As you may know, Kelly was asked to present to the PMB Review committee on rare diseases. The content of the presentation was very well received by the committee, and we seem to have a few real ambassadors within the committee ensuring our needs are remembered.
Some of the key issues are as follows:
1) We know that the PMB review process is long overdue, and the current review process is taking a long time to progress. The CMS is aware that this is the perception of the stakeholders, and are trying to move the process forward. However, it is of equal importance that this process is deemed transparent, and consultative.
2) Development of the Terms of Reference and Technical process to follow to determine the benefits is underway and should be ready for distribution within the next month. This is to ensure that the “rules of engagement” are clear prior to the benefits actually being reviewed.
In terms of the Rare Diseases discussion, the following items were addresses by RDSA.
1) The need for a local definition for Rare Diseases – so that it can be determined exactly what needs to included from the onset. You can’t discuss a problem without understanding what is included in the problem. At the current stage, rare diseases are not locally defined so its unclear what conditions are being discussed when referring to “rare diseases”.
2) We need improved surveillance, so that we have appropriate data in place to be able to determine the needs of the population impacted with rare diseases. A registry seems to the best way to do this. This is critical to determine the threat and impact of rare diseases.
3) South Africa does not have an orphan drug policy in place. This leaves us with no way to fast track medicines being registered. Sect 21 medications are automatically excluded from PMB level of care currently, and due to the few number of patients receiving them this is unlikely to change. Therefore, we need to develop an Orphan Drug Policy which provides a framework for accessing unregistered drugs.
4) Recognition of supportive and adjunctive therapies for the treatment of rare diseases is important. Very often, a rare condition does not have commercial therapies available and supportive care and symptom control are all that is available.
5) When evaluating treatment options for a patient, the social circumstances needs to be taken into account. Example of such is where an oxygen concentrator is prescribed and considered for a pulmonary condition, but oxygen tanks are declined. If a patient is living in an area with electricity is not readily available, then oxygen tanks should be deemed appropriate.
6) Patients need to understand their treatments, as well as the associated risks and side effects. Patients also need to have consented to receiving the prescribed treatment. Very often, they are removed from the decision making process which can impact compliance and willingness to complete the treatment, which in turn results in high cost medication not being effective.
So what is the way foreward?
1) Continuous engagement with all stakeholders on development on local definition.
2) Development of a registry.
3) Engagement with SAHPRA on an Orphan Drug Policy
4) Continuous engagement with the Diagnostic treatment pairs for rare conditions according to the PMB list.
5) Development of a patient-centric decision tree which can facilitate discussions around suitable treatment methods.
6) Improved development around patient materials to ensure that patients are educated in terms of their consumer rights, as well as the consent process.
7) Possible development of materials and training workshops with CMS to better educate patients on PMBs, patients rights and the complaints process.
A very good thing that came from this meeting was the understanding from all on the PMB review commitee, that no existing benefits currently being received under PMBs currently should be removed, but rather improved.
For more info, please see the attached presentation, and email us at info@rarediseases.co.za.
Link to presentation: https://www.dropbox.com/s/zqavl4ofo79euyg/PMB%20Review%20benefits%20.pdf?dl=0
“If we are serious about leaving nobody behind, we cannot behind those living with rare diseases just because they are few.” – Nata Menabde, Executive Director, World Health Organization