New Medicines Profile: Template Guidance Notes
Information on New Medicines
New Medicines Profile
Template Guidance Notes
The profile should not exceed two pages, excluding the references and appended tables. Text should be 9-10 point size. 8.5 is an absolute minimum.
Summary
The summary is laid out in bullet points. It should accurately reflect the key points in the text and should not introduce any new information. The summary should include the following:-
- A very brief description of what the medicine is and what it is indicated for.Information on whether it is a new class of medicine, new indication or new formulation and why the product may have been launched e.g. patent expiry of existing product, may also be useful.
- A brief outline of efficacy and safety.
- An indication of any advantages, clinical or practical, the drug may have over existing therapies. A comment on its likely/potential place in therapy.
- Cost implications.
The summary should leave the reader with a clear message about the medicine even if they do not read the rest of the evaluation.
Brand Name, (Manufacturer)
Cite as e.g. Exelon, (Novartis)
BNF Therapeutic Class
Use BNF wording and section number or SPCdescription.
Licensed Indications
As per SPC, abbreviate if necessary.
Dosage and Administration
As per SPC
Marketed
Insert month and year
Cost Comparisons
Generally give the cost for 28 days but annual costs may be used for chronic therapy. State where the prices are from (usually the latest edition of MIMs or the Drug Tariff). Select no more than 5 comparator medicines and include mid-range SPC doses or give ranges (not Defined Daily Doses).
N.B. Doses shown for general comparison and do not imply therapeutic equivalence
Introduction
Briefly describe what the medicine is and what it is indicated for. Outline existing treatment options and include prevalence ofdisease, if appropriate.
Evidence
Give a brief overview of the major studies (phase 3). Phase 2b studies should only be included as a last resort. Do not include dose ranging studies.
The text should include a brief description of the trials and critique the trial data rather than detail them in depth. Use the table (appendix 1) to present details of the trials.
- Read papers thoroughly and conduct a fair, independent critical evaluation of the data. Ensure all statements can be corroborated.
- Do not express results as relative risks if possible. Try and use absolute risks and/or numbers needed to treat (NNTs) if appropriate. Do not rely on published NNTs - re-calculate.
- If there are no comparative trials this should be stated.
- Use fully published randomised controlled trials (RCTs) where available.
- State if using conference abstracts/posters or company ‘in-house’ data, although the latter should only be quoted as a last resort. The limitations of such data should be pointed out e.g. this requires confirmation in published studies.
- Include details of any patient impact assessments if possible e.g. quality of life studies.
- Include comment on pharmacoeconomic studies. Highlight whether studies are independent or sponsored and state if there are none.
Checklist to help critically evaluate a study:
a)Why was the study done?
b)What type of study was it?
c)If the inclusion and exclusion criteria were very strict what implication could this have for real world clinical practice?
d)What were the outcome measures? Were they appropriate? Were theypatient orientated e.g. death, MI, or surrogate measures e.g. blood pressure?
e)Were scoring systems e.g. QoL questionnaires etc used validated methods?
f)Were the numbers recruited sufficient?
g)What was the drop-out rate? Were all drop-outs accounted for?
h)Was the study adequately controlled?
i)Was the study randomised - was the randomisation method described and appropriate?
j)Was there a pre-randomisation study phase and what was the purpose of it? Were all potential subjects exposed to the study drug and only those who tolerated it then randomised?
k)How was the study ‘blinded’? Were there any factors that might have jeopardised blinding e.g. was the active drug associated with a high frequency of a particular adverse effect?
l)If there was a non-placebo comparator, was it a fair choice and used at an appropriate dose? Was the comparatoranestablished evidence-based alternative?
m)Were the study groups comparable?
n)Was the analysis done on an intention-to-treat or a per protocol basis? What influence may the latter have had on the results?
o)Has any sub-group analysis potentially introduced bias?
p)Is the study credible? What is the quality of the results? How relevant are they to clinical practice?
q)Are there confidence intervals?
r)Are the results of the study consistent with other similar studies?
Highlight any study deficiencies in the text.
Avoid:
- Quotation out of context.
- Biased selection of studies.
- Uncritical acceptance of conclusions.
- Representation of opinion as fact.
All sources should be fully referenced.
