Striking a Balance in Post-Trial Access

4th October 2016 (Last Updated July 18th, 2018 11:54)

Marie Eklund, Head of Clinical Operations, Boehringer Ingelheim, briefly discusses post trial access in South Africa

Striking a Balance in Post-Trial Access

As has been discussed in previous articles, the South African trial environment is attractive to pharma companies for study purposes. This is due to the well-established, highly experienced trial network of doctors, as well as the variety of pathologies offered in the potential patient population – there are developing world pathologies as well as 1st world pathologies, all spread over a population of 55 million people.

However, recently there have been growing calls from regulatory bodies and physicians alike, for pharma companies to offer post-trial access (PTA) to all patients, including those who have insurance (i.e. not state patients) and benefit from the study medication during the trial. As a result, a new guideline, developed by the MCC (Medicines Control Council), is being reviewed and discussed in an effort to deal with this issue. What the regulatory guideline is potentially requiring versus what the pharma industry is currently offering is at this stage not in alignment. Therefore, a balance needs to be found that is mutually acceptable to all parties. Although none of these issues are insurmountable, there needs to be open dialogue and a greater understanding from both sides.

Let’s be frank, pharma is a business not a philanthropy. Even though most pharma companies do a fair amount of social welfare outreach and support programs, they need to get a return on the investment they make in developing a drug. On the other hand, both state and medical aids have a responsibility to provide care for their patients whether they are participating in a trial or not. So, it is understandable they expect a medication – that is obviously beneficial to patients – not to be stopped abruptly at the end of a trial before the medical aid or state can supply it. The costing of drugs is one of the main issues here. Understanding the need to recoup the costs of development and production while balancing that with realistic pricing would allow wider access in a developing world scenario (accepted that this is a simple analogy that does not take into account the issues of legislated pricing, government tenders, etc.).

So, how can we reach a solution? Let’s address some of the issues that need to be discussed and detailed to get there.

What is the overall ‘benefit,’ and who judges and measures that? Who decides that benefit is not solely down to having easier access to a doctor’s care (regular visits, no long queuing, more individual ‘attention’) not only leading to greater compliance in taking medications but also with non-medical management (diet, exercise, smoking  etc)? Obviously some benefits are easier to measure and are more obvious than others, but the question remains, what benefit are you actually measuring, and is it at a level where it would be ethically acceptable to keep the patient on an unregistered drug for any period of time?

What drugs would be classified as life threatening if withdrawn? For those post trial patients where it is truly immediately life threatening, it is usual to arrange section 21 access (section 21 refers to a regulatory application where you can import an unregistered drug, for South Africa, for named patient use outside a trial environment). As this is quite a capacity intensive process that can only be used if the drug is registered elsewhere, it cannot be seen as the best way to deal with the majority of PTA. Rollover protocols can only be established once some data (efficacy and safety) from the trial has been made available – which is usually at database lock, and well after the patient is off the trial drug in most cases. For instance, would diabetes and asthma be considered life threatening, even in the face of a lot of alternative registered treatments being readily available, even in state?

The cost of supplying PTA as well as the cost of managing the safety profile and the logistics (insurance, labelling, IMP importation, contractual arrangements with doctors) of long term supply of an unregistered drug might be seen as a potential deterrent to do trial work in South Africa, something we cannot afford. Maybe a more structured outline of what and for how long needs to be supplied would allow for easier and more transparent budgeting and planning. Possibly there should be a set timeline for PTA, for example, two years and with renegotiation each two years using updated safety and efficacy data as well as availability of comparative drugs.

Could tax refunds on pharma R&D potentially be used to fund PTA programs? There are tax rebates available on certain aspects of clinical research – this would incentivize companies to continue with a high volume of trials in South Africa, as they will recoup some costs, and could plough some of that returned money into PTA overhead costs.

This has not been intended to be anything more than a superficial scratching of the proverbial PTA surface, and has not really delved into all the other related issues that also need to be discussed. Take for instance, orphan drugs, where you have a drug developed for a rare disease which makes them not profitable to market and thus limits their availability. This perversely incentivises patients to enter trials purely to access free long term medications.

Where does the buck actually stop when it comes to medical care and drug access? Is it ethical to test drugs that will be unaffordable to the majority of patients? Or is it ethical to NOT test, thus denying the professionals and patients the exposure to new drug developments?

What we do need is to make a concerted effort to meet the needs of both sides as much as possible. It’s essential we do not suffer any form of underinvestment in clinical research in South Africa, while ensuring patients are safe giving them longer term access to beneficial medications.

 

 

*The views in the article are solely mine and don’t reflect Boehringer Ingelheim