Somatropin for patients under 2 years with Prader-Willi Syndrome

Medicines

Decision

PHARMAC is pleased to announce that somatropin (human growth hormone) will be fully funded for patients with Prader-Willi Syndrome (PWS) under the age of 2 years, subject to certain clinical criteria being met, from 1 January 2017.

Widened access to somatropin for patients with PWS under the age of 2 years was the subject of a consultation letter issued in May 2015.  No decision was made in 2015 as it was determined that further clinical advice was needed on the issues raised during consultation before a decision could be made.

Clinical advice has now been sought and this advice is reflected in the new criteria.

Details of the decision

The funding criteria for Omnitrope (somatropin injection 5 mg, 10 mg and 15 mg) in the Pharmaceutical Schedule will be amended from 1 January 2017 as follows (additions in bold, deletions in strikethrough, showing only the amended criteria for PWS and not the entire set of somatropin criteria);

Initial application — (Prader-Willi syndrome) only from a paediatric endocrinologist or endocrinologist. Approvals valid for 9 months for applications meeting the following criteria:

All of the following: 

  1. The patient has a diagnosis of Prader-Willi syndrome that has been confirmed by genetic testing or clinical scoring criteria; and
  2. The patient’s height velocity is < 25th percentile (adjusted for bone age/pubertal status if appropriate) as calculated over 6 to 12 months using the standards of Tanner and Davies (1985); and
  3. Either: 

3.1  The patient is under two years of age and height velocity has been assessed over a minimum six month period from the age of 12 months, with at least three supine length measurements over this period demonstrating clear and consistent evidence of linear growth failure (with height velocity < 25th percentile); or
3.2  The patient is aged two years or older; and

  1. The patient is aged six months or older; and
  2. A current bone age is < 14 years (female patients) or < 16 years (male patients); and
  3. Sleep studies or overnight oximetry have been performed and there is no obstructive sleep disorder requiring treatment, or if an obstructive sleep disorder is found, it has been adequately treated under the care of a paediatric respiratory physician and/or ENT surgeon; and
  4. Either:

5.1  Both:

5.1.1    The patient is aged two years or older; and
5.1.2   There is no evidence of type II diabetes or uncontrolled obesity defined by BMI that has increased by ≥ 0.5 standard deviations in the preceding 12 months; or

5.2  The patient is aged between six months and two years and a thorough upper airway assessment is planned to be undertaken prior to treatment commencement and at six to 12 weeks following treatment initiation.

There will be no change to the renewal criteria for somatropin for people with PWS.

Feedback received

Responses to the May 2015 consultation, while generally supportive of the proposal, raised a number of issues with regards to the proposed criteria for children; particularly around the growth velocity requirements for renewal of funding, risks of harm if somatropin is given to young babies, and requests for access to be further widened by extending funded treatment to include adult patients with PWS.

In July 2015 we notified that no decision had been made on the proposal and interested parties were invited to submit additional information to PHARMAC for consideration by our clinical advisors alongside all consultation responses.

Clinical advice was sought from both the Endocrinology Subcommittee of the Pharmacology and Therapeutics Advisory Committee (PTAC) and from PTAC itself regarding widened access to somatropin for children with PWS under the age of 2 years and the issues raised during consultation.

The new criteria which will be in place from 1 January 2017 are in line with the advice we received and differ slightly from the criteria detailed in the consultation letter issued in May 2015.

We note that advice on the funding of somatropin for adolescents and adults with PWS was also sought from both the Endocrinology Subcommittee and PTAC. Both committees recommended that funding of somatropin for this patient group be declined, primarily because of lack of evidence.

More information regarding consideration of somatropin, including links to the PTAC and Subcommittee minutes, can be found here(external link). This will be updated with the November 2016 PTAC minutes later in the month.

More information

If you have any questions about this decision, you can email us at enquiry@pharmac.govt.nz or call our toll free number (9 am to 5 pm, Monday to Friday) on 0800 66 00 50.