Safety
Include the major and most common adverse effects with an indication of frequency, if possible. Refer to the SPC where appropriate. Include clinically important interactions. State if long term safety data are lacking. Include monitoring requirements if appropriate.
NHS Impact
Briefly discuss the existing treatment option(s) of choice for the condition and comment on the new products likely place in therapy. Include a statement as to when (or indeed if) it should be prescribed and to whom i.e. does it offer any advantages, clinical, service or financial, over existing therapies?
Include those issues that local decision makers need to take into consideration. These could include:
-likely target population
-disease prevalence
-limits on licensed indications compared to existing comparators
-limits on who should prescribe it
-relevance of drug comparators used in clinical trials
-relative cost
-any service implications (positive or negative)
Recommendations should clearly reflect best available supporting evidence.
Include the status and anticipated date of any NICE guidance.
Risk Management Issues
If applicable, discuss any issues which may increase the risk of medication errors. This could include information relating to compliance, packaging, administration and drug name. Other issues include whether any particular disposal methods are required, if there are environmental issues e.g. chemotherapy, or if disposal may have an adverse effect on the environment e.g. hormonal products.
Include a picture of the product if it illustrates a risk issue or is a novel presentation.
Author Details
Insert author and name, address, telephone number of Medicines Information Centre.
References
Reference all sources used and highlight the key papers in bold.
References are numbered in the order they appear in the text. The citation number should be placed in the text at the end of the appropriate sentence in superscript, after the full stop.
References must be cited in the Vancouverstyle; using Index Medicus abbreviations for journal titles (see following examples).
List the first three authors, followed by “et al” if there are more than three. Give the full title of the article,using US spelling if in the original. This is followed by the title of the journal, year of the publication, the volume number and the first and last page numbers in full. References to books should be given the names of the authors, any editors, the title, edition, place of publication and year. For references accessible via the web include a web address together with the date accessed.
Examples:
Journal Articles
- Janne PA and Mayer RJ. Chemoprevention of colorectal cancer. N Engl J Med 2000; 342: 1958-1968.
Abstracts
- Raskin P,Kapp A, Gupta K et al. The effect of HOE 901 on glycemic control in type 2 diabetes. American Diabetes Association 58th Annual Meeting. Chicago, Il. 13-16June 1998. Abs 404.
Books
- Mehta DK, editor. British National Formulary No. 52. London: British Medical Association and Royal Pharmaceutical Society of Great Britain, September 2006.
On-line Resources
- Summary of Product Characteristics: Baraclude, Bristol Myers Squibb Pharmaceuticals Ltd June 2006. Accessed via on 20/03/07.
- National Institute for Clinical Excellence. Dyspepsia: Management of dyspepsia in adults in primary care. Clinical Guideline No. 17. August 2004. Available at:
Accessed 20/03/07.
- Exubera, European Public Assessment Report Scientific discussion. Available at:
Accessed 25/05/06.
Personal Communication (do not include name of person unless they have consented)
7.Personal Communication 20/03/07, Janssen-Cilag Ltd
Appendix I: Table of Clinical Trials
The table should be headed with a number and a title.
The aim of the table is to present details of the trials in a concise manner. Some information mayneed repeating in the text.
The table should be fully explanatory so that it can be understood without reference to the text. If necessary a key should be included.
The table may be adapted as necessary but should include the following data as a minimum: ref no, trial design, trial population, treatment regimen, primary outcomes.
Additional headings which may be required include: inclusion/exclusion criteria, secondary outcomes, comments.
The following examples of tables illustrate appropriate alternative layouts.
