- Chronic obstructive pulmonary disease in over 16s guideline updated.
- Final guidance recommends letermovir for cytomegalovirus disease after a stem cell transplant.
- Updated colorectal cancer guideline recommends daily aspirin for people with lynch syndrome (draft guidance).
- Fluocinolone acetonide intravitreal implant not recommended for chronic diabetic macular oedema in phakic eyes after an inadequate response to previous therapy (draft guidance).
- Draft interventional procedures guidance:
- Selective internal radiation therapy for unresectable colorectal metastases in the liver - to be used under special arrangements and research only
- Pressurised intraperitoneal aerosol chemotherapy for peritoneal carcinomatosis – for research only
- Five medicines recommended for approval: Epidyolex, Inbrija, Trogarzo, Deferasirox Mylan, Vitrakvi
- Extensions of therapeutic indication recommended for eight medicines
- Vitrakvi recommend for the treatment of solid tumours with a specific gene mutation.
- Updated restrictions for Gilenya: multiple sclerosis medicine not to be used in pregnancy.
- Negative opinions re-examined for Evenity and extensions of therapeutic indications for Revolade and Translarna
- Latest experimental statistics about FGM published.
- New report on the global tobacco epidemic launched.
- Statement on governance and oversight of human genome editing published.
NATIONAL INSTITUTE FOR HEALTH AND CARE EXCELLENCE
CHRONIC OBSTRUCTIVE PULMONARY DISEASE IN OVER 16s GUIDELINE UPDATED
NICE has updated its guideline on the diagnosis and management of chronic obstructive pulmonary disease (COPD), which includes emphysema and chronic bronchitis, in people aged 16 and older. It aims to help people with COPD to receive a diagnosis earlier so that they can benefit from treatments to reduce symptoms, improve quality of life and keep them healthy for longer. NICE has reviewed the evidence and made new recommendations on:
The guideline also supplements existing recommendations on:
- diagnosing COPD using symptoms, spirometry and other tests
- managing stable COPD using nebulisers, oral therapy and pulmonary rehabilitation
- multidisciplinary management of stable COPD, including physiotherapy, occupational therapy, nutrition and palliative care
- managing exacerbations of COPD in primary care and in hospital
This guideline updates and replaces NICE guideline CG101 (June 2010). In addition, NICE has also produced a guideline on antimicrobial prescribing for acute exacerbations of COPD and a visual summary covering non-pharmacological management and use of inhaled therapies.
LETERMOVIR RECOMMENDED FOR CYTOMEGALOVIRUS DISEASE AFTER A STEM CELL TRANSPLANT
NICE has published final guidance which recommends letermovir (Prevymis), within its marketing authorisation, as an option for preventing cytomegalovirus (CMV) reactivation and disease after an allogeneic haematopoietic stem cell transplant (HSCT) in adults who are seropositive for CMV. It is recommended only if the company provides it according to the commercial arrangement. Current management of CMV after an allogeneic HSCT involves regular blood tests to monitor CMV levels (with or without aciclovir). If CMV levels rise, treatment with ganciclovir, valganciclovir or foscarnet (pre-emptive therapy) is started to prevent disease but this can cause severe side effects. NICE committee says Letermovir is an option for reducing CMV and is safer than pre-emptive therapy. Clinical trial evidence shows that letermovir is effective in reducing CMV infection and also reduces the need for pre-emptive therapy. The most plausible cost-effectiveness estimates for letermovir are within the range that NICE usually considers acceptable.
