When it comes to medicines access, numerous factors may delay or prevent medical products from reaching patients. It is the role of medicines access professionals to manage these disruptive factors and navigate obstacles to ensure patients receive supplies of vital medicines and drugs when they need them, wherever they are located.
Supply disruptions can have serious consequences for the long-term health of patients. An example of a supply disruption could be the time between a clinical trial ending and when the product gains regulatory approval in the patient’s country so they can receive their next treatment. Furthermore, the product may not get approved in the patient’s home country. This is where medicines access solutions can help.
Professionals working in medicines access must be fully aware of the constantly moving hurdles. Even minor changes in documentation can cause significant delays and prevent products from being delivered in a timely manner or at all, and patients, such as those with cancer who may not be able to wait for treatment. This is why the flexibility to adapt is so important in successfully delivering access solutions.
An additional challenge can be the lack of understanding among some smaller pharma companies and manufacturers about the mechanisms for providing access to medicines on unlicensed, named patient or compassionate grounds. While they may have the products that can make a difference, they could lack the available resources to get them to patients before commercial launch. There may also be misconceptions about exactly what the access process involves and how to instigate it.
“Many companies don’t have that knowledge in-house; providing access to medicines pre-licence is a niche area. There is often a fear of taking part in access programs without really understanding the breadth of regulatory guidance that supports providing pre-licence access for patients,” says Rebecca Bibby, General Manager for BAP Pharma, who was heavily involved with establishing the company’s specialist Medicines Access Division.
Issues with regulations for investigational medical products
When providing medicines access solutions, speed is of the essence. Any delays with health authority approval or customs can have a serious impact on the condition of a medicinal product and the wait may also be damaging for patients. Issues with regulations and documentation for specific countries are a recurring challenge that can only be resolved by experts with knowledge of the intricacies of regulatory small print and operations on the ground. Navigating such issues takes specialist skills and experience that even seasoned drug manufacturers and pharmacists may not have.
Regulations differ from one country to the next – even if they are in the same trade bloc. Regulatory issues within the UK resulting from Brexit are widely reported. France is a prominent member of the European Union, but it has different rules around access to other member states. France has made changes to its access processes in recent years, with more restrictions creating a more defined process for where access is permitted and commitments from manufacturers surrounding marketing authorisation submissions relating to the provision of access.
“There is a perception that because a country is an EU member state that the pre-licence access or early access regulations that must be adhered to will be aligned country by country,” says Bibby. “This is simply not true.
“Each country has very different mechanisms for access. They have their own unique regulations that need to be followed. If they are not fully understood or adhered to, the access solution could have an impact later in the commercialisation of a product. For example, in France, if there’s a price the product is made available at in that market for pre-approval access, the ANSM expects a commitment that the price will remain the same when the product launches and that it does not increase.
“This can cause difficulties for in-house commercial/launch teams within a manufacturer or pharmaceutical biotech company that wants to make the product available. As a result, it can negatively impact launch plans and delay making a product available in the market.”
Dealing with cost issues in medicines access
Costs have traditionally been a barrier to pre-licence medicines access. Due to the financial pressures that companies have been under in the aftermath of the global pandemic, they may not want to consider access participation due to the perceived expense involved. There can be some uncertainty as to who should pay for treatment and at what price. In addition, rising energy prices and shipping costs are making the supply chain and production more expensive in general.
“When we’re dealing with medicines access programs, often it’s either a health authority or a patient who has to pay, or via personal insurance. The escalating costs of even physically shipping the product to the patient are having an impact because it has to be absorbed somewhere,” says Bibby.
“We’re trying our best to keep the most cost-effective and efficient supply chain. This again, is something we see as a constant challenge within not just the clinical trials space we work within, but also the medicines access space.
“When a pharmaceutical company is supportive of an early access program or access to a medicine pre-licence, the medicine often has to be supplied on a free-of-charge or compassionate use basis. In the current climate, many smaller biotech companies can’t afford to do that,” she adds.
However, it is possible for pharmaceutical companies and drug manufacturers to generate revenue through medicines access during the clinical trial phase before regulatory approval and commercial launch. While potential proceeds are not comparable with a commercially available product, the possibility of gaining revenue from access to investigational medicinal products (IMPs) could be enough to persuade companies to get involved in programs.
“For smaller companies, there is a real squeeze on budgets. So, any revenue that can be brought back into the organisation for research or development purposes is really important,” says Bibby. “We’ve had programs that have brought in significant revenues and those that have brought in small revenues. It really depends on the product, demand, and how long the access program is open.
“It isn’t going to return the multi-billions that we used to see from the ‘big blockbuster’ molecules. But it’s certainly revenue that is significant – especially when every penny counts.”
In addition, real-world data from medicines access during clinical trials is another notable benefit. It can help to support the regulatory application of an IMP and may even help to speed up the approval process. Real-world data can also provide a useful indicator of the potential success of a product in a particular market.
To unlock the full benefits of medicines access, BAP Pharma suggests having discussions about this in the early stages of the clinical trial planning process.
How BAP Pharma can help with medicines access
What provides BAP Pharma with a distinct advantage in medicines access is that the company was founded in 2011, by Dr Bashir Parkar, to focus on supplying clinical trials with high-quality supplies of comparators. Since then, the company has expanded to launch a Medicines Access Division, as well as a Packaging and Labelling Division. This experience provides a thorough understanding of regulatory frameworks and allows the company to keep track of the latest changes while remaining ahead of the curve when it comes to medicines access.
“There was a gap in this access space for a truly bespoke, transparent provider of medicines access or named patient programs that work alongside the client in the very same way that we do in our Comparator Division and our Packaging and Labelling Division,” adds Bibby. “We think about both the client and the patient at all times. Everything we do centres around that.”
The company is UK-based, but operates globally, and has the capabilities to send products wherever in the world they may be required – in compliance with all national and international regulations. Furthermore, the company’s motto of ‘Promise Delivered’ ensures teams will never commit to a project unless there is certainty that it can be achieved.
“We have a truly global reach. From our BAP Pharma-owned facilities globally, we can leverage that when providing medicines access solutions. We have extensive experience in-house and know the mechanisms to supply medicines compliantly to any market across the world where there is a patient or demand for a medicine to treat an unmet clinical need,” adds Bibby.
“Patients are at the heart of everything we do. At BAP Pharma, we are committed to providing access to much sought-after medicines – compliantly, efficiently, and quickly. Our extensive experience and established networks for managing complex supply chains ensure we are successful when providing medicines access solutions. This is something the whole BAP team are proud of, and all find hugely rewarding.”
To learn more about BAP Pharma’s medicines access capabilities, download the specially commissioned whitepaper below.