Contestable RFP 2014
In 2014 PHARMAC tested a commercial process aimed at improving people’s access to medicines for rare disorders. This contestable Request for Proposals pilot ultimately led to 10 medicines being approved for listing on the Pharmaceutical Schedule.
PHARMAC has now introduced a set of dedicated features for considering rare disorders medicines so we can get the best out of our existing processes.
The features are:
- A standing Pharmacology and Therapeutics Advisory Committee (PTAC) expert subcommittee for rare disorders will be established. This committee will provide objective clinical advice on rare disorder medicines.
- PHARMAC will regularly call for rare disorder funding applications commencing in the latter part of 2018.
- PHARMAC will undertake dedicated pre-engagement with new, as well as existing, suppliers prior to each call for funding applications.
- PHARMAC will formally adopt adjusted policy settings for rare disorders treatments; notably the set of prerequisites that define ‘treatments for rare disorders’, and that Medsafe approval would no longer be required for rare disorders treatments to participate in the funding application process (but would be required prior to listing).
- The portfolio of rare disorders treatments would be regularly reviewed and good or reasonable opportunities for investment would progress through our routine process. Additionally, if a portfolio of rare disorders investments offers an opportunity to better obtain health gain, then we would run another round of contestable funding or develop an alternate commercial approach, dependent on circumstances.
Medicines approved for listing
We approved 10 medicines for funding as a result of the contestable RFP.
Betaine (Cystadane) for homocystinuria
PHARMAC has agreed to list betaine (Cystadane) on the Pharmaceutical Schedule, a treatment for the rare disorder homocystinuria. Betaine would be listed in the Pharmaceutical Schedule following any approval by Medsafe.
Betaine would be listed for patients with homocystinuria who have not achieved an appropriate homocysteine level with vitamin supplementation. We think this would benefit about 20 patients.
Enzyme replacement therapies - 3 listings
We’re listing three medicines on the Pharmaceutical Schedule for rare enzyme deficiency disorders, for defined uses. These are the result of an agreement with Sanofi Genzyme.
The medicines are:
- Alglucosidase alfa (Myozyme) for infantile-onset Pompe Disease (listed 1 December 2016).
- Idursulfase (Elaprase) for Hunter Syndrome (MPS II) to stabilise patients awaiting stem cell transplant (listed 1 December 2016).
- Laronidase (Aldurazyme) for Hurler Syndrome (MPS 1-H) to stabilise patients awaiting stem cell transplant (listed 1 February 2018).
Listing laronidase and idursulfase will make these medicines more readily available and reduce the administrative task for clinicians wanting to access them. We’ve previously approved their use through exceptions policies.
Alglucosidase alfa will be funded for infantile-onset Pompe Disease, because evidence shows this is where there is the greatest potential for patients to benefit. Our expert clinical advisers told us that, because the disease can progress very quickly in children, treatment can be potentially life-saving. This, combined with a lower cost in children (dosage is weight-based) makes a funding proposal possible.
Cholic acid (Cholebiol)
PHARMAC has approved funding for cholic acid (Cholebiol), for the treatment of rare forms of bile acid synthesis disorders in infants with metabolic liver disease. This is the sixth medicine funded from our rare disorders contestable funding pilot.
Cholebiol would be listed on the Pharmaceutical Schedule once it was approved for use in NZ by Medsafe.
Sodium phenylbutyrate (Pheburane)
PHARMAC has agreed to fund sodium phenylbutyrate granules (Pheburane), a treatment for the metabolic disorders known as urea cycle disorders, which can lead to a fatal build-up of nitrogen and ammonia in the body. Pheburane will be funded from 1 July 2016.
PHARMAC consulted on a proposal to fund Pheburane in November 2015. Following feedback on that proposal, PHARMAC has also agreed to fund sodium benzoate powder for oral solution (Amzoate) from 1 July 2016.
Siltuximab and bedaquiline
PHARMAC has approved funding for two medicines that treat rare disorders, as a result of an agreement with pharmaceutical company Janssen-Cilag.