UPDATED COLORECTAL CANCER GUIDELINE RECOMMENDS DAILY ASPIRIN FOR PATIENTS WITH LYNCH SYNDROME (DRAFT GUIDANCE)
NICE has published draft updated guidance which covers the management of colorectal cancer in people aged 18 and over. It aims to improve quality of life and survival for adults with colorectal cancer through management of local disease and management of secondary tumours. The updated guidance states that aspirin taken daily for more than two years could reduce the risk of colorectal cancer in people with Lynch syndrome (LS) - which increases the lifetime risk of developing colorectal cancer. Regular screening with colonoscopy and polypectomy to identify pre-cancerous cells has until now been the main strategy to detect early colorectal cancer in people with LS and offer appropriate management. NICE published diagnostics guidance in 2017 recommending that everyone who is diagnosed with colorectal cancer should be tested for LS. If the test shows they do have LS, they can be monitored for other cancers and their close relatives can also be offered testing for LS. Also included in the updated guideline is a recommendation for clinicians to consider neoadjuvant chemotherapy for people with the most severe form of colon cancer. This recommendation is based on evidence showing that it improved survival. This guideline will update and replace NICE guidelines CG131 (published November 2011) and CSG5 (published June 2004).
FLUOCINOLONE ACETONIDE INTRAVITREAL IMPLANT NOT RECOMMENDED FOR CHRONIC DIABETIC MACULAR OEDEMA IN PHAKIC EYES AFTER AN INADEQUATE RESPONSE TO PREVIOUS THERAPY (DRAFT GUIDANCE)
NICE has published draft guidance which does not recommend fluocinolone acetonide intravitreal implant as an option for treating chronic diabetic macular oedema that is insufficiently responsive to available therapies in an eye with a natural (phakic) lens. Treatments for untreated chronic diabetic macular oedema include laser therapy and anti-vascular endothelial growth factors (VEGFs). There are no further treatment options for eyes with natural lens (phakic eyes). Clinical trial evidence compares the effectiveness of fluocinolone acetonide intravitreal implant and sham in people with chronic diabetic macular oedema who already had at least one laser treatment. NICE committee says only very few people had anti-VEGFs before the trial and few people had phakic eyes with symptomatic cataracts. Also, non-comparative studies used to support the company’s submission only include few people with phakic eyes and symptomatic cataract. No other data for this group have been identified. NICE says this makes it difficult to establish if fluocinolone acetonide intravitreal implant works better than usual care for these people, especially in the long-term. Because of the lack of clinical evidence, the cost-effectiveness estimates for fluocinolone acetonide intravitreal implant are also uncertain. Even the lowest clinically plausible cost-effectiveness estimates are substantially higher than what NICE normally considers an acceptable use of NHS resources.
SELECTIVE INTERNAL RADIATION THERAPY FOR UNRESECTABLE COLORECTAL METASTASES IN THE LIVER - TO BE USED UNDER SPECIAL ARRANGEMENTS AND RESEARCH ONLY
NICE has published draft guidance which finds the evidence on the safety of selective internal radiation therapy (SIRT) for unresectable colorectal metastases in the liver shows there are well-recognised and potentially serious safety concerns.
- In people who cannot tolerate chemotherapy or have liver metastases that are refractory to chemotherapy, there is evidence of efficacy but this is limited, particularly for important outcomes such as quality of life. Therefore, in these people, this procedure should only be used with special arrangements for clinical governance, consent, and audit or research.
- In people who can have chemotherapy, evidence on efficacy does not show a benefit on overall survival or quality of life. Therefore, in these people, this procedure should only be used in the context of research.
PRESSURISED INTRAPERITONEAL AEROSOL CHEMOTHERAPY FOR PERITONEAL CARCINOMATOSIS – FOR RESEARCH ONLY
NICE has published draft guidance which finds the evidence on the safety of pressurised intraperitoneal aerosol chemotherapy for peritoneal carcinomatosis shows well-recognised, serious side effects. Evidence on its efficacy is inadequate in quality and quantity. Therefore, this procedure should only be used in the context of research. NICE recommends that further research should report details of patient selection, including type of tumour and quality-of-life outcomes.