Date of preparation: August 2003
Last updated: July 2007
New Medicines Profile: Template Guidance Notes
Example I: Table of key clinical trials evaluating omalizumab in patients with severe persistent allergic asthma
Ref No / Trial Design / Common Criteria - all trials / Additional Criteria / Outcome Measures / ResultsRef 5
INNOVATE / Multicentre, double-blind, RCT over 28 weeks
omalizumab vs. placebo
4 phases:
1 week screening
8 week run-in
28 week treatment
16 week follow-up (unpublished) / Inclusion Criteria
Age 12 – 75 years
Positive skin prick test to ≥ 1 perennial allergen
Moderate to severe allergic asthma*
Duration of asthma ≥ 1 year
Total serum IgE ≥ 30 IU/ml to ≤ 700 IU/ml
FEV1 ≥ 40 to ≤ 80% of predicted normal value and continuing asthma symptoms
FEV1 reversibility ≥ 12% from baseline within 30 minutes after administration of inhaled β2 -agonist
Exclusion Criteria
Prior exposure or sensitivity to omalizumab
Elevated IgE levels other than atopy
*See individual trials for further details / n=482 at entry
n=419 efficacy analyses
Additional Inclusion Criteria
Severe persistent asthma (GINA 2002 step 4#), receiving ICS and LABA (100% patients) and additional controller medications including oral corticosteroids (22% patients)
At least 2 exacerbations requiring systemic corticosteroids or 1 severe exacerbation resulting in hospitalisation or emergency room treatment in past 12 months
Additional Exclusion Criteria
Smoker
Treatment for an exacerbation within 4 weeks of randomisation / Primary outcome / Omalizumab / Placebo / P value
Rate of clinically significant asthma exacerbations during treatment phase / 0.68 / 0.91 / 0.042
Rate ratio 0.738 (95% CI: 0.552-0.998)
Secondary outcomes
Severe exacerbation rate (PEF or FEV1<60% requiring treatment with systemic corticosteroids) / 0.24 / 0.48 / 0.002
Total number of emergency visits / 50 / 93 / 0.038
Rate ratio 0.561 (95% CI 0.325-0.968)
Hospital admissions / 13 / 25 / Not sig
Asthma-related QoL using Juniper AQLQ instrument
>0.5-point improvement from baseline / 60.8% / 47.8% / 0.008
Improvement in FEV1 / 190ml / 96ml
Ref 2
Busse et al / Multicentre, double-blind, RCT over 28 weeksomalizumab vs. placebo
4 phases:
4-6 week run-in
16 week stable steroid phase
12 week ICS reduction
24 week extension / n=525
Additional Inclusion Criteria
Severe allergic asthma requiring daily ICS
Treatment with beclometasone dipropionate 420-840mcg/day or equivalent ICS for > 3 months prior to randomisation
Additional Exclusion Criteria
Acute upper respiratory tract infection within 1 month
<3 months stable immunotherapy, regular treatment with β2 blockers
Required dose omalizumab >750mg / Primary outcome / Omalizumab / Placebo / P value
Mean number of exacerbation episodes per patient in stable steroid phase / 0.28 / 0.54 / 0.006
Secondary outcomes
% of patients experiencing at least one exacerbation in stable steroid phase / 14.6% / 23.3% / 0.009
% of patients achieving >50% reduction in beclometasone dipropionate dose / 72.4% / 54.9% / <0.001
Mean change in PEF at week 16 / 18.5L/min / 6.9L/min
Example II: Published short-term efficacy studies
Ref No / Trial Design / Trial Population / Treatment / Primary Outcomes / Comments1 / Randomised, open-label, 6-month study / 328 patients with type-1 diabetes receiving a stable regimen of SC insulin /
- Morning and bedtime SC NPH plus pre-prandial INH or
- Morning and bedtime SC NPH plus pre-prandial SI.
2 / Randomised, open-label, 6-month study / 335 patients with type-1 diabetes receiving a stable regimen of SC insulin /
- Pre-prandial INH plus a single bedtime dose of SC insulin zinc suspension or
- Two to three daily injections of a SI/NPH insulin regimen.
3 / Randomised, open-label, 6-month study / 299 patients with type-2 diabetes receiving a stable regimen of SC insulin /
- Pre-prandial INH plus a single bedtime dose of SC insulin zinc suspension or
- At least two mixed SI/NPH injections per day.
4 / Randomised, open-label, 3-month study / 309 patients with type-2 diabetes uncontrolled on dual OA therapy /
- Pre-prandial INH only or
- Pre-prandial INH plus OA (at existing doses) or
- OA only.
*(P<0.001 for difference vs. OA only) / Changing to, or adding, insulin is standard practice in people with type 2 diabetes poorly controlled on OA therapy.
5 / Randomised, open-label, 3-month study / 68 patients with type-2 diabetes inadequately controlled on OA /
- Pre-prandial INH plus OA (at existing doses) or
- OA only.
(P<0.001 for difference) / As above