The two medicines are bedaquiline (Sirturo), which treats a rare extensively multi-drug resistant form of tuberculosis, and siltuximab (Sylvant), which treats a rare form of immune system disorder called HHV-negative idiopathic multicentric Castleman’s disease (iMCD).
Siltuximab is approved by Medsafe, and was listed on the Pharmaceutical Schedule from 1 June 2016. Bedaquiline has been approved by Medsafe, will be listed once distribution arrangements are confirmed.
Listed from 1 May 2016, galsulfase (Naglazyme) is a treatment for people with the rare enzyme deficiency condition Maroteaux-Lamy Syndrome, or mucopolysaccharidosis (MPS) VI. Galsulfase is the second medicine we’ve agreed to fund from our medicines for rare disorders pilot commercial process. It’s for a very small but high need patient group – we think about 4-5 people in New Zealand.
Because of the very low numbers of people with MPS VI, there’s uncertainty of benefits in the clinical evidence. But there’s a high unmet need for some people with the disease, and the potential for people diagnosed early to be severely affected.
So we’re pleased to have reached a commercial agreement with BioMarin that makes funding possible and manages cost and risk. The agreement makes it possible to offer funding to all existing patients, regardless of severity, and despite the uncertainty over the clinical benefits for these patients.
The rare disorders process, and the resulting commercial agreement for galsulfase manage risks and mean funding galsulfase is affordable within the funding provision of $25 million over five years for rare disorders.
MPS VI is usually diagnosed in children and can cause damage to bones, joints, eyes, heart valves and the nervous system.
People with the rare blood condition hereditary angioedema were the first to benefit from the increased competition for rare disorders medicines.
Icatibant (Firazyr), which treats hereditary angioedema (HAE), was listed on the Pharmaceutical Schedule from 1 January 2016. HAE causes episodic attacks of swelling that can be life-threatening.
Icatibant was approved for use in New Zealand by Medsafe in November 2015.
Icatibant can be used to treat severe attacks of hereditary angioedema. An injection that people can have at home, its use can avoid the need for people to go to hospital for infusion treatment. Because it avoids the need for people to visit hospital for treatment, PHARMAC estimates the funding decision should save DHBs money.
PHARMAC estimates there may be up to 90 patients with hereditary angioedema in New Zealand, of whom up to 25 may meet the criteria for funded treatment with icatibant.
Rare disorders contestable funding pilot evaluation
PHARMAC commissioned an evaluation of the pilot, from Grant Thornton. The evaluation has assessed whether the pilot met the objectives outlined in the original discussion document.
As part of the evaluation, PHARMAC also sought feedback from stakeholders that were involved in the pilot process. We’re grateful for the input from stakeholders into the evaluation, which is published below. Note that not all submissions are published at the request of the submitter and some detail is withheld, either by PHARMAC or at the request of the submitter.
There was a mixture of positive and negative feedback received. Overall the evaluation has found that the pilot met most of the criteria set out in the initial discussion document. It found that, while the value of decisions was less than what PHARMAC would usually expect through our regular processes, it did elicit better commercial proposals than previously for some medicines, and resulted in the funding of medicines that PHARMAC has not previously received applications for.
This evaluation report completes the rare disorders contestable funding pilot.
Process - what we did
We released a Request for Proposals in August 2014. There was a positive response from the pharmaceutical industry, which resulted in 28 proposals from eight suppliers of medicines for rare disorders. Many of these medicines had not previously been seen in NZ before and were from suppliers PHARMAC has not previously done business with.
We conducted internal analysis and sought further clinical advice before deciding on our priority list, then negotiated with suppliers.
Each proposal progressed individually, so contracts and listing agreements occured progressively through 2015 and 2016.
The RFP sought bids from suppliers of medicines for rare disorders. Its aim was to improve access to medicines for people with rare disorders by encouraging competition among suppliers.
PHARMAC identified up to $25 million over five years that would be available for rare disorders medicines funding. Listing of medicines and funding for them is open-ended.
Last updated: 10 May 2019