EUROPEAN MEDICINES AGENCY
FIVE MEDICINES AND EIGHT EXTENSIONS OF THERAPEUTIC INDICATION RECOMMENDED
The European Medicines Agency’s human medicines committee (CHMP) recommended five medicines for approval at its July 2019 meeting. The Committee gave a positive opinion for:
- Epidyolex (cannabidiol) for the treatment of seizures associated with Lennox-Gastaut syndrome or Dravet syndrome. Epidyolex contains an active substance derived from cannabis and is the first to receive a positive opinion in the EU centralised procedure.
- Inbrija (levodopa) for the treatment of symptoms of ‘off’ periods in Parkinson’s disease.
- Trogarzo (ibalizumab), for the treatment of HIV infection.
- The generic medicine Deferasirox Mylan (deferasirox), for the treatment of chronic iron overload due to blood transfusions in patients with beta thalassaemia major, non-transfusion-dependent thalassaemia syndromes and other anaemias.
- Vitrakvi for the treatment of solid tumours with a specific gene mutation (see below for more information)
The CHMP has also recommended eight extensions of therapeutic indication for the medicines Empliciti (elotuzumab), Keytruda (pembrolizumab), Lonsurf (trifluridine/tipiracil), Lucentis (ranibizumab), Soliris (eculizumab), Stelara (ustekinumab), Tecentriq (atezolizumab) and Zerbaxa (ceftolozane / tazobactam).
VITRAKVI RECOMMEND FOR THE TREATMENT OF SOLID TUMOURS WITH A SPECIFIC GENE MUTATION
The CHMP has recommended granting a marketing authorisation in the European Union (EU) for Vitrakvi (larotrectinib) for the treatment of adult and paediatric patients with solid tumours that display a Neurotrophic Tyrosine Receptor Kinase (NTRK) gene fusion. Treatment with Vitrakvi is recommended for patients whose disease has spread or cannot be surgically removed, and who have no other satisfactory treatment options. Vitrakvi is the first ‘histology-independent’ cancer treatment recommended for approval in the EU. This means that it can be used to treat non-haematological tumours with this specific mutation, regardless of where in the body the tumour originated. However, before patients can be started on the medicine, the presence of the mutation in the tumour should be confirmed by a validated test. The active substance – larotrectinib - targets a very specific genomic alteration of a patient’s tumour. This occurs when NTRK genes that encode specific proteins are abnormally fused to a gene. This mutation leads to the development of proteins that can cause cancer cells to grow. Vitrakvi blocks the action of these proteins and in doing so inhibits the growth of the cancer. The efficacy and safety of Vitrakvi were studied in three single-arm trials that included a total of 102 adults and children with cancer that were evaluated. These patients had either already received standard therapy, or would have had to undergo disfiguring surgery, or were unlikely to respond to available therapies. The share of patients who responded to treatment with Vitrakvi was 67%. Of those, the response lasted six months or longer in 88% and 12 months or longer in 75%. Tumour responses were seen both in rare tumour types such as infantile fibrosarcoma and salivary gland tumours, as well as in common diseases such as lung and colon cancer.
UPDATED RESTRICTIONS FOR GILENYA: MULTIPLE SCLEROSIS MEDICINE NOT TO BE USED IN PREGNANCY
The EMA has recommended that the multiple sclerosis medicine Gilenya (fingolimod) must not be used in pregnant women and in women able to have children who are not using effective contraception. If a woman becomes pregnant while using Gilenya, the medicine must be stopped and the pregnancy will have to be closely monitored. This is because the active substance in Gilenya, fingolimod, can harm the unborn baby and may cause birth defects. To minimise this risk, women able to have children must have a pregnancy test before starting treatment with Gilenya to ensure they are not pregnant, and must use effective contraception during treatment and for two months after stopping the medicine. These recommendations follow a review triggered by reports suggesting that the risk of birth defects in infants who have been exposed to Gilenya during pregnancy is twice as high as the 2% to 3% risk observed in the general population. The most frequently reported birth defects in infants exposed to Gilenya were those affecting the heart, kidneys, bones and muscles. Doctors, patients and carers will receive updated educational materials with information about this risk and what actions and precautions need to be taken to ensure the safe use of Gilenya.
