Can’t you just give me the medicine…

By Pekka Simula 13 September 2016

Most people who live with a poorly treated disease keep searching for potential new treatments. When there’s nothing else there can at least be some hope.

At Herantis, we develop novel treatments for several indications with unmet clinical needs. So patients and their families from all over the world frequently enquire about our drug candidates. When do we expect our drug to be marketed globally? Are clinical studies ongoing and how could one participate? And the most desperate ones: Can’t we just give our investigational drug to a patient who has no hope and therefore couldn’t care less about the risks?

As a very patient-centric company we take pride in responding to every such request, doing our best to explain what we can do and why our hands are often tied. That raises new questions; so I will now try to elaborate a bit more on some of the common challenges and constraints in the development of innovative new drugs.

Before market approval, drugs must be carefully tested in the clinic. This is generally known and understood. Our drug candidates are still in the development phase so we cannot sell them to anyone, which is easily justified. It wouldn’t be ethical to make a profit on desperate patients as we don’t yet have sufficient evidence on the safety and efficacy of our products in the treatment of human patients. So we will politely reject even those enquiries where “money is not an issue.” Not only would it break the law to sell them our products; it would simply be wrong.

We can however provide the investigational drugs to patients in clinical studies provided we have all necessary approvals by regulatory authorities, ethics committees, etc. Even then a large majority of patients is unfortunately turned away. The clinical development process is extremely strict and regulated with patient safety always the #1 priority. One practical consequence is that clinical studies have very constraining patient inclusion and exclusion criteria, which easily rule out 95% of the seemingly eligible patients. Risks of the unknown are associated with any novel therapeutics; even if preclinical data suggest a safe treatment it could potentially have implications for instance on the patient’s blood pressure, or liver function, or heart rate, or any other vital system. Therefore a patient with a medical history of, say, blood pressure will typically be ineligible: An unexpected impact on blood pressure might be serious for such a patient while it could be quite easily treated in an otherwise healthy patient. This is why patient recruitment can be very slow in clinical studies. It can be a challenge to find patients who are at the same time seriously ill and relatively healthy…

For the reasons above we will not sell the investigational drugs to anyone; and we can only enroll a small fraction of interested patients in our clinical studies. And unfortunately we can absolutely not recruit pediatric patients in our present clinical studies. Much more safety data in adults would first be required.

Isn’t there any other way?

Couldn’t we just give the drugs to a desperate patient who really doesn’t mind the risks? That would at least give some hope to such patients and their loved ones. Take ALS as an example. ALS diagnosis is basically a death sentence with such projection that many patients would probably accept a treatment with a 10% chance of significant benefit against 90% immediate mortality.
The first challenge here is very practical. For instance our biological drug candidates are manufactured in small batches at this early stage of development. We still need to comply with all commercial drug manufacturing requirements, i.e. basically a full-blown drug factory with all bells and whistles and tests. That means an extremely high unit cost. In this regard we’re like a boutique car manufacturer who wants to build just five cars of a unique model; if a safety directive requires first wrecking a hundred of those cars in crash tests the unit price increases quite a bit!

Even if that challenge was overcome there’s still a far greater challenge, an ethical challenge. Think about the word desperate. What if we provided the treatment to a dozen desperate and all-risk-accepting patients outside of clinical studies, and everything went perfectly with no serious adverse events?

At least some of these patients would probably show some benefit – even if we had given them saltwater. There’s always a placebo effect, which can be amazingly strong. And we humans tend to see things we want to see. Family members would believe, and convince each other, that dad is definitely improving thanks to the treatment, because they would want to see improvement. Suddenly the word would spread, tabloids would make headlines on a miracle drug, and patient advocacies would go crazy requiring the authorities to immediately approve this miraculous treatment with no scientific basis. No-one would notice if the patients then actually declined; and questionable clinics would cash on those headlines and internet stories by providing the treatment at a high price in territories with a less strict legal environment. They would do exactly what we don’t want: Make a profit on desperate patients.

It is absolutely heart breaking to explain this all to a parent who begs for the last chance for a child suffering from an awful, unfair disease. We just have to do what we believe is right. We will focus on honest drug development and hope one day our response is: Yes, we can and we will help you.

Herantis is currently recruiting patients in Finland in a clinical study in breast cancer associated lymphedema, and plans to start recruiting patients in a clinical study in Parkinson’s disease in Sweden and Finland in the near future. Herantis also hopes to launch a development program in the treatment of ALS.