Translate the text from Welsh to English.



 
Cyflwr difrifol a etifeddir yw ffeibrosis systig (CF) pan fo nam genetig yn achosi ysgyfaint a llwybr treuliad person i’w llenwi â mwcws gludiog sy’n arwain at rwystrau niweidiol.  Mae symptomau yn cynnwys peswch parhaol, heintiau sy’n digwydd dro ar ôl tro, diffyg maeth a phroblemau difrifol eraill fel diabetes.  Ar hyn o bryd, nid oes triniaeth i wella o ffeibrosis systig; nod y triniaethau presennol yw rheoli symptomau unigol.


Wrth reswm, rwy’n rhannu pryderon y cleifion a’u teuluoedd a’u dymuniad i gael mynediad i driniaethau effeithiol newydd i’r clefyd gwanychol hwn.


Mae’r GIG yn defnyddio dull seiliedig ar dystiolaeth i benderfynu pa driniaethau sy’n gweithio orau i gleifion, a sydd hefyd yn gost\-effeithiol.  Mae prosesau cadarn, sefydlog i weithredu’r dull hwn.  Yn gyntaf, os bydd cwmni fferyllol am werthu meddyginiaeth yn y DU, rhaid iddo gael trwydded oddi wrth yr Asiantaeth Rheoleiddio Meddyginiaethau a Chynhyrchion Gofal Iechyd (MHRA).  Mae’n orfodol.  Mae’r MHRA yn archwilio holl dystiolaeth y cwmni bod y feddyginiaeth yn ddiogel ac yn effeithiol, ac os yw’r dystiolaeth yn ddigon cryf, mae’r MHRA yn dyfarnu trwydded.  Mae meddyginiaethau â thrwydded ar gael yn y GIG yng Nghymru.


Yn ail, yn y DU gall meddyginiaeth gael ei gwerthuso gan un o amryw gorff gwerthuso sef y Grŵp Strategaeth Feddyginiaethau Cymru (AWMSG), y Sefydliad Cenedlaethol dros Ragoriaeth mewn Iechyd a Gofal (NICE) a Chonsortiwm Meddyginiaethau yr Alban (SMC).  Gwirfoddol yw’r broses hon a’r gwneuthurwr sy’n penderfynu cymryd rhan ai peidio.  Mae pob un o’r cyrff arbenigol annibynnol hyn yn craffu ar dystiolaeth y gwneuthurwr o effeithiolrwydd clinigol y feddyginiaeth wrth drin y cyflwr meddygol, yn ogystal â’i chost\-effeithiolrwydd o’i chymharu â’r triniaethau presennol, gan gymryd mewnbwn oddi wrth glinigwyr, gweithwyr iechyd proffesiynol eraill a chleifion i ystyriaeth.  Os bydd AWMSG neu NICE yn argymell y feddyginiaeth, bydd ar gael fel rheol yn y GIG yng Nghymru.  Os nad ydynt, nid yw’r feddyginiaeth yn driniaeth safonol, ond gellir ei darparu drwy wneud Cais Cyllido Cleifion Unigol (IPFR).


Mae Vertex Pharmaceuticals yn gwneud sawl meddyginiaeth i drin ffeibrosis systig, gan gynnwys Orkambi® (lumacaftor/ivacaftor).  Penderfynodd y dylai Orkambi®, ar gyfer cleifion sy’n 12 oed neu’n hŷn, gael ei gwerthuso gan NICE.  Ym mis Gorffennaf 2016 nid argymhellodd NICE hi. Daeth NICE i’r casgliad nad oedd Orkambi® yn gost\-effeithiol.  Gwerthusodd yr SMC yn yr Alban Orkambi® yn 2016 hefyd ond nid argymhellodd hi.  Yn 2017, ailgyhoeddodd NICE ei argymhelliad yn ganllawiau “Do Not Do” gan bwysleisio na ddylai’r driniaeth hon fod ar gael fel rheol.


Ym mis Tachwedd 2017, derbyniodd y Pwyllgor Deisebau ddeiseb am Orkambi® a chael gwybod bod tystiolaeth newydd gan Vertex am ei heffeithiolrwydd clinigol.  Yn hytrach na chyflwyno’r dystiolaeth newydd i NICE, a wnaeth y gwerthusiad gwreiddiol, dywedodd Vertex y byddent ei hanfon at AWMSG.  Llythyrais at Vertex y llynedd a’i annog i gyflawni’r ymrwymiad hwn.  Hyd yn hyn, ni chyflwynwyd dim tystiolaeth newydd i AWMSG.