NEGATIVE OPINIONS RE-EXAMINED FOR ONE NEW MEDICINE AND TWO EXTENSIONS OF THERAPEUTIC INDICATIONS
The applicant for Evenity (romosozumab) has requested a re-examination of the Committee's negative opinion for this medicine adopted at the June 2019 meeting. The CHMP will re-examine the opinion and issue a final recommendation.
The applicants for Revolade (eltrombopag) and Translarna (ataluren) have also requested re-examination of the Committee's negative opinions, adopted at the June 2019 meeting, for a change to the marketing authorisation of these medicines. The CHMP will re-examine the opinions and issue final recommendations.
LATEST EXPERIMENTAL STATISTICS ABOUT FEMALE GENITAL MUTILATION PUBLISHED
NHS Digital has published official figures that provide experimental statistics on female genital mutilation (FGM) in England, from April 2018 to March 2019. The FGM Enhanced Dataset collates data collected by healthcare providers in England, including acute hospital providers, mental health providers and GP practices on the recording of FGM. The figures show:
- There were 6,415 individual women and girls who had an attendance where FGM was identified in the period April 2018 to March 2019. These accounted for 11,575 attendances reported at NHS trusts and GP practices where FGM was identified.
- The increase in total attendances during 2018-19 would appear to be a change in recording practice, as the number of distinct individuals has remained broadly stable.
- There were 4,120 newly recorded women and girls in the period April 2018 to March 2019. Newly recorded means this is the first time they have appeared in this dataset. It does not indicate how recently their FGM was undertaken, nor does it mean that this is the woman or girl’s first attendance for FGM. The number of newly recorded women and girls has reduced over time. This is to be expected as the longer the collection continues, the greater the chance of a women or girl having been recorded in it previously.
WORLD HEALTH ORGANISATION
NEW REPORT ON THE GLOBAL TOBACCO EPIDEMIC LAUNCHED
A new report from the World Health Organisation (WHO) shows many countries are still not adequately implementing policies, including helping people quit tobacco, that can save lives from tobacco. The seventh WHO Report on the global tobacco epidemic analyses national efforts to implement the most effective measures from the WHO Framework Convention on Tobacco Control that are proven to reduce demand for tobacco. These measures, like the “MPOWER” interventions, have been shown to save lives and reduce costs from averted healthcare expenditure. The MPOWER report was launched in 2007 to promote government action on six tobacco control strategies in-line with the WHO FCTC to:
- Monitor tobacco use and prevention policies.
- Protect people from tobacco smoke.
- Offer help to quit tobacco use.
- Warn people about the dangers of tobacco.
- Enforce bans on tobacco advertising, promotion and sponsorship.
- Raise taxes on tobacco.
The report showed that while only 23 countries have implemented cessation support policies at the highest level, 116 more provide fully or partially cost-covered services in some or most health facilities, and another 32 offer services but do not cost-cover them, demonstrating a high level of public demand for support to quit.
STATEMENT ON GOVERNANCE AND OVERSIGHT OF HUMAN GENOME EDITING PUBLISHED
The WHO expert advisory committee on governance and oversight of human genome editing has made an interim recommendation to the WHO Director-general stating that “it would be irresponsible at this time for anyone to proceed with clinical applications of human germline genome editing.” WHO supports this interim recommendation and advises regulatory or ethics authorities to refrain from issuing approvals concerning requests for clinical applications for work that involves human germline genome editing. WHO’s Expert Advisory Committee will continue to consider the matter, and will, at its forthcoming meeting in Geneva on 26-28 August 2019, evaluate effective governance instruments to deter and prevent irresponsible and unacceptable uses of genome edited embryos to initiate human pregnancies.