The specifics of early access programmes
pharmafile | December 3, 2010 | Feature | Sales and Marketing |Â Â Expanded Access Programs, IDIS, early access programmes, named patient programmesÂ
With increasing requests from healthcare professionals and patients globally for access to drugs that are still in clinical development or awaiting approval, pharmaceutical and biotechnology companies can no longer sit back and wait until launch. Responsible and sustainable early market access solutions are required to give a drug the right start.
As more and more pharmaceutical and biotechnology companies seek ways to respond to early access pressures from patients and physicians, it is imperative that they do so by implementing strategies that enable them to respond in a responsible and ethical manner.
Regulating bodies worldwide have created mechanisms for granting access to medicines not currently licensed in their country. These mechanisms fall under the titles of Named Patient Programmes (NPPs) outside the US and Expanded Access Programs (EAPs) in the US (also referred to as early access, managed access, named patient supply, and compassionate use programmes).
If early access is provided in a timely, regulated, ethical and sustainable way, there are clear benefits for the three key main stakeholders – patients, physicians and pharmaceutical and biotechnology companies:
• For patients, these programmes can literally mean the difference between life and death, and access to pre-licensed medicines can offer them a new treatment option when all other therapies have failed.
• For the physician, these programmes provide them with an opportunity to give their seriously ill patients another chance. Also, they can gain experience with a new drug and better understand its potential before the drug becomes more widely available.
• For the pharmaceutical and biotechnology companies, early access programmes allow them to give access to pre-licensed medicines outside of clinical trials in response to requests from clinicians wanting early access to new agents. Data collected from the programmes can offer novel insights into the profiling of the product which can be used to shape its development.
This is why the pharmaceutical industry is increasingly building early access programmes into their product development plans at an early stage. Despite an ongoing debate about the intricacies of early access, most stakeholders would agree that this is a good approach for all parties involved.
Opportunities
Early access programmes can provide real life data to support market forecast models pre-launch, as well as providing an opportunity for testing the quality, allocation and supply chain of pre-launch drugs.
Well-planned programmes enable companies to include a risk management strategy, which facilitates a controlled route to pre-launch medicines.
This protects against parallel trade and, importantly, reduces the risk of patients accessing counterfeit drugs.
Early access programmes can provide an opportunity for a company to demonstrate the benefit of its drug to physicians and health economists from its first use in patients. Greater patient numbers, coupled with longer trial periods, establishes a more thorough safety and efficacy profile. Appropriate health economic data collected before marketing authorisation can also help with reimbursement post-approval.
Early visibility of pre-licensed medicines will increase physician awareness of the drug before it has even launched, creating an opportunity for the company to develop close relationships with those physicians and gain valuable feedback about its administration and effects.
Healthcare practitioners who have experience of the product may well become early adopters, using the drug earlier and also with more patients once it is licensed.
In this way, physicians are well educated in the safe use and administration of the drug, which in turn will ensure patients have access to the most appropriate treatment at the right time to ensure the best patient outcome.
Whilst the opportunities provided by early access programmes are considerable, challenges also exist. Numerous legal, regulatory and logistical challenges across different countries need to be considered – and specialist expertise is required when developing these early access management solutions through NPPs or EAPs.
An increasingly important approach
Effectively designed and well run early access solutions benefit patients; healthcare professionals; and pharmaceutical and biotechnology companies.
For these companies a well designed NPP or EAP can ultimately give their drug the right start with their customers, and also enables them to respond ethically, effectively and in a timely way to patients’ needs.
Simon Estcourt is senior vice president and director at Idis, and can be contacted by email at: sestcourt@idispharma.com
Related Content
Clinigen buys Idis for £225 million
UK speciality pharma group Clinigen is paying £225 million to acquire the Surrey-based unlicensed supplier …
IDIS and Ogilvy to promote named patient programme
Specialist medicines supplies company IDIS has hired a leading PR agency to help promote its …