Ym mis Hydref 2018 gofynnodd Vertex i NICE werthuso Orkambi® ar gyfer cleifion sy’n 2\-11 oed.  Ym mis Rhagfyr 2018 gofynnodd Vertex i’r SMC werthuso Orkambi® am yr un grŵp o gleifion.  Ym mis Ionawr 2019 datganodd Vertex nad oedd proses werthuso sefydlog NICE – a ddefnyddiwyd i werthuso meddyginiaethau yn llwyddiannus am ddau ddegawd – yn briodol am eu meddyginiaethau.


Heb y dull seiliedig ar dystiolaeth – set clir o feini prawf ac arbenigwyr clinigol annibynnol i werthuso effeithiolrwydd clinigol a chost\-effeithiolrwydd meddyginiaethau newydd – ni fyddai modd i’r GIG nodi’r meddyginiaethau sy’n fwyaf cost\-effeithiol a mwyaf effeithiol yn glinigol, neu hyd yn oed gwahaniaethu rhyngddynt.  Y dull seiliedig ar dystiolaeth sydd i’w gael yw’r dull mwyaf diogel i sicrhau’r triniaethau mwyaf effeithiol i gleifion a’r defnydd mwyaf effeithiol o adnoddau’r GIG.  Rwy’n annog cwmnïau fferyllol i gymryd rhan yn y broses werthuso a gofynnwn i Aelodau gefnogi’r dull hwn. 







Translation:


 
Cystic fibrosis (CF) is a serious inherited condition where a gene defect causes a person’s lungs and digestive system to become clogged with sticky mucus. This leads to damaging blockages. Symptoms include a persistent cough, recurring infections, malnutrition and other serious complications such as diabetes. At present, there is no cure for cystic fibrosis and current treatments aim to manage the individual symptoms.


Naturally I share the concerns of patients and their families and their desire for access to effective new treatments for this debilitating disease.


The NHS uses an evidence\-based approach to decide which treatments work best for patients, and are also cost\-effective. There are long\-established, robust processes to implement this approach. Firstly, if a pharmaceutical company wants to sell a medicine in the UK, they must have a licence from the Medicines and Healthcare products Regulatory Authority (MHRA). This is mandatory. The MHRA examines all the evidence the company has that the medicine is safe and effective, and if the evidence is strong enough, the MHRA grants a licence. Licensed medicines are available on the NHS in Wales.  


Secondly, in the UK a licensed medicine may be appraised by any of several appraisal bodies namely the All\-Wales Medicines Strategy Group (AWMSG), the National Institute for Health and Care Excellence (NICE) and the Scottish Medicines Consortium (SMC). This process is voluntary and entirely at the manufacturer’s discretion. Each of these independent expert bodies scrutinise the manufacturer’s evidence of the medicine’s clinical effectiveness in treating the medical condition, as well as its cost\-effectiveness in comparison with existing treatments, taking into account input from clinicians, other healthcare professionals and patients. If AWMSG or NICE recommend the medicine, it becomes routinely available on the NHS in Wales. If they do not, the medicine is not a standard treatment, but can still be provided via an Individual Patient Funding Request (IPFR).


Vertex Pharmaceuticals manufacture several medicines to treat cystic fibrosis, including Orkambi® (lumacaftor/ivacaftor). They chose to have Orkambi®, for patients aged 12 and over, appraised by NICE. In July 2016, NICE did not recommend it. NICE concluded the Orkambi® was not cost\-effective. The SMC in Scotland also appraised Orkambi® in 2016 but did not recommend it. In 2017, NICE re\-issued its recommendation as “Do Not Do” guidance, emphasising this treatment should not be routinely available.


In November 2017 the Petitions Committee received a petition about Orkambi® and was told that Vertex had new evidence of its clinical effectiveness. Instead of presenting the new evidence to NICE, who carried out the original appraisal, Vertex said that they would send it to AWMSG. I wrote to Vertex last year urging them to fulfil this commitment. To date no new evidence has been submitted to AWMSG.


In October 2018 Vertex asked NICE to appraise Orkambi® for treating patients aged 2\-11\. In December 2018 Vertex asked the SMC to appraise Orkambi® for the same group of patients. In January 2019, Vertex stated that NICE’s well\-established appraisal process – used to successfully appraise medicines for 2 decades – was not appropriate for their medicines.


Without the evidence\-based approach \- a clear set of criteria and independent clinical experts to appraise the clinical and cost\-effectiveness of new medicines \- the NHS would have no way of identifying medicines which are the most cost\-effective and most clinically effective, or even distinguishing between them. The evidence\-based approach that is in place is the safest method for ensuring the most effective treatments for patients and the most effective use of NHS resources. I encourage pharmaceutical companies to engage in the appraisal process and I would ask Members to support this approach.