P2.1 Details of proposed product and its comparators (Section 1)
Page last updated: September 2016
Additional Information Requests
- Comply with all information requests in Part A of these guidelines, where applicable
- Describe the main comparator(s) (Subsection 1.1)
- Provide additional information for the following medical conditions (Subsection 1.1):
- multifood allergy
- patients requiring products with modified carbohydrate, protein or fat for malabsorption or disorders of metabolism
- patients requiring ketogenic diets
- infant formula products, such as a formula used in infants younger than 12 months
- Confirm regulatory compliance with the Australia New Zealand Food Standards Code (Subsection 1.3)
- Provide additional information about the proposed product and its use (Subsection 1.4):
- a list of all ingredients
- justification for the requested maximum quantity allowed and repeats
- macronutrient and micronutrient content per 100 kcal of product and per 100 mg or 100 mL of product
- a table of nutrient contents in relation to recommended dietary intakes (RDIs) and the nutritional needs of patients
- instructions for preparation and use of the proposed product, including the proposed dilution and scoop size
- a comparison of the proposed product against the nutritional needs of patients, whether given in conjunction with other foods or not
P2.1.1 Main comparator(s) (Subsection 1.1)
The main comparator for a nutritional product is identified according to guidance in Subsection 1.1.3, and is commonly the therapy that prescribers would most replace in practice. In some cases, comparisons with more than one comparator will be necessary, or will provide the NPWP and the PBAC with sufficient information on which to base their recommendations.
The description of the main comparator product(s) in Subsection 1.1 should be based on a relevant amount of nutrient in relation to the RDI rather than to the total product volume. As an example, for an amino acid formula, this description for comparative purposes should be based on a stated protein equivalent, not 100 g of the comparator product(s).
The following sections will help sponsors of nutritional products to select the appropriate main comparator product(s).
Existing products with similar mechanisms of action
If the proposed product is in a class that contains other, already-listed dietary supplements with the same or similar mechanism of action, use the product in the class that is prescribed on the PBS for the largest number of patients in the appropriate age group as the main comparator. A comparison with a more appropriate form (similar in mechanism of action) that is not necessarily subsidised on the PBS, but is available internationally, might provide the NPWP and the PBAC with the necessary nutritional comparison and the necessary scientific data to support an assessment of the proposed product’s clinical effectiveness and safety. However, this comparison would not necessarily inform the economic factors involved in considering the proposed product.
New therapeutic classes
If the proposed product is in a new therapeutic class (eg has a new or additional mechanism of action), use the product that is prescribed on the PBS to treat that indication for the largest number of patients in the appropriate age group as the main comparator. If there is no similarly listed PBS product, a comparison with any other alternative product for which data exist might help the NPWP and the PBAC to make an assessment of the proposed product’s clinical effectiveness and safety. However, such a comparison would not necessarily inform the economic factors involved in considering the proposed product.
No currently listed products
If no currently listed product is available, use standard medical management (this could include special dietary restrictions) as the main comparator. This should be clearly and consistently defined in both the submission and the direct randomised trials.
P2.1.2 Specific medical conditions, if applicable (Subsection 1.1)
Confirm that the formula of the proposed product will supply the protein, energy, fatty acid, vitamin and mineral requirements for a child younger than two years of age. Note that such a child might consume a limited range and amount of food, and so greater volumes of formula might be necessary than for a child on a normal diet.
Malabsorption or disorders of metabolism
Confirm that the formula of the proposed product, containing modified carbohydrate, protein and/or fat content, will supply the protein, energy, fatty acid, vitamin and mineral requirements for the patient if used as a sole source of nutrition. Identify any additional nutritional needs (eg for catch-up growth, other necessary ingredients to meet nutritional needs).
Patients requiring ketogenic diets
Confirm that the formula of the proposed product will supply the patient’s protein, energy, fatty acid, vitamin and mineral requirements if used as a sole source of nutrition. For this to occur, the formula could be multi-ingredient and individually calculated, and a fat source may need to be added (eg Calogen or Liquigen oil emulsions), together with a small prescribed amount of carbohydrates. Patients who can eat foods would need less or no formula after about four years of age, but address the nutritional contribution of the formula in the submission.
Infant formula products, such as a formula used in infants younger than 12 months
Present a table comparing the proposed product with the requirements of the Australia New Zealand Food Standards Code – Standard 2.9.1: Infant Formula Products, using the terminology of the code. Confirm that the proposed product complies with this code or justify any deviations from particular parts of the code.
P2.1.3 Regulatory compliance (Subsection 1.3)
The Australia New Zealand Food Standards Code – Standard 2.9.5: Food for Special Medical Purposes sets out the requirements under these standards for foods that have medical purposes. Confirm that these requirements have been met in Subsection 1.3.
P2.1.4 Product and use (Subsection 1.4)
List of ingredients
For nutritional products, provide information about all the ingredients. In the case of products that will be used to treat allergies or food intolerances, include information on the origin of the ingredients.
Provide a table that lists the micronutrient and macronutrient content per 100 kcal, and per 100 g or 100 mL of product.
Maximum quantity and repeats
Justify the requested maximum quantity and repeats for the proposed product, based on the understanding that these are usually calculated as a one-month supply with five repeats for an infant or child on an appropriate dose to meet the nutritional need for the age range for one of the following:
- total nutrition
- when the proposed product is used in conjunction with solid foods (eg in severe multiprotein food allergy), the amount of product that would be needed to supply total nutrition to children younger than two years of age, and thereafter the expected decreased amount as other foods are introduced into the diet
- when the proposed product is an amino acid supplement used in disorders of protein metabolism, the amount of product that is expected to increase with age and weight, and to be in inverse proportion to the amount of regular foods tolerated.
Tables of RDIs and nutritional needs of patients
Australian RDIs are listed in the Nutrient reference values for Australia and New Zealand.
Present the nutrient contents in tables to allow an assessment of whether the proposed product and its main comparator product(s) provide the required amount of key nutrient for patients for whom the proposed product is intended. Include the following age ranges, as applicable:
- infants younger than one year
- children 1–2 years
- children >2–5 years
- children >5–10 years
- older children >10–15 years
- adolescents >15–20 years
- adults >20 years.
Use the midpoint of the age range. For the non-adult age ranges, compare the nutrient calculations for a child whose weight is on the 50th percentile for weight, using accepted growth charts (eg from the World Health Organization [0–2 years] or the Centers for Disease Control and Prevention [>2 years]). For the adult age range, include pregnancy and lactation tables for the product, unless the product is unsuitable for pregnant or lactating women.
Comparison of proposed product with nutritional needs of patients
For the comparison of the composition of the proposed product with the nutritional needs of the patients who would be eligible to receive it, the key nutrient will vary according to the product. For example:
- for amino acid–type products, the comparison should be based on amino acid or protein equivalents
- for a protein-free supplement, the comparison should be based on an energy index
- for an infant formula, the comparison should be based on the volume that meets the Australia New Zealand Food Standards Code – Standard 2.9.1: Infant Formula Products.
Identify where the proposed product is used in conjunction with other foods. Where this is the case, give the percentage of nutrients provided by the proposed product as proportions of a strict dietary regimen.
Instructions for use
Provide the instructions for preparation and use of the proposed product, including per cent solution (weight per volume), scoop volumetric size and weight of product it holds, and scoops to water volume for a ‘normal’ dilution. Provide the osmolality of the ‘normal’ dilution.