In this article I will describe;
What Medical Access Programs are
Who they benefit
The role of the medical science liaison in Medical Access Programs
Guidance around the management of Medical Access Programs in different parts of the world
How you can learn more about typical medical science liaison activities
What is a medical access program?
Usually, a medicinal product needs to be registered (authorised) in a country in order to be used. However, this means that certain patients might be deprived of potentially lifesaving or life-prolonging medications where there is no alternative available.
Medicine Access Programs enable patients to get access to treatments which are not yet authorised in that country. In some cases, medicine access programs enable patients to access medicinal products for free before they are reimbursed (payed for by the government).
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What are the benefits of medicine access programs?
The benefits to medicine access programs can be huge and there are 4 main groups who benefit.
Patients gain access to treatments which could be potentially life changing for them.
Physicians are given another option to offer to their patients, and they also gain valuable early experience with a product.
Payers (stakeholders which finance medicinal products e.g. government ministers deciding the price or reimbursement of products, administrators within health technology assessment bodies, medical insurance companies or pharmacists in a hospital who helps determine which products the hospital uses). Payers benefit from having the opportunity to evaluate outcomes in a real-world setting outside of clinical trials, and gain an insight into the potential value of the drug.
4. The Pharma Company
The pharma company gets local KOLs to gain clinical experience with their product. If these KOLs have a positive clinical experience and patients respond well to treatment, the KOL will likely be an early adopter or advocate and use their influence to educate other KOLs on the most appropriate types of patients to treat. Medicine access programs can also help pharma companies have good launch preparedness and also offer the opportunity to develop real world evidence which can be used for reimbursement and pricing discussions.
What is the role of the MSL in medical access programs?
Please note that the role of the MSL in access programs will vary from company to company and from country to country. Always adhere to your company policies.
The responsibility of the MSL in access programs can include:
Identification of suitable KOLs
Engaging KOLs to be involved
Explaining the process to KOLs
Liaising with colleagues in the medical/pharmacovigilance department to ensure all adverse events will be reported in a timely manner
Regularly meeting with KOLs to understand their clinical experience and their patients experience with the drug
Sharing of insights with the rest of the pharma company
Liaising with KOLs to enable them to educate other healthcare professionals (HCPs) about their clinical experience (where appropriate) via webinars/conferences
Where appropriate and possible, managing the collection and publication of real world data from the access program. It is important to note that real world data is has lots of limitations and there are often significant challenges with robust data collection.
How to choose which KOLs should be involved in the access program?
As an MSL, you will want to identify KOLs who have the following characteristics;
Extensive experience treating the disease of interest
A clear understanding of the medical product and the most appropriate patients
Highly influential, so that they can share their clinical experience to educate other KOLs
Ideally you also want to have a spread of KOLs across different geographical locations and institutions. Furthermore, you want to choose KOLs that are passionate about the new treatment. Choosing a KOL who has experience with medical access programs is always a good idea as it will ensure the whole process will run smoother.
Examples of different medicines access programs throughout the world
Australia: Special Access Scheme (SAS)
Special Access Scheme (SAS)
The Special Access Scheme (SAS) provides patients in Australia with access to unapproved therapeutic goods.
It relates to individual patients, not groups of patients.
Health care professionals submit SAS applications for individual patients on a case by case basis.
Individual patients cannot apply for access to unapproved therapeutic goods through the SAS, they must consult with their doctors about the suitability of using an unapproved therapeutic good.
Relevant guidance authority: TGA
Australia: Product Familiarisation Programs (PFP):
Product Familiarisation Programs are another type of medicine access program available in Australia. The aim of product familiarisation programs is to allow the medical profession to evaluate and become familiar with a product. They are often run by pharma companies when a product has been registered but has not yet been reimbursed. Product Familiarisation Programs benefit the patient because they get free access to medication that is not reimbursed.
There are some guidelines to keep in mind with regards to PFP in Australia:
PFPs must involve patients being treated for approved indications of the product
If the Product Familiarisation Programs will be prescribed in hospital a formal agreement should exist between the pharmaceutical company and hospitals
Pharma companies must not offer any monetary or any other type of reward to healthcare professionals, their families and/or employees for taking part in PFPs
Suspected adverse drug reactions reported during the PFP must be reported to the TGA
Relevant guidance authority/code: MA Code of Conduct
USA: Expanded Access Program (EAP)
Expanded access is a potential pathway for a patient with an immediately life-threatening condition or serious disease or condition to gain access to an investigational medical product (drug, biologic, or medical device) for treatment outside of clinical trials when no comparable or satisfactory alternative therapy options are available.
Expanded access may be appropriate when all the following apply:
Patient has a serious disease or condition, or whose life is immediately threatened by their disease or condition.
There is no comparable or satisfactory alternative therapy to diagnose, monitor, or treat the disease or condition.
Patient enrolment in a clinical trial is not possible.
Potential patient benefit justifies the potential risks of treatment.
Providing the investigational medical product will not interfere with investigational trials that could support a medical product’s development or marketing approval for the treatment indication.
Relevant guidance authority: FDA
Germany - Compassionate use program
Only relevant for groups of patients
Patients must have a seriously debilitating or life-threatening disease which cannot be treated satisfactorily by an authorised medicinal product
The compassionate use programme ends at the latest after one year
The pharmaceutical company initiates the process
Relevant guidance authority: BfArM
FAQ: Is compassionate use the same as off-label use?
No. Off-label use refers to when a product is registered (authorized) for a certain indication and it is used for a different indication. An example of off-label use would be if Drug X is indicated to treat breast cancer and an oncologist prescribes it to treat lung cancer.
Compassionate use is the use of a product that has not (yet) been authorized at all.
Medical Access Programs are a fantastic way to enable suitable patients to gain access to treatments which are not yet registered or reimbursed. MSLs play a key role in the facilitation of medical access programs.
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