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HTA, CLINICAL PROTOCOLS AND GUIDELINES
HOW TO ENGAGE HEALTH PROFESSIONAL,
PATIENTS, PRESCRIBERS AND THE COMMUNITY?
Prof. Ida Vanessa D. Schwartz, MD, PhD
MY BIAS
GAUCHER DISEASE AND
RARE DISORDERS
Outline
- National guidelines for high-cost drugs in Brazil
& lines of attention (Clinical Protocols)
- Gaucher Disease
Clinical Protocols - Development
- No conflict of interest
- Executive group for the creation of guidelines (Chair:
Prof. Paulo D. Picon, MD, PhD)
For each guideline: one or two physicians (“experts”) prepared a comprehensive review of the literature about the disease and its
treatment.
The first draft guideline is submitted to an expert advisor.
Question: “What does the evidence allow you to recommend as an ideal treatment in that specific situation?”
1
The draft is discussed by the Executive Group
Draft is submitted do the health authorities
Main question: What best-evidence recommendations are feasible in the Brazilian public health system?
The first complete version of the guideline was then published in the Brazilian Official Diary as Public consultancy.
The first complete draft of the guideline is prepared (cost/effectiveness estimates are included whenever inclusion of a new
drug was recommended)
2
Comments were received and analysed, ant the final text is developed.
Final text is submitted to the chair of the responsible department in the MOH.
The final version of the guideline is published in the Brazilian Official Diary as the final decision of the Brazilian Minister of Health
3
Clinical Protocols
Dissemination strategies:
- Publishing of books: In 2002: 300 copies; in 2010: 10,000 copies
(this later version was also published in English); in 2014…
- Electronic publishing available on the sites of OPAS and the
Brazilian Ministry of Health
- Meetings held all over the country
- Meeting for the Brazilian Judiciary System
Clinical Protocols and Therapeutic Guidelines (CPTG) for Gaucher Disease - History
Gaucher Disease
The most common of the lysosomal diseases
The GBA gene (acid beta-glucosidase enzyme) – 1q21.31
Accumulation of glucocerebroside in macrophages
Clinical manifestations
Clinical
manifestations found
in children and
adolescents with
type 1 Gaucher
Disease
Kaplan et al., 2006
Enzyme Replacement Therapy (ERT)
ERT
Imiglucerase
Velaglucerase alfa
Taliglucerase
alfa
Imiglucerase Velaglucerase alfa
Taliglucerase alfa
Miglustate
Date of registration at ANVISA
1994 2010 2013 yes
Date of registration at FDA
1994 2010 2012 yes
Date of registration at EMA
1994 2010 - yes
Presentation 12 U and 424 U Vials
400 U Vial 200 U Vial 100 mg Cap
Excipients Mannitol, tri and disodium citrate,
polysorbate
Sucrose, sodium citrate,
monohydrate citric acid, polysorbate
Mannitol, polysorbate,
sodium citrate, anhydro citric acid
SAS/MS ordinance n. 449, July 9, 2002
SAS/MS ordinance n. 708, October 25, 2011
CLINICAL PROTOCOL AND THERAPEUTIC GUIDELINES
GAUCHER DISEASE
Rational
Evidence-based medicine
Context: SUS (Brazilian National Health
System)
Symptomatic/support treatment is an
important part of treatment of patients with
Gaucher disease (GD)
CPTG for Gaucher Disease - History
CPTG 2002
Imiglucerase
Criteria for the beginning of treatment
Minimum and maximum dose
International consultant: Prof. Ari Zimran (Israel)
CPTG for Gaucher Disease - History
CPTG 2010 – shortage of imiglucerase
3 recombinant enzymes (imiglucerase, velaglucerase,
and taliglucerase = tali still experimental, exceptionally
approved)
Miglustate
Emphasis on clinical monitoring
Rational
Emphasis on clinical monitoring
Specific treatment:
Enzyme
Replacement
(ERT)
Substrate
Reduction
(SRT or SSI)
CPTG for Gaucher Disease - History
CPTG 2014
Taliglucerase alfa is not experimental
Taliglucerase alfa as the first choice for new patients
aged ≥18 years
Brazil
Approximately 700 Brazilians with Gaucher disease (1:60,000 – there should be 3,000 patients!!!)
Number of new diagnosis/year= (?) 5-10
7% miglustate
650 in ERT
Velaglucerase alfa = 4
Taliglucerase alfa = 40 (?)
Other = imiglucerase
Thank You!
Professor Paulo Picon
Pharmacist Barbara Krug
Nutritionist Tatiele Nalin
Clinical Studies
Naïve for Tali (Zimran et al., 2011) = 30 x 60 U/kg/inf – 32 pt (hematological, visceral, chitotriosidase)
Comparison untreated patients (vela x imi x tali) = Inexistent
Comparison untreated patients (vela x tali) = Inexistent
Comparison untreated patients (imi x tali) = Inexistent
Comparison untreated patients (imi x tali) = Inexistent
Comparison untreated patients (imi x vela)= Ben Turkia, 2013 (n= 34)
Switch imi to tali = Pastores et al., 2014 (n= 31)
Switch vela to tali= Inexistent
