-206 Studies of LentiGlobin® at ASH Annual Meeting

HGB-205 is an ongoing, open-label, single-center Phase 1/2 study designed to evaluate the safety and efficacy of LentiGlobin BB305 drug product in the treatment of patients with beta-thalassemia major and severe SCD. As of November 10, 2015, four subjects with beta-thalassemia major and one subject with severe SCD have undergone infusion with LentiGlobin BB305 product candidate in this study. Results as of November 10, 2015, include: Subject 1201 with the β0/βE genotype of beta-thalassemia major has 23.4 months of transfusion independence with total hemoglobin of 10.8 g/dL, of which 7.9 g/dL was HbAT87Q. Subject 1202 with the β0/

View the full release here:http://www.businesswire.com/news/home/20151206005067/en/

Luspatercept Development in Beta-Thalassaemia
Celgene Corporation in partnership with Acceleron Pharma Celgene, in partnership with Acceleron is developing luspatercept, an investigational drug that inhibits the activin receptor type IIB (ActRIIb) which is involved in the late-stage maturation and differentiation of red blood cells. Given this potential mechanism of action and the results of Phase 2 studies, luspatercept is now in Phase 3 development for the treatment of ineffective erythropoiesis in beta-thalassaemia and myelodysplastic syndromes. The majority of the BELIEVE study sites around the world will begin recruiting patients in the first half of 2016. Positive results from this trial could result in the approval of luspatercept, making a new treatment option available for patients with beta-thalassaemia.